fenfluramine has been researched along with Cryptogenic Infantile Spasms in 17 studies
Fenfluramine: A centrally active drug that apparently both blocks serotonin uptake and provokes transport-mediated serotonin release.
fenfluramine : A secondary amino compound that is 1-phenyl-propan-2-amine in which one of the meta-hydrogens is substituted by trifluoromethyl, and one of the hydrogens attached to the nitrogen is substituted by an ethyl group. It binds to the serotonin reuptake pump, causing inhbition of serotonin uptake and release of serotonin. The resulting increased levels of serotonin lead to greater serotonin receptor activation which in turn lead to enhancement of serotoninergic transmission in the centres of feeding behavior located in the hypothalamus. This suppresses the appetite for carbohydrates. Fenfluramine was used as the hydrochloride for treatment of diabetes and obesity. It was withdrawn worldwide after reports of heart valve disease and pulmonary hypertension.
Excerpt | Relevance | Reference |
---|---|---|
" Before the approval of fenfluramine (FFA) for the treatment of seizures in DS, patients in Germany could receive treatment under a compassionate use program (CUP)." | 8.02 | Efficacy, tolerability, and retention of fenfluramine for the treatment of seizures in patients with Dravet syndrome: Compassionate use program in Germany. ( Alber, M; Klotz, KA; Kluger, G; Kurlemann, G; Mayer, T; Muhle, H; Polster, T; Pringsheim, M; Schubert-Bast, S; Spors, H; Strzelczyk, A; Trollmann, R, 2021) |
"Median time-to-nth seizure was longer after fenfluramine than after placebo (Study 1: fenfluramine ." | 7.11 | Fenfluramine significantly reduces day-to-day seizure burden by increasing number of seizure-free days and time between seizures in patients with Dravet syndrome: A time-to-event analysis. ( Auvin, S; Dai, D; Devinsky, O; Galer, BS; Gammaitoni, AR; Gil-Nagel, A; Perry, MS; Specchio, N; Strzelczyk, A; Sullivan, J, 2022) |
"Fenfluramine (FFA), an antiseizure medication (ASM) indicated for treating convulsive seizures in Dravet syndrome, was assessed in six patients (five female; 83%) with CDD whose seizures had failed 5-12 ASMs or therapies." | 5.62 | Effect of fenfluramine on convulsive seizures in CDKL5 deficiency disorder. ( Conway, E; Devinsky, O; King, L; Price, D; Schwartz, D, 2021) |
" Fenfluramine is a new ASM for the treatment of seizures associated with DS as add-on therapy to other ASMs for patients aged 2 years and older." | 5.22 | The contribution of fenfluramine to the treatment of Dravet syndrome in Spain through Multi-Criteria Decision Analysis. ( Abad-Sazatornil, MR; Aibar, JÁ; Aras, LM; Cardenal-Muñoz, E; Falip, M; Gil, A; Gil-Nagel, A; Poveda, JL; Sánchez, R; Sánchez-Carpintero, R; Sancho-López, A; Torrejón, M; Trillo-Mata, JL, 2022) |
"Although cannabidiol and fenfluramine have been recently approved by the US Food and Drug Administration (FDA) for seizures in children with Dravet syndrome (DS), the comparative efficacy and safety of these and stiripentol as an add-on therapy for DS has not been evaluated in head-to-head trials." | 5.12 | Comparative short-term efficacy and safety of add-on anti-seizure medications in Dravet syndrome: An indirect treatment comparison. ( Asrar, MM; Bansal, D; Devi, N; Madaan, P; Sahu, JK, 2021) |
" Before the approval of fenfluramine (FFA) for the treatment of seizures in DS, patients in Germany could receive treatment under a compassionate use program (CUP)." | 4.02 | Efficacy, tolerability, and retention of fenfluramine for the treatment of seizures in patients with Dravet syndrome: Compassionate use program in Germany. ( Alber, M; Klotz, KA; Kluger, G; Kurlemann, G; Mayer, T; Muhle, H; Polster, T; Pringsheim, M; Schubert-Bast, S; Spors, H; Strzelczyk, A; Trollmann, R, 2021) |
"Median time-to-nth seizure was longer after fenfluramine than after placebo (Study 1: fenfluramine ." | 3.11 | Fenfluramine significantly reduces day-to-day seizure burden by increasing number of seizure-free days and time between seizures in patients with Dravet syndrome: A time-to-event analysis. ( Auvin, S; Dai, D; Devinsky, O; Galer, BS; Gammaitoni, AR; Gil-Nagel, A; Perry, MS; Specchio, N; Strzelczyk, A; Sullivan, J, 2022) |
" The mainstay of treatment is with multiple anti-seizure medications (ASMs); however, the ASMs themselves can be associated with psychobehavioural adverse events, and effects (negative or positive) on cognition and sleep." | 2.82 | Psychobehavioural and Cognitive Adverse Events of Anti-Seizure Medications for the Treatment of Developmental and Epileptic Encephalopathies. ( Schubert-Bast, S; Strzelczyk, A, 2022) |
"Experts in Dravet syndrome flagged the main barriers in caring for adults with DS and created a 2-page transition summary guide based on their expertise and a literature review." | 2.72 | Dravet syndrome: A quick transition guide for the adult neurologist. ( Andrade, DM; Berg, AT; Hood, V; Knupp, KG; Koh, S; Laux, L; Meskis, MA; Miller, I; Perry, MS; Scheffer, IE; Sullivan, J; Villas, N; Wirrell, E, 2021) |
"Fenfluramine appears to be a safe and efficacious antiseizure medication in patients with Dravet syndrome." | 2.72 | Efficacy and tolerability of fenfluramine in patients with Dravet syndrome: A systematic review and meta-analysis. ( Dawman, L; Joshi, K; Kasinathan, A; Panda, P; Panda, PK; Sharawat, IK, 2021) |
"The potential antiseizure effects of fenfluramine were first identified in patients with photosensitive epilepsy in the 1980s but it was not rigorously explored as a treatment option until 30 years later." | 2.72 | Fenfluramine for treatment-resistant epilepsy in Dravet syndrome and other genetically mediated epilepsies. ( Simmons, R; Sullivan, J, 2021) |
" This practical guide provides an overview of these main ASMs including their indications/contraindications, mechanism of action, efficacy, safety and tolerability profile, dosage requirements, and laboratory and clinical parameters to be evaluated." | 1.72 | A Practical Guide to the Treatment of Dravet Syndrome with Anti-Seizure Medication. ( Schubert-Bast, S; Strzelczyk, A, 2022) |
"The most commonly reported seizure-related benefits (> 50 % of participants) of FFA treatment included a reduction in seizure activity, fewer seizure triggers, and shorter post-ictal recovery." | 1.72 | Fenfluramine treatment for dravet syndrome: Real-world benefits on quality of life from the caregiver perspective. ( Amtmann, D; Galer, BS; Gammaitoni, AR; Jensen, MP; Salem, R; Wilkie, D, 2022) |
"Fenfluramine has a potent anticonvulsive effect in DS." | 1.72 | A critical evaluation of fenfluramine hydrochloride for the treatment of Dravet syndrome. ( Ceulemans, B; Schoonjans, AS, 2022) |
"Fenfluramine (FFA), an antiseizure medication (ASM) indicated for treating convulsive seizures in Dravet syndrome, was assessed in six patients (five female; 83%) with CDD whose seizures had failed 5-12 ASMs or therapies." | 1.62 | Effect of fenfluramine on convulsive seizures in CDKL5 deficiency disorder. ( Conway, E; Devinsky, O; King, L; Price, D; Schwartz, D, 2021) |
"Fenfluramine (Fintepla) has demonstrated profound reduction in convulsive seizure frequency in DS and was recently approved for use in DS in the US and EU." | 1.62 | Treatment with fenfluramine in patients with Dravet syndrome has no long-term effects on weight and growth. ( Agarwal, A; Ceulemans, B; Cortes, RM; Davis, R; Devinsky, O; Farfel, G; Galer, BS; Gammaiton, AR; Gil-Nagel, A; Knupp, KG; Lock, M; Nabbout, R; Polster, T; Scott Perry, M; Sullivan, J; Wirrell, E, 2021) |
Timeframe | Studies, this research(%) | All Research% |
---|---|---|
pre-1990 | 0 (0.00) | 18.7374 |
1990's | 0 (0.00) | 18.2507 |
2000's | 0 (0.00) | 29.6817 |
2010's | 1 (5.88) | 24.3611 |
2020's | 16 (94.12) | 2.80 |
Authors | Studies |
---|---|
Andrade, DM | 1 |
Berg, AT | 1 |
Hood, V | 1 |
Knupp, KG | 2 |
Koh, S | 1 |
Laux, L | 1 |
Meskis, MA | 1 |
Miller, I | 1 |
Perry, MS | 2 |
Scheffer, IE | 1 |
Sullivan, J | 5 |
Villas, N | 1 |
Wirrell, E | 2 |
Trowbridge, S | 1 |
Poduri, A | 1 |
Olson, H | 1 |
Specchio, N | 1 |
Devinsky, O | 3 |
Auvin, S | 2 |
Strzelczyk, A | 4 |
Gil-Nagel, A | 3 |
Dai, D | 1 |
Galer, BS | 3 |
Gammaitoni, AR | 2 |
Schubert-Bast, S | 3 |
Falip, M | 1 |
Sánchez-Carpintero, R | 1 |
Abad-Sazatornil, MR | 1 |
Poveda, JL | 1 |
Aibar, JÁ | 1 |
Cardenal-Muñoz, E | 1 |
Aras, LM | 1 |
Sánchez, R | 1 |
Sancho-López, A | 1 |
Trillo-Mata, JL | 1 |
Torrejón, M | 1 |
Gil, A | 1 |
Jensen, MP | 1 |
Salem, R | 1 |
Wilkie, D | 1 |
Amtmann, D | 1 |
Berkovic, SF | 1 |
Schoonjans, AS | 1 |
Ceulemans, B | 2 |
Sharawat, IK | 1 |
Panda, PK | 1 |
Kasinathan, A | 1 |
Panda, P | 1 |
Dawman, L | 1 |
Joshi, K | 1 |
King, L | 1 |
Schwartz, D | 1 |
Conway, E | 1 |
Price, D | 1 |
Helen Cross, J | 1 |
Simmons, R | 1 |
Devi, N | 1 |
Madaan, P | 1 |
Asrar, MM | 1 |
Sahu, JK | 1 |
Bansal, D | 1 |
Nabbout, R | 1 |
Scott Perry, M | 1 |
Polster, T | 2 |
Davis, R | 1 |
Lock, M | 1 |
Cortes, RM | 1 |
Gammaiton, AR | 1 |
Farfel, G | 1 |
Agarwal, A | 1 |
Pringsheim, M | 1 |
Mayer, T | 1 |
Klotz, KA | 1 |
Muhle, H | 1 |
Alber, M | 1 |
Trollmann, R | 1 |
Spors, H | 1 |
Kluger, G | 1 |
Kurlemann, G | 1 |
Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
---|---|---|---|---|---|---|---|
A Multicenter, Randomized, Double-blind, Parallel Group, Placebo-controlled Trial of Two Fixed Doses of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children and Young Adults With Dravet Syndrome[NCT02682927] | Phase 3 | 262 participants (Actual) | Interventional | 2016-01-15 | Completed | ||
Fenfluramine in CKDL5 Deficiency Disorder (CDD)[NCT03861871] | Phase 2 | 7 participants (Actual) | Interventional | 2019-10-29 | Completed | ||
[information is prepared from clinicaltrials.gov, extracted Sep-2024] |
AUC0-24 is the area under the concentration time curve from time zero to 24 hours. (NCT02682927)
Timeframe: At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose
Intervention | nanogram*hour per milliliter (ng*h/mL) (Geometric Mean) |
---|---|
Study 1: ZX008 0.2 mg/kg/Day | 356 |
Study 1: ZX008 0.8 mg/kg/Day | 1390 |
Study 3: ZX008 0.2 mg/kg/Day | 348 |
Study 3: ZX008 0.8 mg/kg/Day | 1290 |
Total seizure frequency were defined as the combination of convulsive and non-convulsive seizures. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). Non-convulsive seizures included focal without clear observable motor signs, absence or atypical absence, myoclonic and atonic. The seizure frequency was based on electronic diary data obtained for each participant. The number of all seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day convulsive or non-convulsive seizure frequency. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | seizure frequency per 28 days (Median) |
---|---|
Study 1: Placebo | -4.45 |
Study 1: ZX008 0.2 mg/kg/Day | -7.40 |
Study 1: ZX008 0.8 mg/kg/Day | -22.95 |
Study 3: Placebo | -1.09 |
Study 3: ZX008 0.2 mg/kg/Day | -6.54 |
Study 3: ZX008 0.8 mg/kg/Day | -11.39 |
Non-convulsive seizures included focal without clear observable motor signs, absence or atypical absence, myoclonic and atonic. The number of non-convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day non-convulsive seizure frequency. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | seizure frequency per 28 days (Median) |
---|---|
Study 1: Placebo | -9.38 |
Study 1: ZX008 0.2 mg/kg/Day | -4.85 |
Study 1: ZX008 0.8 mg/kg/Day | -20.06 |
Study 3: Placebo | -0.68 |
Study 3: ZX008 0.2 mg/kg/Day | -0.67 |
Study 3: ZX008 0.8 mg/kg/Day | -4.35 |
Monthly (28 day) convulsive seizure frequency (CSF) was based on electronic diary data obtained for each participant. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). The number of convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day CSF. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | seizure frequency per 28 days (Mean) |
---|---|
Study 1: Placebo | -6.71 |
Study 1: ZX008 0.8 mg/kg/Day | -13.11 |
Study 3: Placebo | 1.54 |
Study 3: ZX008 0.8 mg/kg/Day | -3.54 |
Monthly (28 day) convulsive seizure frequency (CSF) was based on electronic diary data obtained for each participant. Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). The number of convulsive seizures reported during the entire time interval was divided by the number of nonmissing diary days and the result was then multiplied by 28 to get a 28-day CSF. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | seizure frequency per 28 days (Mean) |
---|---|
Study 1: Placebo | -6.71 |
Study 1: ZX008 0.2 mg/kg/Day | -18.81 |
Study 3: Placebo | 1.54 |
Study 3: ZX008 0.2 mg/kg/Day | -5.89 |
The Pediatric Quality of Life Inventory (PedsQL) is a pediatric modular measure of health related quality of life (QoL) completed by the parent/caregiver on behalf of the participant. It consisted of 23 items across 4 core scales that measure physical (8 items), emotional, social, and school functioning (5 items each). Each of the responses to the 23 items is initially scored on a 5-point Likert scale from 0 (Never) to 4 (Almost always). Scores are linearly transformed to a scale of 0 to 100, where 0=100, 1=75, 2=50, 3=25 and 4=0, and higher scores correspond to better health-related QoL. The Overall Quality of Life is the average of all the items over the number of items answered on all the Scales. (NCT02682927)
Timeframe: From Baseline to Day 99
Intervention | score on a scale (Mean) |
---|---|
Study 1: Placebo | -1.6 |
Study 1: ZX008 0.2 mg/kg/Day | 6.8 |
Study 1: ZX008 0.8 mg/kg/Day | 5.9 |
Study 3: Placebo | 1.9 |
Study 3: ZX008 0.2 mg/kg/Day | 4.2 |
Study 3: ZX008 0.8 mg/kg/Day | 2.1 |
QOLCE is a low-burden parent/caregiver completed assessment that evaluates how epilepsy affects day-to day functioning of the participant in various life areas, including physical activities, well being, cognition, social activities, behavior, and general health. QOLCE scores items on 16 subscales with possible 5-point response for each, where scores of 5 was best possible response and 1 was worst possible response. Item scores were then transformed to a 0-100 scale as follows: 1-0, 2-25, 3-50, 4-75, 5-100. A score for each participant for each subscale was calculated by averaging that participant's responses to each item in the subscale. Subscale scores per participant were averaged to obtain an overall QoL score for each participant. Higher the subscale and overall QoL scores, better the response. (NCT02682927)
Timeframe: From Baseline to Day 99
Intervention | score on a scale (Mean) |
---|---|
Study 1: Placebo | 1.5 |
Study 1: ZX008 0.2 mg/kg/Day | 0.8 |
Study 1: ZX008 0.8 mg/kg/Day | 5.8 |
Study 3: Placebo | 1.2 |
Study 3: ZX008 0.2 mg/kg/Day | 6.1 |
Study 3: ZX008 0.8 mg/kg/Day | 5.5 |
The PedsQL Family Impact measured the impact of pediatric chronic health conditions on parents and the family by measuring parent self-reported physical, emotional, social, and cognitive functioning, communication, worry, and family daily activities and relationships. There are a total of 36 items in the PedsQL: 6 items for Physical Functioning, 5 items each for Emotional Functioning, Cognitive Functioning and Worry, 4 for Social Functioning, 3 for Communication, 3 questions for Daily Activities, and 5 for Family Relationships. Each of the responses are initially scored on a 5-point Likert scale from 0 (Never) to 4 (Almost always) and then linearly transformed to a scale of 0 to 100, where 0=100, 1=75, 2=50, 3=25 and 4=0, and higher scores mean better health-related QoL. (NCT02682927)
Timeframe: From Baseline to Day 99
Intervention | score on a scale (Mean) |
---|---|
Study 1: Placebo | -4.4 |
Study 1: ZX008 0.2 mg/kg/Day | 3.9 |
Study 1: ZX008 0.8 mg/kg/Day | 5.4 |
Study 3: Placebo | 1.3 |
Study 3: ZX008 0.2 mg/kg/Day | 0.7 |
Study 3: ZX008 0.8 mg/kg/Day | 6.3 |
t1/2 beta is the elimination half-life. (NCT02682927)
Timeframe: At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose
Intervention | hours (h) (Geometric Mean) |
---|---|
Study 1: ZX008 0.2 mg/kg/Day | 18.4 |
Study 1: ZX008 0.8 mg/kg/Day | 21.1 |
Study 3: ZX008 0.2 mg/kg/Day | 18.1 |
Study 3: ZX008 0.8 mg/kg/Day | 18.6 |
The longest interval between convulsive seizures was calculated over the entire Titration and Maintenance Period and was derived as the maximum of the number of days between consecutive convulsive seizures. (NCT02682927)
Timeframe: During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days)
Intervention | days (Median) |
---|---|
Study 1: Placebo | 9.50 |
Study 1: ZX008 0.2 mg/kg/Day | 15.00 |
Study 1: ZX008 0.8 mg/kg/Day | 25.00 |
Study 3: Placebo | 10 |
Study 3: ZX008 0.2 mg/kg/Day | 18.5 |
Study 3: ZX008 0.8 mg/kg/Day | 30 |
Cmax is the maximum observed concentration determined directly from the concentration-time profile. (NCT02682927)
Timeframe: At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose
Intervention | nanograms per milliliter (ng/mL) (Geometric Mean) |
---|---|
Study 1: ZX008 0.2 mg/kg/Day | 17.7 |
Study 1: ZX008 0.8 mg/kg/Day | 67.9 |
Study 3: ZX008 0.2 mg/kg/Day | 17.4 |
Study 3: ZX008 0.8 mg/kg/Day | 64.5 |
A convulsive seizure free day was defined as a day for which diary data are available and no convulsive seizures were reported. Convulsive seizure free days were taken from the electronic diary data. (NCT02682927)
Timeframe: During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days)
Intervention | seizure free days (Median) |
---|---|
Study 1: Placebo | 15.14 |
Study 1: ZX008 0.2 mg/kg/Day | 20.86 |
Study 1: ZX008 0.8 mg/kg/Day | 24.43 |
Study 3: Placebo | 20.20 |
Study 3: ZX008 0.2 mg/kg/Day | 23.36 |
Study 3: ZX008 0.8 mg/kg/Day | 25.33 |
Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 50% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | percentage of participants (Number) |
---|---|
Study 1: Placebo | 12.5 |
Study 1: ZX008 0.2 mg/kg/Day | 38.5 |
Study 1: ZX008 0.8 mg/kg/Day | 67.5 |
Study 3: Placebo | 6.3 |
Study 3: ZX008 0.2 mg/kg/Day | 45.7 |
Study 3: ZX008 0.8 mg/kg/Day | 72.9 |
Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 75% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | percentage of participants (Number) |
---|---|
Study 1: Placebo | 2.5 |
Study 1: ZX008 0.2 mg/kg/Day | 23.1 |
Study 1: ZX008 0.8 mg/kg/Day | 50.0 |
Study 3: Placebo | 4.2 |
Study 3: ZX008 0.2 mg/kg/Day | 28.3 |
Study 3: ZX008 0.8 mg/kg/Day | 47.9 |
Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 100% reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | percentage of participants (Number) |
---|---|
Study 1: Placebo | 0 |
Study 1: ZX008 0.2 mg/kg/Day | 7.7 |
Study 1: ZX008 0.8 mg/kg/Day | 7.5 |
Study 3: Placebo | 0 |
Study 3: ZX008 0.2 mg/kg/Day | 0 |
Study 3: ZX008 0.8 mg/kg/Day | 12.5 |
Convulsive seizures included hemiclonic, focal with clear observable motor signs, generalized tonic clonic, secondarily generalized tonic clonic, tonic, clonic, and drop seizures (tonic/atonic). A responder was a participant who experienced a 25% or greater reduction in convulsive seizure frequency per 28 days during Titration and Maintenance Period. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | percentage of participants (Number) |
---|---|
Study 1: Placebo | 35.0 |
Study 1: ZX008 0.2 mg/kg/Day | 66.7 |
Study 1: ZX008 0.8 mg/kg/Day | 90.0 |
Study 3: Placebo | 27.1 |
Study 3: ZX008 0.2 mg/kg/Day | 71.7 |
Study 3: ZX008 0.8 mg/kg/Day | 83.3 |
Participants who utilized medical center care to treat a seizure during the study were reported. (NCT02682927)
Timeframe: During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days)
Intervention | percentage of participants (Number) |
---|---|
Study 1: Placebo | 22.5 |
Study 1: ZX008 0.2 mg/kg/Day | 17.9 |
Study 1: ZX008 0.8 mg/kg/Day | 15.0 |
Study 3: Placebo | 12.5 |
Study 3: ZX008 0.2 mg/kg/Day | 19.6 |
Study 3: ZX008 0.8 mg/kg/Day | 14.6 |
Rescue medication was administered according to each participant's usual or prescribed regimen consisting of 1 or more medications. The usage of rescue medication (number of days and number of medications used per seizure episode) was based on electronic diary data obtained for each participant. The number of days rescue medication was taken (normalized to 28 days) was calculated for each participant. Multiple medications taken on the same day were counted once for that day. (NCT02682927)
Timeframe: From Baseline up to 14 weeks [Titration Period (2 weeks) plus Maintenance Period (12 weeks)]
Intervention | percentage of participants (Number) |
---|---|
Study 1: Placebo | 77.5 |
Study 1: ZX008 0.2 mg/kg/Day | 59.0 |
Study 1: ZX008 0.8 mg/kg/Day | 45.0 |
Study 3: Placebo | 60.4 |
Study 3: ZX008 0.2 mg/kg/Day | 65.2 |
Study 3: ZX008 0.8 mg/kg/Day | 47.9 |
The participants who either had SE episode recorded as an adverse event (AE) during treatment or a seizure greater than 10 minutes were reported for each treatment group. Additionally, a single participant who may had more than one episode of SE, and an episode of SE recorded as both an AE and as a seizure longer than 10 minutes was counted as a single event. (NCT02682927)
Timeframe: During 14 weeks Titration (2 weeks) and Maintenance Period (12 weeks) (average of 99 days)
Intervention | percentage of participants (Number) |
---|---|
Study 1: Placebo | 27.5 |
Study 1: ZX008 0.2 mg/kg/Day | 28.2 |
Study 1: ZX008 0.8 mg/kg/Day | 35.0 |
Study 3: Placebo | 16.7 |
Study 3: ZX008 0.2 mg/kg/Day | 19.6 |
Study 3: ZX008 0.8 mg/kg/Day | 25.0 |
Tmax is the time to maximum concentration at steady state. (NCT02682927)
Timeframe: At Visit 8 (Day 43): pre-dose, 1, 2, and 4-6 hours postdose
Intervention | hours (h) (Median) |
---|---|
Study 1: ZX008 0.2 mg/kg/Day | 2.90 |
Study 1: ZX008 0.8 mg/kg/Day | 3.00 |
Study 3: ZX008 0.2 mg/kg/Day | 2.90 |
Study 3: ZX008 0.8 mg/kg/Day | 2.90 |
The HADS is a tool that was validated to assess presence of anxiety or depression in an outpatient non-psychiatric population. The HADS a 14-item scale that generates ordinal data for 2 dimensions: 1) Anxiety (7 items), and 2) Depression (7 items). Each item has 4 possible answers rated 0 to 3, of which 0 = No distress and 3 = worst distress. All answers to the items for a dimension with their respective rating are added resulting in a range for each dimension from 0-21, out of which of 0-7 = normal; 8-10=borderline abnormal; 11-21=abnormal. Scores for the entire scale (emotional distress) range from 0 to 42, with higher scores indicating more distress. (NCT02682927)
Timeframe: From Baseline to Day 99
Intervention | score on a scale (Mean) | ||
---|---|---|---|
Anxiety | Depression | Total emotional distress | |
Study 1: Placebo | -0.4 | 0.8 | 0.4 |
Study 1: ZX008 0.2 mg/kg/Day | -0.8 | 0.2 | -0.6 |
Study 1: ZX008 0.8 mg/kg/Day | -0.8 | 0.1 | -0.7 |
Study 3: Placebo | -0.6 | -0.7 | -1.2 |
Study 3: ZX008 0.2 mg/kg/Day | 0.2 | 2.0 | 2.2 |
Study 3: ZX008 0.8 mg/kg/Day | -0.7 | -0.8 | -1.5 |
Duration of single convulsive seizures during the Baseline and the duration over the Titration and Maintenance Period were reported by treatment group using categories as <2 minutes, 2 to 10 minutes and > 10 minutes as collected in the seizure diary. (NCT02682927)
Timeframe: At Baseline and 14 weeks of Titration (2 weeks) and Maintenance Period (12 weeks)
Intervention | percentage of seizures (Number) | |||||
---|---|---|---|---|---|---|
<2 min (Baseline) | 2-10 min (Baseline) | >10 min (Baseline) | <2 min (Titration + Maintenance Period) | 2-10 min (Titration + Maintenance Period) | >10 min (Titration + Maintenance Period) | |
Study 1: Placebo | 69.28 | 26.86 | 3.86 | 71.31 | 26.31 | 2.38 |
Study 1: ZX008 0.2 mg/kg/Day | 64.13 | 34.95 | 0.93 | 71.59 | 25.61 | 2.79 |
Study 1: ZX008 0.8 mg/kg/Day | 71.61 | 24.22 | 4.17 | 72.27 | 22.91 | 4.82 |
Study 3: Placebo | 64.21 | 34.83 | 0.96 | 65.84 | 33.74 | 0.43 |
Study 3: ZX008 0.2 mg/kg/Day | 63.90 | 33.66 | 2.45 | 63.45 | 31.34 | 5.22 |
Study 3: ZX008 0.8 mg/kg/Day | 74.11 | 22.78 | 3.10 | 84.67 | 13.71 | 1.62 |
CGI-I scale measures improvement in the participant's clinical status from Baseline. The severity of a participant's condition was rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse), as follows: 1-very much improved,2-much improved, 3-minimally improved, 4- no change, 5-minimally worse, 6-much worse and 7-very much worse. The Principal Investigator rated their global impression of the participant's condition during the study. (NCT02682927)
Timeframe: At Visit 6 (Day 15), 8 (Day 43), 10 (Day 71) and 12 (Day 99)
Intervention | percentage of participants (Number) | |||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 = Very much improved (Visit 6) | 2 = Much improved (Visit 6) | 3 = Minimally improved (Visit 6) | 4 = No change (Visit 6) | 5 = Minimally worse (Visit 6) | 6 = Much worse (Visit 6) | 7 = Very much worse (Visit 6) | 1 = Very much improved (Visit 8) | 2 = Much improved (Visit 8) | 3 = Minimally improved (Visit 8) | 4 = No change (Visit 8) | 5 = Minimally worse (Visit 8) | 6 = Much worse (Visit 8) | 7 = Very much worse (Visit 8) | 1 = Very much improved (Visit 10) | 2 = Much improved (Visit 10) | 3 = Minimally improved (Visit 10) | 4 = No change (Visit 10) | 5 = Minimally worse (Visit 10) | 6 = Much worse (Visit 10) | 7 = Very much worse (Visit 10) | 1 = Very much improved (Visit 12) | 2 = Much improved (Visit 12) | 3 = Minimally improved (Visit 12) | 4 = No change (Visit 12) | 5 = Minimally worse (Visit 12) | 6 = Much worse (Visit 12) | 7 = Very much worse (Visit 12) | |
Study 1: Placebo | 5.0 | 12.5 | 20.0 | 40.0 | 5.0 | 0 | 0 | 0 | 12.5 | 30.0 | 30.0 | 5.0 | 2.5 | 0 | 2.5 | 7.5 | 30.0 | 35.0 | 10.0 | 0 | 0 | 2.5 | 7.5 | 30.0 | 47.5 | 2.5 | 2.5 | 0 |
Study 1: ZX008 0.2 mg/kg/Day | 23.1 | 12.8 | 20.5 | 25.6 | 7.7 | 0 | 0 | 5.1 | 30.8 | 20.5 | 17.9 | 5.1 | 5.1 | 0 | 17.9 | 17.9 | 25.6 | 28.2 | 7.7 | 2.6 | 0 | 12.8 | 28.2 | 17.9 | 28.2 | 10.3 | 2.6 | 0 |
Study 1: ZX008 0.8 mg/kg/Day | 17.5 | 25.0 | 20.0 | 17.5 | 5.0 | 2.5 | 0 | 17.5 | 37.5 | 10.0 | 10.0 | 0 | 2.5 | 2.5 | 20.0 | 47.5 | 5.0 | 7.5 | 0 | 0 | 0 | 27.5 | 35.0 | 15.0 | 12.5 | 0 | 0 | 2.5 |
Study 3: Placebo | 4.2 | 2.1 | 27.1 | 54.2 | 4.2 | 0 | 0 | 4.2 | 6.3 | 16.7 | 50.0 | 4.2 | 2.1 | 0 | 4.2 | 10.4 | 12.5 | 60.4 | 0 | 0 | 0 | 4.2 | 4.2 | 16.7 | 58.3 | 6.3 | 0 | 0 |
Study 3: ZX008 0.2 mg/kg/Day | 21.7 | 21.7 | 17.4 | 26.1 | 2.2 | 0 | 0 | 17.4 | 10.9 | 26.1 | 34.8 | 0 | 0 | 0 | 15.2 | 19.6 | 26.1 | 28.3 | 0 | 0 | 0 | 8.7 | 28.3 | 21.7 | 28.3 | 10.9 | 0 | 0 |
Study 3: ZX008 0.8 mg/kg/Day | 16.7 | 27.1 | 27.1 | 12.5 | 2.1 | 4.2 | 0 | 29.2 | 31.3 | 14.6 | 8.3 | 4.2 | 0 | 0 | 35.4 | 18.8 | 16.7 | 4.2 | 0 | 2.1 | 0 | 33.3 | 31.3 | 10.4 | 16.7 | 6.3 | 0 | 0 |
CGI-I scale measures improvement in the participant's clinical status from Baseline. The severity of a participant's condition was rated on a 7-point scale ranging from 1 (very much improved) to 7 (very much worse), as follows: 1-very much improved,2-much improved, 3-minimally improved, 4- no change, 5-minimally worse, 6-much worse and 7-very much worse. The Parent/Caregiver rated their global impression of the participant's condition during the study. (NCT02682927)
Timeframe: At Visit 6 (Day 15), 8 (Day 43), 10 (Day 71) and 12 (Day 99)
Intervention | percentage of participants (Number) | |||||||||||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 = Very much improved (Visit 6) | 2 = Much improved (Visit 6) | 3 = Minimally improved (Visit 6) | 4 = No change (Visit 6) | 5 = Minimally worse (Visit 6) | 6 = Much worse (Visit 6) | 7 = Very much worse (Visit 6) | 1 = Very much improved (Visit 8) | 2 = Much improved (Visit 8) | 3 = Minimally improved (Visit 8) | 4 = No change (Visit 8) | 5 = Minimally worse (Visit 8) | 6 = Much worse (Visit 8) | 7 = Very much worse (Visit 8) | 1 = Very much improved (Visit 10) | 2 = Much improved (Visit 10) | 3 = Minimally improved (Visit 10) | 4 = No change (Visit 10) | 5 = Minimally worse (Visit 10) | 6 = Much worse (Visit 10) | 7 = Very much worse (Visit 10) | 1 = Very much improved (Visit 12) | 2 = Much improved (Visit 12) | 3 = Minimally improved (Visit 12) | 4 = No change (Visit 12) | 5 = Minimally worse (Visit 12) | 6 = Much worse (Visit 12) | 7 = Very much worse (Visit 12) | |
Study 1: Placebo | 2.5 | 22.5 | 12.5 | 45.0 | 2.5 | 5.0 | 0 | 0 | 15.0 | 25.0 | 20.0 | 15.0 | 2.5 | 0 | 2.5 | 12.5 | 22.5 | 32.5 | 12.5 | 2.5 | 2.5 | 2.5 | 7.5 | 20.0 | 35.0 | 17.5 | 7.5 | 0 |
Study 1: ZX008 0.2 mg/kg/Day | 17.9 | 20.5 | 28.2 | 12.8 | 7.7 | 2.6 | 0 | 15.4 | 25.6 | 25.6 | 12.8 | 10.3 | 5.1 | 0 | 20.5 | 17.9 | 20.5 | 25.6 | 7.7 | 7.7 | 0 | 20.5 | 20.5 | 15.4 | 20.5 | 15.4 | 7.7 | 0 |
Study 1: ZX008 0.8 mg/kg/Day | 15.0 | 27.5 | 22.5 | 20.0 | 2.5 | 7.5 | 2.5 | 20.0 | 37.5 | 15.0 | 5.0 | 2.5 | 5.0 | 2.5 | 35.0 | 30.0 | 7.5 | 10.0 | 0 | 0 | 2.5 | 27.5 | 27.5 | 10.0 | 15.0 | 5.0 | 5.0 | 2.5 |
Study 3: Placebo | 6.3 | 2.1 | 25.0 | 45.8 | 10.4 | 2.1 | 0 | 4.2 | 8.3 | 20.8 | 47.9 | 6.3 | 4.2 | 4.2 | 2.1 | 6.3 | 25.0 | 45.8 | 6.3 | 0 | 0 | 2.1 | 6.3 | 18.8 | 50.0 | 10.4 | 2.1 | 0 |
Study 3: ZX008 0.2 mg/kg/Day | 13.0 | 23.9 | 26.1 | 19.6 | 2.2 | 2.2 | 0 | 6.5 | 30.4 | 28.3 | 17.4 | 6.5 | 2.2 | 0 | 8.7 | 28.3 | 26.1 | 26.1 | 0 | 4.3 | 0 | 6.5 | 28.3 | 30.4 | 13.0 | 8.7 | 4.3 | 2.2 |
Study 3: ZX008 0.8 mg/kg/Day | 16.7 | 31.3 | 31.3 | 2.1 | 6.3 | 2.1 | 2.1 | 39.6 | 29.2 | 14.6 | 6.3 | 0 | 2.1 | 0 | 41.7 | 22.9 | 8.3 | 6.3 | 4.2 | 0 | 0 | 33.3 | 29.2 | 20.8 | 4.2 | 4.2 | 2.1 | 2.1 |
"The EuroQOL-5 Dimensions-5 Levels scale produced by European QOL Group (EQ-5D-5L) health questionnaire is a health-related QOL instrument with 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The 5 dimensions of EQ-5D-5L health questionnaire were assessed on a Likert scale with 5 possible levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. The categories slight problems, moderate problems, severe problems and extreme problems are collapsed into one response category problems. The QOL of the parent/caregiver was assessed and percentage of participants was reported for each item." (NCT02682927)
Timeframe: At Baseline and Day 99
Intervention | percentage of participants (Number) | |||||||||||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Mobility- No problems (Baseline) | Mobility- Problems (Baseline) | Mobility- No problems (Day 99) | Mobility- Problems (Day 99) | Self-care - No problems (Baseline) | Self-care - Problems (Baseline) | Self-care - No problems (Day 99) | Self-care - Problems (Day 99) | Usual activities- No problems (Baseline) | Usual activities- Problems (Baseline) | Usual activities- No problems (Day 99) | Usual activities- Problems (Day 99) | Pain/discomfort- No problems (Baseline) | Pain/discomfort- Problems (Baseline) | Pain/discomfort- No problems (Day 99) | Pain/discomfort- Problems (Day 99) | Anxiety/depression- No problems (Baseline) | Anxiety/depression- Problems (Baseline) | Anxiety/depression- No problems (Day 99) | Anxiety/depression- Problems (Day 99) | |
Study 1: Placebo | 33.33 | 66.67 | 40.00 | 60.00 | 25.64 | 74.36 | 28.57 | 71.43 | 23.08 | 76.92 | 25.71 | 74.29 | 48.72 | 51.28 | 48.57 | 51.43 | 74.36 | 25.64 | 65.71 | 34.29 |
Study 1: ZX008 0.2 mg/kg/Day | 52.94 | 47.06 | 45.95 | 54.05 | 41.18 | 58.82 | 43.24 | 56.76 | 41.18 | 58.82 | 32.43 | 67.57 | 41.18 | 58.82 | 51.35 | 48.65 | 61.76 | 38.24 | 67.57 | 32.43 |
Study 1: ZX008 0.8 mg/kg/Day | 46.15 | 53.85 | 51.35 | 48.65 | 38.46 | 61.54 | 48.65 | 51.35 | 35.90 | 64.10 | 48.65 | 51.35 | 46.15 | 53.85 | 64.86 | 35.14 | 56.41 | 43.59 | 67.57 | 32.43 |
Study 3: Placebo | 40.00 | 60.00 | 52.38 | 47.62 | 22.50 | 77.50 | 30.95 | 69.05 | 25.00 | 75.00 | 30.95 | 69.05 | 45.00 | 55.00 | 76.19 | 45.24 | 60.00 | 40.00 | 69.05 | 30.95 |
Study 3: ZX008 0.2 mg/kg/Day | 54.55 | 45.45 | 51.16 | 48.84 | 36.36 | 63.64 | 34.88 | 65.12 | 39.39 | 60.61 | 25.58 | 74.42 | 51.52 | 48.48 | 46.51 | 53.49 | 63.64 | 36.36 | 67.44 | 32.56 |
Study 3: ZX008 0.8 mg/kg/Day | 28.57 | 71.43 | 46.67 | 53.33 | 22.86 | 77.14 | 35.56 | 64.44 | 20.00 | 80.00 | 35.56 | 64.44 | 51.43 | 48.57 | 64.44 | 35.56 | 74.29 | 25.71 | 73.33 | 26.67 |
Change between baseline and Week 14 in the median number of monthly convulsive seizures. (NCT03861871)
Timeframe: Baseline, Week 14
Intervention | Number of monthly seizures (Median) |
---|---|
Fenfluramine Hydrochloride | 88.429 |
The CGIC is a 1-item, parent/caregiver-completed assessment used determine how much their child/care-recipient has improved with treatment. The instrument asks parents/caregivers to rate their child's/care-recipient's improvement as: 1) very much improved; 2) much improved; 3) minimally improved; 4) unchanged; 5) a little worse; 6) much worse; 7) very much worse; the total score correspondingly ranges from 1-7. (NCT03861871)
Timeframe: Baseline, Week 14
Intervention | score on a scale (Mean) |
---|---|
Fenfluramine Hydrochloride | -2.429 |
The IGIC is a 1-item, investigator-completed assessment used determine how much a patient has improved with treatment. The instrument asks the investigator to rate patients' improvement as: 1) very much improved; 2) much improved; 3) minimally improved; 4) unchanged; 5) a little worse; 6) much worse; 7) very much worse; the total score correspondingly ranges from 1-7. (NCT03861871)
Timeframe: Baseline, Week 14
Intervention | score on a scale (Mean) |
---|---|
Fenfluramine Hydrochloride | 1.571 |
The PedsQL Epilepsy Module is a 29-item measure with five scales: Impact, Cognitive, Sleep, Executive Function, and Mood/Behavior. The Impact scale (nine items) assesses how epilepsy interferes with daily activities, interacting with peers, independence, and increased disease burden due to treatment. The Cognitive Scale (six items) assesses memory, ability to learn new materials, school-related difficulties, and reading difficulties. The Sleep Scale (three items) assesses fatigue and sleep difficulties. The Executive Function Scale (six items) assesses organization, task initiation, impulsivity, and inattention. The Mood/Behavior Scale (five items) assesses feelings of anger, sadness, worries, and frustration tolerance. Scores range from 0-100 for each subscale, with higher scores representing better quality of life. The raw score is the sum of each subscale score and ranges from 0-500. (NCT03861871)
Timeframe: Baseline, Week 14
Intervention | score on a scale (Mean) |
---|---|
Fenfluramine Hydrochloride | -103.571 |
Parent/caregiver-completed assessment assessing how epilepsy affects day-to-day functioning of their child/care-recipient in various life areas. Each item is ranked on a 5-point Likert scale from 1 (response correlated with the lowest possible quality of life) to 5 (response correlated with the highest possible quality of life). Item scores are then transformed to a 0-100 scale as follows: 1 = 0, 2 = 25, 3 = 50, 4=75, and 5=100. The total score is the average of all item scores and ranges from 0-100. Higher scores indicate greater quality of life; an increase in scores indicates quality of life increased during the observational period. (NCT03861871)
Timeframe: Baseline, Week 14
Intervention | score on a scale (Mean) |
---|---|
Fenfluramine Hydrochloride | -0.429 |
6 reviews available for fenfluramine and Cryptogenic Infantile Spasms
Article | Year |
---|---|
Dravet syndrome: A quick transition guide for the adult neurologist.
Topics: Anticonvulsants; Child; Epilepsies, Myoclonic; Fenfluramine; Humans; Neurologists; Spasms, Infantile | 2021 |
The contribution of fenfluramine to the treatment of Dravet syndrome in Spain through Multi-Criteria Decision Analysis.
Topics: Anticonvulsants; Decision Support Techniques; Epilepsies, Myoclonic; Epileptic Syndromes; Fenflurami | 2022 |
Psychobehavioural and Cognitive Adverse Events of Anti-Seizure Medications for the Treatment of Developmental and Epileptic Encephalopathies.
Topics: Autism Spectrum Disorder; Bromides; Cannabidiol; Clobazam; Cognition; Ethosuximide; Everolimus; Felb | 2022 |
Efficacy and tolerability of fenfluramine in patients with Dravet syndrome: A systematic review and meta-analysis.
Topics: Child; Epilepsies, Myoclonic; Female; Fenfluramine; Humans; Infant; Male; Seizures; Spasms, Infantil | 2021 |
Fenfluramine for treatment-resistant epilepsy in Dravet syndrome and other genetically mediated epilepsies.
Topics: Animals; Anticonvulsants; Epilepsies, Myoclonic; Fenfluramine; Humans; Randomized Controlled Trials | 2021 |
Comparative short-term efficacy and safety of add-on anti-seizure medications in Dravet syndrome: An indirect treatment comparison.
Topics: Adolescent; Anticonvulsants; Child; Child, Preschool; Epilepsies, Myoclonic; Fenfluramine; Humans; I | 2021 |
2 trials available for fenfluramine and Cryptogenic Infantile Spasms
Article | Year |
---|---|
Fenfluramine significantly reduces day-to-day seizure burden by increasing number of seizure-free days and time between seizures in patients with Dravet syndrome: A time-to-event analysis.
Topics: Anticonvulsants; Epilepsies, Myoclonic; Epileptic Syndromes; Fenfluramine; Humans; Quality of Life; | 2022 |
A patient centered view of randomized control trial data: An example with fenfluramine for Dravet syndrome.
Topics: Anticonvulsants; Epilepsies, Myoclonic; Fenfluramine; Humans; Patient-Centered Care; Spasms, Infanti | 2021 |
9 other studies available for fenfluramine and Cryptogenic Infantile Spasms
Article | Year |
---|---|
Early diagnosis and experimental treatment with fenfluramine via the Investigational New Drug mechanism in a boy with Dravet syndrome and recurrent status epilepticus.
Topics: Anticonvulsants; Drugs, Investigational; Early Diagnosis; Epilepsies, Myoclonic; Epileptic Syndromes | 2021 |
A Practical Guide to the Treatment of Dravet Syndrome with Anti-Seizure Medication.
Topics: Adult; Anticonvulsants; Cannabidiol; Child; Clobazam; Drug Therapy, Combination; Epilepsies, Myoclon | 2022 |
Fenfluramine treatment for dravet syndrome: Real-world benefits on quality of life from the caregiver perspective.
Topics: Adolescent; Adult; Caregivers; Child; Child, Preschool; Epilepsies, Myoclonic; Epileptic Syndromes; | 2022 |
Epileptic encephalopathies of infancy: welcome advances.
Topics: Double-Blind Method; Epilepsies, Myoclonic; Fenfluramine; Humans; Infant; Seizures; Spasms, Infantil | 2019 |
A critical evaluation of fenfluramine hydrochloride for the treatment of Dravet syndrome.
Topics: Anticonvulsants; Epilepsies, Myoclonic; Epileptic Syndromes; Fenfluramine; Humans; Seizures; Spasms, | 2022 |
Effect of fenfluramine on convulsive seizures in CDKL5 deficiency disorder.
Topics: Adolescent; Adult; Anticonvulsants; Child; Child, Preschool; Epilepsies, Myoclonic; Epilepsy, Tonic- | 2021 |
Raising the bar: Fenfluramine sets new treatment standards for Dravet syndrome.
Topics: Anticonvulsants; Epilepsies, Myoclonic; Fenfluramine; Humans; Spasms, Infantile | 2021 |
Treatment with fenfluramine in patients with Dravet syndrome has no long-term effects on weight and growth.
Topics: Adult; Child; Epilepsies, Myoclonic; Fenfluramine; Humans; Obesity; Seizures; Spasms, Infantile | 2021 |
Efficacy, tolerability, and retention of fenfluramine for the treatment of seizures in patients with Dravet syndrome: Compassionate use program in Germany.
Topics: Adolescent; Adult; Anticonvulsants; Child; Child, Preschool; Compassionate Use Trials; Epilepsies, M | 2021 |