deferiprone has been researched along with Hallervorden-Spatz Disease in 5 studies
Deferiprone: A pyridone derivative and iron chelator that is used in the treatment of IRON OVERLOAD in patients with THALASSEMIA.
deferiprone : A member of the class of 4-pyridones that is pyridin-4(1H)-one substituted at positions 1 and 2 by methyl groups and at position 3 by a hydroxy group. A lipid-soluble iron-chelator used for treatment of thalassaemia.
| Excerpt | Relevance | Reference |
|---|---|---|
| "Deferiprone was well tolerated and adverse events were similar between the treatment groups, except for anaemia, which was seen in 12 (21%) of 58 patients in the deferiprone group, but was not seen in any patients in the placebo group." | 6.90 | Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study. ( Aguilar, C; Basu, A; Blamire, AM; Büchner, B; Chinnery, PF; Dušek, P; Fradette, C; Hayflick, SJ; Hogarth, P; Horvath, R; Kálmán, B; Karin, I; Klopstock, T; Kmieć, T; Küpper, C; Nardocci, N; Neuhofer, C; Neumayr, L; Spino, M; Steele, HE; Tricta, F; Vichinsky, E; Wilson, I; Yapici, Z; Zhao, F; Zibordi, F; Zorzi, G, 2019) |
| "Deferiprone was well tolerated and adverse events were similar between the treatment groups, except for anaemia, which was seen in 12 (21%) of 58 patients in the deferiprone group, but was not seen in any patients in the placebo group." | 2.90 | Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study. ( Aguilar, C; Basu, A; Blamire, AM; Büchner, B; Chinnery, PF; Dušek, P; Fradette, C; Hayflick, SJ; Hogarth, P; Horvath, R; Kálmán, B; Karin, I; Klopstock, T; Kmieć, T; Küpper, C; Nardocci, N; Neuhofer, C; Neumayr, L; Spino, M; Steele, HE; Tricta, F; Vichinsky, E; Wilson, I; Yapici, Z; Zhao, F; Zibordi, F; Zorzi, G, 2019) |
| 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 | 5 (100.00) | 24.3611 |
| 2020's | 0 (0.00) | 2.80 |
| Authors | Studies |
|---|---|
| Ayton, S | 1 |
| Bush, AI | 1 |
| Klopstock, T | 1 |
| Tricta, F | 1 |
| Neumayr, L | 1 |
| Karin, I | 1 |
| Zorzi, G | 1 |
| Fradette, C | 1 |
| Kmieć, T | 1 |
| Büchner, B | 1 |
| Steele, HE | 1 |
| Horvath, R | 1 |
| Chinnery, PF | 1 |
| Basu, A | 1 |
| Küpper, C | 1 |
| Neuhofer, C | 1 |
| Kálmán, B | 1 |
| Dušek, P | 1 |
| Yapici, Z | 1 |
| Wilson, I | 1 |
| Zhao, F | 1 |
| Zibordi, F | 1 |
| Nardocci, N | 1 |
| Aguilar, C | 1 |
| Hayflick, SJ | 1 |
| Spino, M | 1 |
| Blamire, AM | 1 |
| Hogarth, P | 1 |
| Vichinsky, E | 2 |
| Lemprière, S | 1 |
| Cossu, G | 1 |
| Abbruzzese, G | 1 |
| Matta, G | 1 |
| Murgia, D | 1 |
| Melis, M | 1 |
| Ricchi, V | 1 |
| Galanello, R | 1 |
| Barella, S | 1 |
| Origa, R | 1 |
| Balocco, M | 1 |
| Pelosin, E | 1 |
| Marchese, R | 1 |
| Ruffinengo, U | 1 |
| Forni, GL | 1 |
| Pratini, NR | 1 |
| Sweeters, N | 1 |
| Neufeld, JA | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| TIRCON International NBIA (Neurodegeneration Associated With Brain Iron Accumulation) Patient Registry and Natural History Study[NCT05522374] | 2,000 participants (Anticipated) | Observational [Patient Registry] | 2012-06-14 | Recruiting | |||
| A Randomized, Double-blind, Placebo-controlled Trial of Deferiprone in Patients With Pantothenate Kinase-associated Neurodegeneration (PKAN)[NCT01741532] | Phase 3 | 89 participants (Actual) | Interventional | 2012-12-13 | Completed | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
Neurodegeneration in patients with PKAN is associated with localized brain iron accumulation, with the highest amount of accumulation seen in the globus pallidus, one of the main areas for motor control. MRI R2* scans of this region were performed at baseline and Month 18 in a subset of patients who did not have a deep brain stimulation (DBS) device implanted, and for whom the use of anesthesia, if required, was deemed acceptable by the investigator. (NCT01741532)
Timeframe: Baseline to 18 Months
| Intervention | Hz (Least Squares Mean) |
|---|---|
| Deferiprone | -36.1 |
| Placebo | -0.50 |
The Barry-Albright Dystonia (BAD) scale rates severity of dystonia (sustained muscle contractions causing twisting and repetitive movements or abnormal postures) in 8 body regions. The individual scores are summed to provide a total score ranging from 0 to 32, with higher scores indicating greater severity. The co-primary endpoint in this study was the change from baseline to Month 18 in BAD total score. (NCT01741532)
Timeframe: Baseline to 18 Months
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Deferiprone | 2.48 |
| Placebo | 3.99 |
The Functional Independence Measure (FIM) scale is used to assess physical and cognitive disability in three areas of daily living: self-care, mobility, and cognition. Within each area, items are scored according to the level of assistance required to perform that activity of daily living. A score of 1-2 indicates that the patient is completely dependent on a helper to perform the task, a score of 3-5 indicates that the patient is moderately dependent, and a score of 6-7 indicates that no help is required. The individual scores are summed to provide a global score from 18 (worst) to 126 (best). (NCT01741532)
Timeframe: Baseline to 18 Months
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Deferiprone | 5.40 |
| Placebo | 0.69 |
"The Pittsburgh Sleep Quality Index (PSQI) is a self-rated questionnaire that assesses sleep quality and disturbances over a 1-month time interval. A total of 19 individual items are used to generate 7 component scores: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction, and a score is generated that ranges from 0 (best) to 21 (worst)." (NCT01741532)
Timeframe: Baseline to 18 Months
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Deferiprone | 0.48 |
| Placebo | 0.14 |
The WeeFIM is the pediatric version of the Functional Independence Measure scale, and is used to assess physical and cognitive disability in three areas of daily living: self-care, mobility, and cognition. Within each area, items are scored according to the level of assistance required to perform that activity of daily living. A score of 1-2 indicates that the patient is completely dependent on a helper to perform the task, a score of 3-5 indicates that the patient is moderately dependent, and a score of 6-7 indicates that no help is required. The individual scores are summed to provide a global score from 18 (worst) to 126 (best). (NCT01741532)
Timeframe: Baseline to 18 Months
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Deferiprone | 4.91 |
| Placebo | -2.40 |
The Patient Global Impression of Improvement (PGI-I) is a global index that assesses the response of a condition to a therapy by asking patients to rate their current state relative to their state at baseline. It consists of a 7-point rating scale, where 1=very much improved, 2= much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse. (NCT01741532)
Timeframe: Month 18
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Deferiprone | 4.55 |
| Placebo | 4.66 |
The Pediatric Quality of Life (PedsQL) questionnaire is used to measure functional health and well-being from the patient's point of view. Separate versions of the questionnaire are available for children, young adults aged 18-25 years, and adults older than 25 years. Patients are asked to indicate how they have felt over the past month, and the scores of the 23 questions are used to generate an overall score that ranges from 0 (worst) to 100 (best). (NCT01741532)
Timeframe: Baseline to 18 Months
| Intervention | score on a scale (Least Squares Mean) | |
|---|---|---|
| Patient self-report, total score | Parent proxy-report, total score | |
| Deferiprone | 1.21 | -4.90 |
| Placebo | 1.34 | -2.37 |
The Unified Parkinson's Disease Rating Scale (UPDRS) is the major rating scale used to assess severity of symptoms of Parkinson's disease, some of which are similar to those of PKAN. The UPDRS subscales used in this study were Part I: Mentation, Behavior and Mood, scored from 0 (best) to 16 (worst); Part II: Activities of Daily Living, scored from 0 (best) to 52 (worst); Part III: Motor Examination, scored from 0 (best) to 108 (worst); and Part VI: Schwab and England Activities of Daily Living Scale, scored from 0% (worst) to 100% (best). (NCT01741532)
Timeframe: Baseline to 18 Months
| Intervention | score on a scale (Least Squares Mean) | |||
|---|---|---|---|---|
| UPDRS Part I | UPDRS Part II | UPDRS Part III | UPDRS Part VI | |
| Deferiprone | -0.25 | 1.09 | 5.38 | -2.17 |
| Placebo | -0.07 | 2.36 | 2.06 | -7.66 |
1 trial available for deferiprone and Hallervorden-Spatz Disease
4 other studies available for deferiprone and Hallervorden-Spatz Disease
| Article | Year |
|---|---|
Decreasing iron neurotoxicity in pantothenate kinase-associated neurodegeneration.
Topics: Brain; Deferiprone; Double-Blind Method; Humans; Iron; Pantothenate Kinase-Associated Neurodegenerat | 2019 |
Drug reduces excess iron in ultra-rare neurodegenerative disease.
Topics: Deferiprone; Double-Blind Method; Humans; Iron; Neurodegenerative Diseases; Pantothenate Kinase-Asso | 2019 |
Efficacy and safety of deferiprone for the treatment of pantothenate kinase-associated neurodegeneration (PKAN) and neurodegeneration with brain iron accumulation (NBIA): results from a four years follow-up.
Topics: Adult; Deferiprone; Female; Humans; Iron Chelating Agents; Iron Metabolism Disorders; Longitudinal S | 2014 |
Treatment of classic pantothenate kinase-associated neurodegeneration with deferiprone and intrathecal baclofen.
Topics: Administration, Oral; Adolescent; Baclofen; Deferiprone; Drug Therapy, Combination; Humans; Injectio | 2013 |