acetyl-dl-leucine has been researched along with Niemann-Pick Disease, Type C in 4 studies
acetylleucine: used for treating vestibular-related imbalance and vertigo
Niemann-Pick Disease, Type C: An autosomal recessive lipid storage disorder that is characterized by accumulation of CHOLESTEROL and SPHINGOMYELINS in cells of the VISCERA and the CENTRAL NERVOUS SYSTEM. Type C (or C1) and type D are allelic disorders caused by mutation of the NPC1 gene, which encodes a protein that mediates intracellular cholesterol transport from LYSOSOMES. Clinical signs include hepatosplenomegaly and chronic neurological symptoms. Type D is a variant in people with a Nova Scotia ancestry.
Excerpt | Relevance | Reference |
---|---|---|
" No treatment-related serious adverse events occurred." | 7.11 | Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C. ( Bremova-Ertl, T; Claassen, J; Foltan, T; Gascon-Bayarri, J; Gissen, P; Hahn, A; Hassan, A; Hennig, A; Jones, SA; Kolnikova, M; Martakis, K; Raethjen, J; Ramaswami, U; Schneider, SA; Sharma, R, 2022) |
" No treatment-related serious adverse events occurred." | 3.11 | Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C. ( Bremova-Ertl, T; Claassen, J; Foltan, T; Gascon-Bayarri, J; Gissen, P; Hahn, A; Hassan, A; Hennig, A; Jones, SA; Kolnikova, M; Martakis, K; Raethjen, J; Ramaswami, U; Schneider, SA; Sharma, R, 2022) |
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 | 2 (50.00) | 24.3611 |
2020's | 2 (50.00) | 2.80 |
Authors | Studies |
---|---|
Fields, T | 1 |
M Bremova, T | 1 |
Billington, I | 1 |
Churchill, GC | 1 |
Evans, W | 1 |
Fields, C | 1 |
Galione, A | 1 |
Kay, R | 1 |
Mathieson, T | 1 |
Martakis, K | 2 |
Patterson, M | 1 |
Platt, F | 1 |
Factor, M | 1 |
Strupp, M | 3 |
Bremova-Ertl, T | 2 |
Claassen, J | 1 |
Foltan, T | 1 |
Gascon-Bayarri, J | 1 |
Gissen, P | 1 |
Hahn, A | 1 |
Hassan, A | 1 |
Hennig, A | 1 |
Jones, SA | 2 |
Kolnikova, M | 2 |
Raethjen, J | 1 |
Ramaswami, U | 1 |
Sharma, R | 1 |
Schneider, SA | 1 |
Cortina-Borja, M | 1 |
Te Vruchte, D | 1 |
Mengel, E | 2 |
Amraoui, Y | 2 |
Imrie, J | 1 |
I Dali, C | 1 |
Fineran, P | 1 |
Kirkegaard, T | 1 |
Runz, H | 1 |
Lachmann, R | 1 |
Platt, FM | 1 |
Bremova, T | 1 |
Malinová, V | 1 |
Reinke, J | 1 |
Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
---|---|---|---|---|---|---|---|
Effects of N-Acetyl-L-Leucine on Niemann Pick Type C Disease: A Multinational, Multicenter, Open-label, Rater-blinded Phase II Study.[NCT03759639] | Phase 2 | 33 participants (Actual) | Interventional | 2019-09-04 | Completed | ||
Effects of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NPC): A Phase III, Randomized, Placebo-controlled, Double-blind, Crossover Study[NCT05163288] | Phase 3 | 53 participants (Anticipated) | Interventional | 2022-06-30 | Active, not recruiting | ||
[information is prepared from clinicaltrials.gov, extracted Sep-2024] |
"The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: 'compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or 8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video?'~The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse)." (NCT03759639)
Timeframe: CI-CS comparing Baseline (Day 1) with IB1001 versus the end of 6-weeks treatment with IB1001 (Approximately Day 42) MINUS the CI-CS comparing the end of 6-weeks treatment with IB1001 (Approximately Day 42) versus the end of 6-weeks post-treatment washout
Intervention | score on a scale (Mean) |
---|---|
Parent Study | 0.86 |
The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: 'compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or 8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video?' The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). (NCT03759639)
Timeframe: CI-CS comparing baseline period and end of treatment period minus the change in CI-S between end of treatment period and end of washout period.
Intervention | score on a scale (Mean) |
---|---|
Parent Study | 0.08 |
"For posting, health-related quality of life based on the EQ-5D visual analogue scale (VAS) was presented as a secondary endpoint.~EQ-5D VAS is a 0-100 scale where patients are asked to indicate their overall health, with a score of 0 indicating worst health and a score of 100 indicating best health." (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | score on a scale (Mean) | |
---|---|---|
Treatment with IB1001 | Post-Treatment Washout | |
Parent Study: | 72.7 | 68.5 |
The Clinical Global Impression of Change assessed by the investigator is evaluated on a 7 point Likert scale ranging from 1='very much improved' to 7='very much worse' (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | score on a scale (Mean) | |
---|---|---|
Treatment with IB1001 | Post-Treatment Washout | |
Parent Study: | 3.4 | 4.5 |
The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). (NCT03759639)
Timeframe: CI-CS of the non-primary anchor test was evaluated, comparing the CI-CS of Visit 4 versus Visit 2 and of Visit 6 versus Visit 4 as done for the primary anchor test.
Intervention | score on a scale (Mean) | |
---|---|---|
Treatment with IB1001 | Post-treatment washout | |
Parent Study: | -0.20 | 0.15 |
The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: 'compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or 8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video?' The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse). (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment);End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | score on a scale (Mean) | |
---|---|---|
Treatment with IB1001 | Post-Treatment Washout | |
Parent Study | 0.48 | -0.38 |
Overall neurological status based on six domains (ambulation, manipulation, language, swallowing, seizures and ocular movements). The Modified Disability Rating Scale ranges from 0-24, where 0 is the best neurological status and 24 is the worst. (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | units on a scale (Mean) | |
---|---|---|
Treatment with IB1001 | Post-Treatment Washout | |
Parent Study: | -0.012 | 0.016 |
The Clinical Global Impression of Change assessed by the parent/caregiver is evaluated on a 7 point Likert scale ranging from 1='very much improved' to 7='very much worse'. (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | score on a scale (Mean) | |
---|---|---|
Treatment With IB1001 | Post-Treatment Washout | |
Parent Study: | 3.4 | 4.4 |
The Clinical Global Impression of Change assessed by the patient (if able) is evaluated on a 7 point Likert scale ranging from 1='very much improved' to 7='very much worse'. (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | score on a scale (Mean) | |
---|---|---|
Treatment With IB1001 | Post-Treatment Washout | |
Parent Study: | 3.3 | 4.3 |
The Scale for Assessment and Rating of Ataxia has 8 items that are related to gait, stance, sitting, speech, finger-chase test, nose-finger test, fast alternating movements, and heel-shin test. The range is 0-40 points, with a lower score representing neurological improvement and a higher score representing neurological worsening. (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | score on a scale (Mean) | |
---|---|---|
Treatment with IB1001 | Post-treatment washout | |
Parent Study: | -1.19 | 1.45 |
"Spinocerebellar Ataxia Functional Index (SCAFI) is composed of 8 Meter Walk Test, 9-Hole Peg Test of Dominant and Non-Dominant Hand (9HPT-D/9HPT-ND) (the 3 tests are timed assessments; each is done twice and values are averaged; the 8MWT and 9HPT-D and 9HPT-ND values are converted from times to rates, and the results expressed as a composite Z-score of each test relative to baseline) and the PATA rate (counted number how often a patient can repeat the syllables PATA within 10 seconds), a measure of speech performance. The scores of these 3 were transformed to Z-scores (=individual's average of both trials to perform the respective task - mean of study population at baseline) / SD of study population at baseline). A Z-score of 0 equates to the population mean at baseline. For all 3, higher Z-scores (above mean) mean better performance. The SCAFI total score was calculated as the arithmetic mean of the non-missing Z-scores for the 3. A higher total score means better performance." (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment); End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | Z-scores (Mean) | |
---|---|---|
Treatment with IB1001 | Post-treatment washout | |
Parent Study: | 0.0995 | 0.0076 |
"The Clinical Impression of Change in Severity assessment will instruct the blinded rater to consider: 'compared to the first video, how has the severity of their performance on the 9 Hole Peg Test of the Dominant Hand (9HPT-D) or8 Meter Walk Test (8MWT) changed (improved or worsened) in 6-weeks as observed in the second video?' The Clinical Impression of Change in Severity is evaluated on a 7 point Likert scale (+3=significantly improved to -3= significantly worse).~CI-CS scores <0 were reclassified as worsened (-1), CI-CS scores 0 remained classified as not changed (0), and CI-CS scores >0 were reclassified as improved (+1)." (NCT03759639)
Timeframe: Baseline to end of treatment with IB1001 (Parent Study 6-weeks treatment);End of treatment with IB1001 to the end of post 6-week treatment washout
Intervention | Participants (Count of Participants) | |||||
---|---|---|---|---|---|---|
Treatment with IB100172504319 | Post-treatment washout72504319 | |||||
0 (No observable Change) | -1 (Worsened) | +1 (Improved) | ||||
Parent Study: | 9 | |||||
Parent Study: | 3 | |||||
Parent Study: | 20 | |||||
Parent Study: | 16 | |||||
Parent Study: | 2 | |||||
Parent Study: | 10 |
2 trials available for acetyl-dl-leucine and Niemann-Pick Disease, Type C
Article | Year |
---|---|
N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study.
Topics: Cross-Over Studies; Double-Blind Method; Humans; Leucine; Niemann-Pick Disease, Type C; Quality of L | 2023 |
Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C.
Topics: Adolescent; Adult; Child; Double-Blind Method; Humans; Leucine; Middle Aged; Niemann-Pick Disease, T | 2022 |
Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C.
Topics: Adolescent; Adult; Child; Double-Blind Method; Humans; Leucine; Middle Aged; Niemann-Pick Disease, T | 2022 |
Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C.
Topics: Adolescent; Adult; Child; Double-Blind Method; Humans; Leucine; Middle Aged; Niemann-Pick Disease, T | 2022 |
Efficacy and safety of N-acetyl-L-leucine in Niemann-Pick disease type C.
Topics: Adolescent; Adult; Child; Double-Blind Method; Humans; Leucine; Middle Aged; Niemann-Pick Disease, T | 2022 |
2 other studies available for acetyl-dl-leucine and Niemann-Pick Disease, Type C
Article | Year |
---|---|
Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials.
Topics: Adult; Clinical Trials as Topic; Cohort Studies; Female; Humans; Leucine; Lysosomal Storage Diseases | 2018 |
Acetyl-dl-leucine in Niemann-Pick type C: A case series.
Topics: Adolescent; Adult; Female; Humans; Leucine; Male; Niemann-Pick Disease, Type C; Quality of Life; You | 2015 |