- Trials with a EudraCT protocol (7)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
7 result(s) found for: Hereditary motor and sensory neuropathy.
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| EudraCT Number: 2019-002827-14 | Sponsor Protocol Number: TreatSPG11 | Start Date*: 2021-01-19 | |||||||||||
| Sponsor Name:IRCCS Fondazione Stella Maris | |||||||||||||
| Full Title: Phase 2 pharmacological experimental study to test the safety of miglustat in subjects with hereditary spastic paraplegia type 11 | |||||||||||||
| Medical condition: Spastic paraparesis type 11 (SPG11), caused by mutations in spatacsin protein, is characterized by the association of peripheral neuropathy, parkinsonism, ataxia, cognitive impairment, hypotrophy o... | |||||||||||||
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| Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
| Trial protocol: IT (Completed) | |||||||||||||
| Trial results: (No results available) | |||||||||||||
| EudraCT Number: 2009-012073-34 | Sponsor Protocol Number: R0000063 | Start Date*: 2009-07-08 | |||||||||||
| Sponsor Name:Radboud University Nijmegen Medical Centre | |||||||||||||
| Full Title: Botulinum toxin for treating calf muscle spasticity in Hereditary Spastic Paraparesis (HSP): functional effects on dynamic balance and gait | |||||||||||||
| Medical condition: Subjects with hereditary spastic paraparesis (HSP) | |||||||||||||
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| Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
| Trial protocol: NL (Completed) | |||||||||||||
| Trial results: View results | |||||||||||||
| EudraCT Number: 2010-022046-25 | Sponsor Protocol Number: Zellweger | Start Date*: Information not available in EudraCT | ||||||||||||||||
| Sponsor Name:Department of Pediatric Gastroenterology, Academic Medical Centre | ||||||||||||||||||
| Full Title: Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects. | ||||||||||||||||||
| Medical condition: Peroxisomal biogenesis disorder Synonym: Zellweger spectrum disorder This syndrome encompasses a spectrum of severity in symptoms Previously the disorder was subdivided into three disorders based... | ||||||||||||||||||
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| Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults | Gender: Male, Female | |||||||||||||||||
| Trial protocol: NL (Ongoing) | ||||||||||||||||||
| Trial results: (No results available) | ||||||||||||||||||
| EudraCT Number: 2006-001066-16 | Sponsor Protocol Number: H23750 | Start Date*: 2007-01-04 |
| Sponsor Name:John L. Berk, M.D. | ||
| Full Title: The effect of diflunisal (IND 68092) on familial amyloidosis | ||
| Medical condition: Familial Amyloid Polyneuropathy (FAP) | ||
| Disease: | ||
| Population Age: Adults, Elderly | Gender: Male, Female | |
| Trial protocol: SE (Completed) IT (Ongoing) GB (GB - no longer in EU/EEA) | ||
| Trial results: (No results available) | ||
| EudraCT Number: 2022-000491-18 | Sponsor Protocol Number: AT-007-1005 | Start Date*: 2022-09-14 | |||||||||||
| Sponsor Name:Applied Therapeutics Inc. | |||||||||||||
| Full Title: A Randomized, Double-Blind, Placebo-Controlled, Two-Part Study to Evaluate the Pharmacodynamic Efficacy and Clinical Benefit of AT 007 in Patients with Sorbitol Dehydrogenase (SORD) Deficiency | |||||||||||||
| Medical condition: SoRbitol Dehydrogenase (SORD) DEficiency | |||||||||||||
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| Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
| Trial protocol: CZ (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
| Trial results: (No results available) | |||||||||||||
| EudraCT Number: 2012-000467-24 | Sponsor Protocol Number: ALN-TTR02-002 | Start Date*: 2012-05-08 | ||||||||||||||||
| Sponsor Name:Alnylam Pharmaceuticals, Inc. | ||||||||||||||||||
| Full Title: A Phase 2, Open-Label, Multi-Dose, Dose Escalation Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intravenous Infusions of ALN-TTR02 in Patients with TTR Amyl... | ||||||||||||||||||
| Medical condition: Transthyretin mediated amyloidosis (ATTR) | ||||||||||||||||||
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| Population Age: Adults, Elderly | Gender: Male, Female | |||||||||||||||||
| Trial protocol: SE (Completed) PT (Completed) DE (Completed) ES (Completed) | ||||||||||||||||||
| Trial results: View results | ||||||||||||||||||
| EudraCT Number: 2020-004805-30 | Sponsor Protocol Number: CLN-PXT3003-06 | Start Date*: Information not available in EudraCT | |||||||||||
| Sponsor Name:Pharnext SCA | |||||||||||||
| Full Title: A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A) | |||||||||||||
| Medical condition: Charcot Marie Tooth Type 1A | |||||||||||||
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| Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
| Trial protocol: DE (Prematurely Ended) BE (Completed) NL (Prematurely Ended) DK (Prematurely Ended) ES (Prematurely Ended) IT (Prematurely Ended) FR (Prematurely Ended) | |||||||||||||
| Trial results: (No results available) | |||||||||||||
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