- Trials with a EudraCT protocol (189)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
189 result(s) found for: Motor Neuron Disease.
Displaying page 7 of 10.
EudraCT Number: 2013-005489-20 | Sponsor Protocol Number: PTC124-GD-020e-DMD | Start Date*: 2014-08-22 | ||||||||||||||||
Sponsor Name:PTC Therapeutics, Inc. | ||||||||||||||||||
Full Title: A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | ||||||||||||||||||
Medical condition: Nonsense mutation dystrophinopathy | ||||||||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | |||||||||||||||||
Trial protocol: SE (Completed) GB (Completed) BE (Completed) DE (Completed) IT (Completed) ES (Completed) CZ (Completed) FR (Completed) BG (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2011-004369-34 | Sponsor Protocol Number: UMC-NMZ-SMA2011 | Start Date*: 2015-04-22 | ||||||||||||||||
Sponsor Name:Universtiy Medical Center Utrecht | ||||||||||||||||||
Full Title: SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients ... | ||||||||||||||||||
Medical condition: Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene... | ||||||||||||||||||
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Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | |||||||||||||||||
Trial protocol: NL (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2015-003195-68 | Sponsor Protocol Number: 2015-003195-68 | Start Date*: 2015-11-11 | |||||||||||
Sponsor Name:The Newcastle upon Tyne NHS Hospitals Foundation Trust | |||||||||||||
Full Title: Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy. | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy | |||||||||||||
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Population Age: Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2020-005522-28 | Sponsor Protocol Number: ION363-CS1 | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:Ionis Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients with Fused in Sarcoma Mutati... | |||||||||||||
Medical condition: Amyotrophic Lateral Sclerosis with Fused in Sarcoma mutations | |||||||||||||
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Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Trial now transitioned) IT (Trial now transitioned) BE (Trial now transitioned) NL (Trial now transitioned) SE (Trial now transitioned) IE (Trial now transitioned) PL (Trial now transitioned) ES (Ongoing) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2017-002754-36 | Sponsor Protocol Number: 3119002 | Start Date*: 2018-05-08 | |||||||||||
Sponsor Name:Orion Corporation | |||||||||||||
Full Title: Effects of oral Levosimendan (ODM-109) on respiratory function in patients with ALS | |||||||||||||
Medical condition: Amyotrophic lateral sclerosis (ALS) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: IE (Completed) GB (Completed) BE (Completed) FI (Completed) AT (Completed) ES (Completed) NL (Completed) FR (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2016-000602-10 | Sponsor Protocol Number: SNT-III-012 | Start Date*: 2016-11-17 | |||||||||||
Sponsor Name:Santhera Pharmaceuticals (Switzerland) Limited | |||||||||||||
Full Title: A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Ste... | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy (DMD) | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
Trial protocol: DE (Prematurely Ended) BE (Completed) NL (Prematurely Ended) SE (Prematurely Ended) GB (Prematurely Ended) ES (Prematurely Ended) AT (Prematurely Ended) FR (Completed) IT (Prematurely Ended) IE (Completed) HU (Prematurely Ended) BG (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2022-002301-24 | Sponsor Protocol Number: NMD670-02-0001 | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:NMD Pharma A/S | |||||||||||||
Full Title: A Phase 2, randomised, double-blind, placebo-controlled, 2-way crossover study to evaluate the efficacy, safety, and tolerability of NMD670 in ambulatory adults with Type 3 spinal muscular atrophy | |||||||||||||
Medical condition: Type 3 spinal muscular atrophy | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Ongoing) BE (Trial now transitioned) DK (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2017-004554-42 | Sponsor Protocol Number: TAMDMD | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:University of Basel Children's Hospital, Division of Neuropediatrics | |||||||||||||
Full Title: Tamoxifen in Duchenne muscular dystrophy: A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial Tamoxifen in Duchenne muscular dystrophy: A 48-week... | |||||||||||||
Medical condition: Duchenne muscular dystrophy | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: DE (Prematurely Ended) GB (GB - no longer in EU/EEA) FR (Completed) ES (Prematurely Ended) NL (Prematurely Ended) BE (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2013-004422-29 | Sponsor Protocol Number: ISIS396443-CS3B | Start Date*: 2014-11-13 | |||||||||||
Sponsor Name:Isis Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients with Infantile-onset Spinal Mus... | |||||||||||||
Medical condition: Spinal Muscular Atrophy (SMA) | |||||||||||||
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Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) GB (Completed) ES (Completed) SE (Completed) DE (Completed) BE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2018-004383-65 | Sponsor Protocol Number: SRK-015-002 | Start Date*: 2019-10-30 | ||||||||||||||||
Sponsor Name:Scholar Rock, Inc. | ||||||||||||||||||
Full Title: Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy | ||||||||||||||||||
Medical condition: Later Onset Spinal Muscular Atrophy (SMA) | ||||||||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: NL (Completed) ES (Ongoing) DE (Completed) IT (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2013-001194-25 | Sponsor Protocol Number: H6D-MC-LVJJ | Start Date*: 2013-09-25 | |||||||||||
Sponsor Name:Eli Lilly and Company | |||||||||||||
Full Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: GB (Prematurely Ended) IT (Prematurely Ended) DE (Completed) ES (Prematurely Ended) BE (Prematurely Ended) NL (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2020-000699-39 | Sponsor Protocol Number: FGCL-3019-094 | Start Date*: 2021-11-04 | |||||||||||
Sponsor Name:FibroGen, Inc. | |||||||||||||
Full Title: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD) | |||||||||||||
Medical condition: Ambulatory Duchenne Muscular Dystrophy | |||||||||||||
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Population Age: Children, Under 18 | Gender: Male | ||||||||||||
Trial protocol: FR (Completed) BE (Completed) AT (Prematurely Ended) NL (Completed) IT (Prematurely Ended) ES (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2016-000778-40 | Sponsor Protocol Number: BP39056 | Start Date*: 2017-01-05 | |||||||||||
Sponsor Name:F. Hoffmann-La Roche Ltd | |||||||||||||
Full Title: A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | |||||||||||||
Medical condition: Type 1 Spinal Muscular Atrophy (SMA) | |||||||||||||
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Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) ES (Prematurely Ended) IT (Completed) BE (Completed) FR (Completed) PL (Completed) HR (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2014-005296-81 | Sponsor Protocol Number: BMN-051-303 | Start Date*: 2016-05-02 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne ... | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy (DMD) | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: BE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-003374-91 | Sponsor Protocol Number: SRP-9001-301 | Start Date*: 2022-09-12 | ||||||||||||||||
Sponsor Name:Sarepta Therapeutics, Inc. | ||||||||||||||||||
Full Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBA... | ||||||||||||||||||
Medical condition: Duchenne Muscular Dystrophy | ||||||||||||||||||
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Population Age: Children, Under 18 | Gender: Male | |||||||||||||||||
Trial protocol: FR (Completed) BE (Completed) ES (Completed) DE (Completed) IT (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2015-003681-87 | Sponsor Protocol Number: BMN-044-201 | Start Date*: 2016-01-12 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy | |||||||||||||
Medical condition: Duchenne muscular dystrophy | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
Trial protocol: BE (Completed) IT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-001955-54 | Sponsor Protocol Number: BMN-051-302 | Start Date*: 2015-12-08 | |||||||||||
Sponsor Name:BioMarin Nederland BV | |||||||||||||
Full Title: An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy (DMD) | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
Trial protocol: NL (Prematurely Ended) DE (Completed) BE (Completed) ES (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2020-004901-29 | Sponsor Protocol Number: NBK154/1/2020 | Start Date*: 2021-06-01 | |||||||||||
Sponsor Name:Medical University of GdaĆsk | |||||||||||||
Full Title: The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-17 years. A randomized, double-blind, ... | |||||||||||||
Medical condition: Duchenne muscular dystrophy Cardiomyopathy Tachycardia | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: PL (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2009-013762-63 | Sponsor Protocol Number: PRO044-CLIN-01 | Start Date*: 2010-04-16 |
Sponsor Name:Prosensa Therapeutics B.V | ||
Full Title: A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular d... | ||
Medical condition: Duchenne Muscular Dystrophy | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18 | Gender: Male | |
Trial protocol: NL (Completed) BE (Completed) SE (Completed) IT (Completed) | ||
Trial results: View results |
EudraCT Number: 2020-000698-26 | Sponsor Protocol Number: FGCL-3019-093 | Start Date*: 2023-01-17 | |||||||||||
Sponsor Name:FibroGen, Inc. | |||||||||||||
Full Title: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) | |||||||||||||
Medical condition: Non-ambulatory Duchenne Muscular Dystrophy | |||||||||||||
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Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male | ||||||||||||
Trial protocol: FR (Prematurely Ended) AT (Prematurely Ended) CZ (Prematurely Ended) NL (Prematurely Ended) BE (Prematurely Ended) GB (GB - no longer in EU/EEA) IT (Prematurely Ended) ES (Prematurely Ended) | |||||||||||||
Trial results: View results |
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