- Trials with a EudraCT protocol (92)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
92 result(s) found for: Genetic distance.
Displaying page 5 of 5.
EudraCT Number: 2011-005042-35 | Sponsor Protocol Number: PRO053-CLIN-01 | Start Date*: 2013-08-23 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053)... | |||||||||||||
Medical condition: Duchenne muscular dystrophy resulting from a mutation correctable by BMN 053-induced DMD exon 53 skipping | |||||||||||||
|
|||||||||||||
Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: GB (Prematurely Ended) BE (Completed) IT (Prematurely Ended) NL (Prematurely Ended) FR (Ongoing) | |||||||||||||
Trial results: View results |
EudraCT Number: 2010-020611-36 | Sponsor Protocol Number: AGLU07310 | Start Date*: 2011-10-12 | |||||||||||
Sponsor Name:Genzyme Europe B.V. | |||||||||||||
Full Title: A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa | |||||||||||||
Medical condition: Pompe disease (acid alpha-glucosidase deficiency) | |||||||||||||
|
|||||||||||||
Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) DE (Completed) NL (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-003992-21 | Sponsor Protocol Number: IM027-040 | Start Date*: 2020-07-03 | ||||||||||||||||
Sponsor Name:Bristol-Myers Squibb International Corporation | ||||||||||||||||||
Full Title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 2 Study of the Efficacy and the Safety and Tolerability of BMS-986278 in Participants with Pulmonary Fibrosis | ||||||||||||||||||
Medical condition: Pulmonary Fibrosis | ||||||||||||||||||
|
||||||||||||||||||
Population Age: Adults, Elderly | Gender: Male, Female | |||||||||||||||||
Trial protocol: BE (Completed) DE (Completed) IT (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2016-003475-21 | Sponsor Protocol Number: ACT14132 | Start Date*: 2017-05-17 | ||||||||||||||||
Sponsor Name:Genzyme Corporation | ||||||||||||||||||
Full Title: An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients with Infantile-onset Pompe Disease ... | ||||||||||||||||||
Medical condition: Pompe disease (acid alpha-glucosidase deficiency) | ||||||||||||||||||
|
||||||||||||||||||
Population Age: Infants and toddlers, Children, Adolescents, Under 18 | Gender: Male, Female | |||||||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) FR (Ongoing) Outside EU/EEA | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2017-001223-49 | Sponsor Protocol Number: PTC124-GD-041DMD | Start Date*: 2018-01-16 | |||||||||||
Sponsor Name:PTC Therapeutics, Inc. | |||||||||||||
Full Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension | |||||||||||||
Medical condition: Nonsense Mutation Duchenne Muscular Dystrophy | |||||||||||||
|
|||||||||||||
Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: BG (Completed) PL (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2020-003200-14 | Sponsor Protocol Number: JR-141-GS31 | Start Date*: 2022-05-16 | |||||||||||
Sponsor Name:JCR Pharmaceuticals Co., Ltd. | |||||||||||||
Full Title: A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients. | |||||||||||||
Medical condition: Mucopolysaccharidosis type II | |||||||||||||
|
|||||||||||||
Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) PL (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2017-003568-10 | Sponsor Protocol Number: VBP15-LTES | Start Date*: 2017-11-23 | |||||||||||
Sponsor Name:ReveraGen BioPharma Inc. | |||||||||||||
Full Title: A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy (DMD) | |||||||||||||
|
|||||||||||||
Population Age: Children, Under 18 | Gender: Male | ||||||||||||
Trial protocol: SE (Completed) GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2016-003257-15 | Sponsor Protocol Number: A083-02 | Start Date*: 2018-04-12 | |||||||||||
Sponsor Name:Acceleron Pharma Inc. | |||||||||||||
Full Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy | |||||||||||||
Medical condition: Facioscapulohumeral Muscular Dystrophy | |||||||||||||
|
|||||||||||||
Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2018-004837-34 | Sponsor Protocol Number: CAEB1102-300A | Start Date*: 2019-06-18 | |||||||||||
Sponsor Name:Aeglea Biotherapeutics, Inc. | |||||||||||||
Full Title: PEACE (Pegzilarginase Effect on Arginase 1 Deficiency Clinical Endpoints): A Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of the Efficacy and Safety of Pegzilarginase in Children and ... | |||||||||||||
Medical condition: Arginase 1 Deficiency Hyperargininemia | |||||||||||||
|
|||||||||||||
Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: AT (Completed) DE (Completed) FR (Completed) GB (GB - no longer in EU/EEA) IT (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2007-004819-54 | Sponsor Protocol Number: PRO051-02 | Start Date*: 2008-01-22 | |||||||||||
Sponsor Name:Prosensa Therapeutics BV | |||||||||||||
Full Title: A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscul... | |||||||||||||
Medical condition: Duchenne Muscular Dystrophy | |||||||||||||
|
|||||||||||||
Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: BE (Completed) NL (Completed) SE (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-002663-10 | Sponsor Protocol Number: 232SM203 | Start Date*: 2020-03-27 | |||||||||||
Sponsor Name:Biogen Idec Research Limited | |||||||||||||
Full Title: Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | |||||||||||||
Medical condition: Muscular Atrophy, Spinal | |||||||||||||
|
|||||||||||||
Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: LV (Completed) IE (Completed) HU (Completed) PL (Completed) ES (Ongoing) GB (GB - no longer in EU/EEA) DE (Completed) FR (Completed) GR (Completed) IT (Completed) NL (Ongoing) | |||||||||||||
Trial results: View results |
EudraCT Number: 2004-005181-20 | Sponsor Protocol Number: CEPO906A2303 | Start Date*: 2006-05-19 |
Sponsor Name:Novartis Pharma Services AG | ||
Full Title: A randomized, parallel group, open-label, active controlled, multicenter Phase III trial of Patupilone (EPO906) versus pegylated liposomal doxorubicin (Doxil/Caelyx) in taxane/platinum refractory/r... | ||
Medical condition: Approximately 75% of women with ovarian cancer present advanced disease. Survival is highly dependent on the stage of disease at the initiation of treatment. Favorable prognostic factors include yo... | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Female | |
Trial protocol: GB (Completed) ES (Completed) FI (Completed) DK (Completed) HU (Completed) GR (Completed) IT (Completed) IE (Completed) | ||
Trial results: View results |
Subscribe to this Search
To subscribe to the RSS feed for this search click here
.
This will provide an RSS feed for clinical trials matching your search that have been added or updated in the last 7 days.
To subscribe to the RSS feed for this search click here

Download Options: | |
---|---|
Number of Trials to download: | |
Download Content: | |
Download Format: | |
Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial. |
