- Trials with a EudraCT protocol (91)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
91 result(s) found for: Genetic Distance.
Displaying page 1 of 5.
EudraCT Number: 2015-003521-34 | Sponsor Protocol Number: CHUBX2015/14 | Start Date*: 2015-10-09 |
Sponsor Name:CHU de Bordeaux | ||
Full Title: Hypertrophic CARdiomyopathy symptom release by alpha stimulant MIDOdrine | ||
Medical condition: Hypertrophic cardiomyopathy | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: FR (Ongoing) | ||
Trial results: (No results available) |
EudraCT Number: 2007-007725-46 | Sponsor Protocol Number: ASBI 307 | Start Date*: 2009-02-06 |
Sponsor Name:Asubio Pharmaceuticals, Inc. | ||
Full Title: A Phase 2b, Multicenter, Randomized, Double-Blind, Parallel, Placebo-Controlled Study to Evaluate the Efficacy and Safety of SUN11031 for Injection Administered Subcutaneously Twice Daily for 12 We... | ||
Medical condition: Cachexia associated with chronic obstructive pulmonary disease | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: BG (Completed) | ||
Trial results: View results |
EudraCT Number: 2018-000692-32 | Sponsor Protocol Number: FARMPre-Proposalcode:TRS-2016-00 | Start Date*: 2019-07-17 | ||||||||||||||||
Sponsor Name:DIP. MEDICINA DEI SISTEMI UNIVERSITà DEGLI STUDI DI ROMA TOR VERGATA | ||||||||||||||||||
Full Title: EFFICACY OF METFORMIN ON MOTILITY AND STRENGTH IN MYOTONIC DYSTROPHY TYPE 1. A randomized, double blind, placebo-controlled, multicenter clinical trial. | ||||||||||||||||||
Medical condition: Myotonic Distrophy type 1, confirmed by genetic testing, with a CTG expansion size >100 | ||||||||||||||||||
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Population Age: Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: IT (Ongoing) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2015-002069-52 | Sponsor Protocol Number: 4045-301 | Start Date*: 2017-01-23 | |||||||||||
Sponsor Name:Sarepta Therapeutics, Inc. | |||||||||||||
Full Title: A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy | |||||||||||||
Medical condition: Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) BE (Ongoing) SE (Completed) DE (Completed) FR (Ongoing) ES (Ongoing) CZ (Ongoing) NL (Ongoing) IT (Ongoing) BG (Ongoing) PL (Ongoing) IE (Ongoing) DK (Ongoing) GR (Ongoing) NO (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2021-003907-16 | Sponsor Protocol Number: SPIMD-301 | Start Date*: 2022-03-02 | |||||||||||||||||||||
Sponsor Name:Stealth BioTherapeutics Inc. | |||||||||||||||||||||||
Full Title: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondr... | |||||||||||||||||||||||
Medical condition: Primary Mitochondrial Disease Resulting from Pathogenic Nuclear DNA Mutations (nPMD) | |||||||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||||||||||||
Trial protocol: HU (Ongoing) NO (Ongoing) ES (Ongoing) IT (Ongoing) NL (Ongoing) DE (Ongoing) | |||||||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2009-015985-75 | Sponsor Protocol Number: HGT-REP-060 | Start Date*: 2010-02-03 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies (HGT), Inc. | |||||||||||||
Full Title: An Open-label Extension of Study TKT028 Evaluating Safety and Clinical Outcomes of Replagal Enzyme Replacement Therapy Administered to Adult Patients with Fabry Disease | |||||||||||||
Medical condition: Fabry disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: PL (Completed) CZ (Completed) GB (Completed) FI (Completed) SI (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-002447-15 | Sponsor Protocol Number: SPIMM-301 | Start Date*: 2018-06-22 | |||||||||||
Sponsor Name:Stealth BioTherapeutics Inc. | |||||||||||||
Full Title: A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondr... | |||||||||||||
Medical condition: Primary Mitochondrial Myopathy | |||||||||||||
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Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Prematurely Ended) DK (Prematurely Ended) DE (Completed) HU (Prematurely Ended) IT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-001629-41 | Sponsor Protocol Number: DSC/15/2357/53 | Start Date*: 2018-11-15 | |||||||||||
Sponsor Name:ITALFARMACO S.P.A. | |||||||||||||
Full Title: A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscu... | |||||||||||||
Medical condition: Distrofia Muscolare di Becker (DMB) | |||||||||||||
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Population Age: Adults | Gender: Male | ||||||||||||
Trial protocol: NL (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-000621-12 | Sponsor Protocol Number: ISIS396443-CS3A | Start Date*: 2017-02-15 |
Sponsor Name:Ionis Pharmaceuticals, Inc. | ||
Full Title: A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy | ||
Medical condition: Spinal Muscular Atrophy (SMA) | ||
Disease: | ||
Population Age: Infants and toddlers, Under 18 | Gender: Male, Female | |
Trial protocol: Outside EU/EEA | ||
Trial results: View results |
EudraCT Number: 2021-003674-32 | Sponsor Protocol Number: ULRICA-PILOT | Start Date*: 2022-12-15 |
Sponsor Name:Region Västerbotten | ||
Full Title: A pilot study to evaluate the safety and efficacy of SGLT2 inhibitors in patients with cardiac amyloidosis | ||
Medical condition: Patients with cardiac amyloidosis. | ||
Disease: | ||
Population Age: Adults | Gender: Male, Female | |
Trial protocol: SE (Ongoing) | ||
Trial results: (No results available) |
EudraCT Number: 2014-004804-31 | Sponsor Protocol Number: SHP-ELA-401 | Start Date*: 2016-04-12 |
Sponsor Name:Shire Human Genetic Therapies, Inc | ||
Full Title: A Long-Term, Open-Label, Multicenter, Phase IV Study to Assess Longitudinal Changes on Height and Weight in Patients with MPS II Who Are Receiving Elaprase and Started Treatment With Elaprase at <6... | ||
Medical condition: Hunter syndrome (Mucopolysaccharidosis II, [MPS II]) | ||
Disease: | ||
Population Age: Children, Under 18 | Gender: Male | |
Trial protocol: DE (Ongoing) | ||
Trial results: (No results available) |
EudraCT Number: 2020-002855-40 | Sponsor Protocol Number: REN001-201 | Start Date*: 2021-04-15 | |||||||||||
Sponsor Name:Reneo Pharma Ltd. | |||||||||||||
Full Title: A DOUBLE-BLIND, PLACEBO-CONTROLLED, STUDY TO EVALUATE THE EFFICACY AND SAFETY OF 24 WEEKS TREATMENT WITH REN001 IN PATIENTS WITH PRIMARY MITOCHONDRIAL MYOPATHY (PMM) | |||||||||||||
Medical condition: Primary Mitochondrial Myopathy | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Completed) CZ (Completed) DK (Completed) HU (Completed) BE (Completed) IT (Ongoing) ES (Ongoing) NL (Completed) NO (Completed) | |||||||||||||
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: 2015-005333-49 | Sponsor Protocol Number: ALN-TTRSC-006 | Start Date*: 2016-06-30 | |||||||||||
Sponsor Name:Alnylam Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Multicenter, Multinational, Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Revusiran in Patients with Transthyretin-mediated Familial Amyloidotic Cardiomyopathy | |||||||||||||
Medical condition: Transthyretin (TTR) mediated familial amyloidotic cardiomyopathy (FAC) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2008-006441-10 | Sponsor Protocol Number: 116-02 | Start Date*: 2009-06-03 |
Sponsor Name:SCIPHARM SáRL | ||
Full Title: A double blind controlled clinical study to investigate the efficacy and tolerability of subcutaneous Treprostinil sodium in patients with severe non-operable Chronic Thromboembolic Pulmonary Hyper... | ||
Medical condition: Patients with Severe (inoperable) Chronic Thromboembolic Pulmonary Hypertension | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: AT (Completed) CZ (Completed) SK (Completed) DE (Prematurely Ended) | ||
Trial results: View results |
EudraCT Number: 2019-001147-51 | Sponsor Protocol Number: COMPIS | Start Date*: 2020-03-31 | |||||||||||
Sponsor Name:Västra götalandsregionen | |||||||||||||
Full Title: Congenital myopathy intervention study | |||||||||||||
Medical condition: Congenital myopathy | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: SE (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2014-005638-71 | Sponsor Protocol Number: UX003-CL301 | Start Date*: 2014-12-22 |
Sponsor Name:Ultragenyx Pharmaceutical Inc. | ||
Full Title: A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients with MPS 7 | ||
Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome) | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |
Trial protocol: Outside EU/EEA | ||
Trial results: View results |
EudraCT Number: 2016-002523-28 | Sponsor Protocol Number: KHAK1001 | Start Date*: 2016-09-16 | |||||||||||
Sponsor Name:Moorfields Eye Hospital | |||||||||||||
Full Title: A Pilot Study Evaluating the Effect of Intravitreal Fluocinolone Acetonide (0.19mg) in Patients with Retinitis Pigmentosa | |||||||||||||
Medical condition: Retinitis Pigmetosa. | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2018-001436-22 | Sponsor Protocol Number: APL2-303 | Start Date*: 2018-12-07 | |||||||||||
Sponsor Name:Apellis Pharmaceuticals Inc. | |||||||||||||
Full Title: A Phase 3, Multi-Center, Randomized, Double-Masked, Sham-Controlled Study to Compare the Efficacy and Safety of Intravitreal Pegcetacoplan Therapy with Sham Injections in Patients with Geographic A... | |||||||||||||
Medical condition: Geographic Atrophy Secondary to Age-Related Macular Degeneration | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) CZ (Completed) DE (Completed) ES (Ongoing) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2013-003771-35 | Sponsor Protocol Number: UX007G-CL201 | Start Date*: 2014-02-11 | |||||||||||
Sponsor Name:Ultragenyx Pharmaceutical Inc | |||||||||||||
Full Title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Adaptive Study to Assess the Safety and Efficacy of UX007 in Subjects with Glucose Transporter Type 1 Deficiency Syndrome | |||||||||||||
Medical condition: Glucose Transporter Type 1 deficiency syndrome | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) GB (Completed) FR (Ongoing) HU (Completed) ES (Completed) DK (Prematurely Ended) | |||||||||||||
Trial results: View results |
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