- 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 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 (Completed) | ||||||||||||||||||
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 (Trial now transitioned) SE (Completed) DE (Completed) FR (Ongoing) ES (Trial now transitioned) CZ (Trial now transitioned) NL (Ongoing) IT (Trial now transitioned) BG (Trial now transitioned) PL (Trial now transitioned) IE (Trial now transitioned) DK (Trial now transitioned) GR (Completed) 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 (Completed) NO (Completed) ES (Completed) IT (Completed) NL (Completed) DE (Completed) | |||||||||||||||||||||||
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. (Shire), a wholly owned subsidiary of Takeda Pharmaceutical Company Limited | ||
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 (Completed) | ||
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 (Completed) ES (Ongoing) NL (Completed) NO (Completed) | |||||||||||||
Trial results: View results |
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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