- Trials with a EudraCT protocol (29)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
29 result(s) found for: Gene structure.
Displaying page 1 of 2.
EudraCT Number: 2016-003967-21 | Sponsor Protocol Number: MGT009 | Start Date*: 2017-06-09 | |||||||||||
Sponsor Name:MeiraGTx UK II Limited | |||||||||||||
Full Title: An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigme... | |||||||||||||
Medical condition: X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-001383-18 | Sponsor Protocol Number: | Start Date*: 2015-11-30 | |||||||||||
Sponsor Name:University of Oxford | |||||||||||||
Full Title: An open label Phase 2 clinical trial of retinal gene therapy for choroideremia using an adeno-associated viral vector (AAV2) encoding Rab-escort protein 1 (REP1) | |||||||||||||
Medical condition: Choroideremia - a currently incurable retinal degeneration caused by null mutations in the CHM gene encoding REP1 protein located on the X chromosome. The condition causes blindness in males by the... | |||||||||||||
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Population Age: Adults | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-003595-36 | Sponsor Protocol Number: ERN_11-0670 | Start Date*: 2012-04-26 |
Sponsor Name:University of Birmingham | ||
Full Title: Can muscle dysfunction in COPD be altered by oxygenation in patients with intermittent hypoxia on exertion? | ||
Medical condition: Chronic obstructive pulmonary disease | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: GB (Completed) | ||
Trial results: View results |
EudraCT Number: 2008-003059-56 | Sponsor Protocol Number: ICAND | Start Date*: 2008-07-29 | |||||||||||
Sponsor Name:ISTITUTO NEUROLOGICO "CARLO BESTA" | |||||||||||||
Full Title: Phase II trial to assess safety and efficacy of Iron chelating agent Deferiprone in patients with Pantothenate Kinase-Associated Neurodegeneration ICAND Trial | |||||||||||||
Medical condition: PKAN | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: IT (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2020-000712-30 | Sponsor Protocol Number: ABR-71432 | Start Date*: 2021-06-07 | |||||||||||
Sponsor Name:Radboudumc | |||||||||||||
Full Title: Targeted glucocorticoid administration to improve safety learning in PTSD patients with HPA axis dysregulation | |||||||||||||
Medical condition: Posttraumatic Stress Disorder (PTSD) | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: NL (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2007-005808-41 | Sponsor Protocol Number: EC 07/90601 | Start Date*: 2008-02-25 | |||||||||||
Sponsor Name:Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | |||||||||||||
Full Title: Estudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con Calcifediol Multicentric study to assess the clinica... | |||||||||||||
Medical condition: Portadores sintomaticos de una mutación en el gen disferlina. Symptomatic carriers of a mutation in the dysferlin gene | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Ongoing) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2018-004740-36 | Sponsor Protocol Number: PTCEMF-GD-004 | Start Date*: 2019-10-14 | |||||||||||
Sponsor Name:PTC Therapeutics, Inc. | |||||||||||||
Full Title: A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | |||||||||||||
Medical condition: Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Prematurely Ended) FR (Completed) DK (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2013-000410-38 | Sponsor Protocol Number: LP0066-1019 | Start Date*: 2013-10-25 | |||||||||||
Sponsor Name:LEO Pharma A/S | |||||||||||||
Full Title: Topical Aprepitant in Prurigo Patients (iTAPP) An exploratory phase IIa trial with topically applied aprepitant in patients with prurigo | |||||||||||||
Medical condition: Prurigo can present either with hyperkeratotic nodules as prurigo nodularis or without nodules as Prurigo simplex. The disease is characterised by intensely pruritic, lichenified, or excoriated pap... | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2018-004393-10 | Sponsor Protocol Number: GN18CA068 | Start Date*: 2019-07-17 | |||||||||||
Sponsor Name:NHS Greater Glasgow & Clyde [...] | |||||||||||||
Full Title: A randomised, double-blind, placebo-controlled, cross-over trial of zibotentan in microvascular angina | |||||||||||||
Medical condition: Microvascular Angina and impaired exercise intolerance. | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2019-004219-29 | Sponsor Protocol Number: 140041 | Start Date*: 2020-04-21 |
Sponsor Name:Amsterdam UMC | ||
Full Title: “Personalized AZithromycin/metronidAZole, in combination with standard induction therapy, to achieve a fecal microbiome community structure and metagenome changes associated with sustained remissio... | ||
Medical condition: Crohn's Disease | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | |
Trial protocol: NL (Completed) | ||
Trial results: (No results available) |
EudraCT Number: 2018-000425-31 | Sponsor Protocol Number: MGT010 | Start Date*: 2018-08-30 | |||||||||||
Sponsor Name:MeiraGTx UK II Ltd | |||||||||||||
Full Title: Long term follow-up study of participants following an open label, multicentre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy o... | |||||||||||||
Medical condition: X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) Outside EU/EEA | |||||||||||||
Trial results: (No results available) |
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: 2019-003303-35 | Sponsor Protocol Number: IB2019-04 | Start Date*: 2021-04-08 | ||||||||||||||||||||||||||||||||||||
Sponsor Name:Institut Bergonié | ||||||||||||||||||||||||||||||||||||||
Full Title: Combining epigenetic and immune therapy to beat cancer | ||||||||||||||||||||||||||||||||||||||
Medical condition: | ||||||||||||||||||||||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | |||||||||||||||||||||||||||||||||||||
Trial protocol: FR (Trial now transitioned) | ||||||||||||||||||||||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2015-000359-26 | Sponsor Protocol Number: ESTEVE-SANF-201 | Start Date*: 2016-04-29 |
Sponsor Name:Laboratorios del Dr. Esteve, S.A. | ||
Full Title: Phase I/II safety, tolerability and initial efficacy study of adeno-associated viral vector serotype 9 containing human sulfamidase gene after intracerebroventricular administration to patients wit... | ||
Medical condition: Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) is an inherited lysosomal storage disease caused by a specific lysosomal enzyme deficiency that leads to intracellular accumulation of the ... | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | |
Trial protocol: ES (Ongoing) | ||
Trial results: (No results available) |
EudraCT Number: 2014-004830-25 | Sponsor Protocol Number: KIT | Start Date*: 2015-09-01 | |||||||||||
Sponsor Name:Oslo University Hospital | |||||||||||||
Full Title: Kidney Cancer Integrated Therapy (KIT) - Personalized integrated therapy for patients with advanced kidney cancer | |||||||||||||
Medical condition: Personalized integrated therapy for patients with advanced kidney cancer | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: NO (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2010-023111-34 | Sponsor Protocol Number: SG1/001/10 | Start Date*: 2011-07-18 |
Sponsor Name:Oxford BioMedica (UK) Ltd | ||
Full Title: A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration. | ||
Medical condition: Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease. | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: FR (Completed) NL (Not Authorised) IT (Prematurely Ended) Outside EU/EEA | ||
Trial results: View results |
EudraCT Number: 2015-004143-39 | Sponsor Protocol Number: SHIP002 | Start Date*: 2016-08-09 | ||||||||||||||||
Sponsor Name:Erasmus MC | ||||||||||||||||||
Full Title: A Phase 3 randomised, double-blind, controlled trial of inhaled 7% hypertonic saline versus 0.9% isotonic saline for 48 weeks in patients with Cystic Fibrosis at 3-6 years of age in parallel with t... | ||||||||||||||||||
Medical condition: Cystic Fibrosis | ||||||||||||||||||
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Population Age: Children, Under 18 | Gender: Male, Female | |||||||||||||||||
Trial protocol: NL (Completed) BE (Completed) DK (Completed) FR (Completed) ES (Completed) IT (Completed) | ||||||||||||||||||
Trial results: View results |
EudraCT Number: 2018-004689-32 | Sponsor Protocol Number: SHP675-301 | Start Date*: 2021-09-24 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies, Inc. | |||||||||||||
Full Title: A Phase 3, Open-label Study to Evaluate the Efficacy and Safety of REPLAGAL® in Treatment-naïve Subjects with Fabry Disease | |||||||||||||
Medical condition: Fabry disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: PT (Prematurely Ended) FI (Prematurely Ended) GR (Prematurely Ended) PL (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-001382-10 | Sponsor Protocol Number: CS/2015/4775 | Start Date*: 2019-01-21 | |||||||||||
Sponsor Name:University Hospitals Bristol NHS Foundation Trust | |||||||||||||
Full Title: Treatment of Barth Syndrome by CARDIOlipin MANipulation (CARDIOMAN): A randomised placebo controlled pilot trial conducted by the nationally commissioned Barth Syndrome Service | |||||||||||||
Medical condition: Barth Syndrome is a rare, life threatening, genetic disease which affects young males. It is caused by abnormal fats (lipids) in the powerhouses of cells (mitochondria) and those who suffer with it... | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-000502-19 | Sponsor Protocol Number: PSY-NIL-0002 | Start Date*: 2016-06-21 |
Sponsor Name:Medical University of Vienna, Department of Psychiatry and Psychotherapy | ||
Full Title: Effects of sex steroid hormones on serotonin synthesis and degradation measured with PET | ||
Medical condition: Gender Dysphoria (Female-to-male transsexuals and male-to-female transsexuals) | ||
Disease: | ||
Population Age: Adults | Gender: Male, Female | |
Trial protocol: AT (Completed) | ||
Trial results: (No results available) |
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