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Clinical trials for Recessive gene

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
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    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44346   clinical trials with a EudraCT protocol, of which   7374   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
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    76 result(s) found for: Recessive gene. Displaying page 1 of 4.
    1  2  3  4  Next»
    EudraCT Number: 2015-004592-74 Sponsor Protocol Number: HOLOGENE7(HTA-HG7-01) Start Date*: 2016-06-01
    Sponsor Name:Holostem Terapie Avanzate s.r.l.
    Full Title: PROSPECTIVE, OPEN LABEL, UNCONTROLLED CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF AUTOLOGOUS CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED WITH A GAMMA-RETR...
    Medical condition: RDEB is characterized by generalized skin blistering, erosions, crusts, atrophic scarring, onychodystrophy and loss of nails, mutilating pseudosyndactyly of hands and feet, as well as oral cavity l...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2017-004783-35 Sponsor Protocol Number: OPH2005 Start Date*: 2018-07-10
    Sponsor Name:IVERIC bio
    Full Title: A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects with Autosomal Recessive Stargardt Disease
    Medical condition: Autosomal Recessive Stargardt Disease
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10062766 Stargardt's disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Ongoing) GB (GB - no longer in EU/EEA) HU (Completed) ES (Ongoing) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-000691-19 Sponsor Protocol Number: BN43881 Start Date*: 2022-10-26
    Sponsor Name:F. Hoffmann-La Roche Ltd
    Full Title: A two-part, open-label systemic gene delivery study to evaluate the safety and expression of RO7494222 (SRP-9001) in subjects under the age of four with Duchenne muscular dystrophy
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Ongoing) DE (Trial now transitioned) BE (Trial now transitioned) IT (Trial now transitioned) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2017-004806-17 Sponsor Protocol Number: PQ-313-002 Start Date*: 2018-05-18
    Sponsor Name:Wings Therapeutics Inc.
    Full Title: A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subj...
    Medical condition: Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10014989 Epidermolysis bullosa PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Prematurely Ended) DE (Prematurely Ended) CZ (Completed) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2020-004803-15 Sponsor Protocol Number: 5051-102 Start Date*: 2023-09-18
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: An Open-Label Extension Study for Patients with Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-024566-22 Sponsor Protocol Number: DMD114118 Start Date*: Information not available in EudraCT
    Sponsor Name:GlaxoSmithKline Research and Development LTD
    Full Title: A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subject...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    12.1 10013801 Duchenne muscular dystrophy LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: FR (Ongoing) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2021-000077-83 Sponsor Protocol Number: SRP-9001-101 Start Date*: 2022-11-10
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: Systemic gene delivery Phase I/IIa clinical trial for Duchenne muscular dystrophy using rAAVrh74.MHCK7.micro-dystrophin (microDys-IV-001)
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-000095-17 Sponsor Protocol Number: HOLOGENE17 Start Date*: 2017-08-17
    Sponsor Name:Holostem Terapie Avanzate s.r.l.
    Full Title: PROSPECTIVE, OPEN-LABEL, UNCONTROLLED CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF AUTOLOGOUS CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED WITH A GAMMA-RETR...
    Medical condition: JEB is genetically and clinically heterogeneous characterized by mutations in COL17A1,encoding for Collagen17 protein. COLXVII is a collagenous transmembrane type II protein component of the hemide...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: AT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2016-002790-35 Sponsor Protocol Number: C12-48 Start Date*: 2019-06-04
    Sponsor Name:INSERM
    Full Title: Phase I/II ex vivo gene therapy clinical trial for RDEB using autologous skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector
    Medical condition: The trial aims to treat the recessive dystrophic epidermolysis bullosa (RDEB) by grafting one to three subjects with RDEB with autologous COL7A1-modified skin equivalents, using SIN-RV encoding COL...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004858 10074980 Epidermolysis bullosa aquisita LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2010-020069-26 Sponsor Protocol Number: DMD114044 Start Date*: Information not available in EudraCT
    Sponsor Name:GlaxoSmithKline Research and Development LTD
    Full Title: A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy.
    Medical condition: Duchenne muscular dystrophy
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: DE (Completed) FR (Completed) NL (Completed) BE (Completed) IT (Completed) PL (Completed) CZ (Completed) ES (Completed) NO (Completed) DK (Completed) HU (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-018412-32 Sponsor Protocol Number: DMD114117 Start Date*: 2010-08-05
    Sponsor Name:GlaxoSmithKline Research and Development LTD
    Full Title: A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambul...
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: DE (Completed) BE (Completed) GB (Completed) NL (Completed) FR (Completed) ES (Completed) Outside EU/EEA
    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
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    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: 2017-004139-35 Sponsor Protocol Number: MNK14112096 Start Date*: 2018-06-12
    Sponsor Name:Mallinckrodt ARD Inc.
    Full Title: A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Mus...
    Medical condition: Duchenne Muscular Distrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: ES (Prematurely Ended) BG (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-000078-27 Sponsor Protocol Number: SRP-9001-102 Start Date*: 2022-11-10
    Sponsor Name:Sarepta Therapeutics, Inc.
    Full Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10052655 Duchenne muscular dystrophy gene carrier PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2012-004527-20 Sponsor Protocol Number: PTC124-GD-020-DMD Start Date*: 2013-07-30
    Sponsor Name:PTC Therapeutics, Inc
    Full Title: A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy
    Medical condition: Nonsense mutation dystrophinopathy
    Disease: Version SOC Term Classification Code Term Level
    17.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    17.0 10010331 - Congenital, familial and genetic disorders 10059117 Becker's muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Completed) CZ (Completed) SE (Completed) GB (Completed) DE (Completed) IT (Completed) ES (Completed) PL (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2010-021343-41 Sponsor Protocol Number: ME040 Start Date*: 2014-03-21
    Sponsor Name:University Medical Center Groningen
    Full Title: Effect of sapropterin on variations of blood phenylalanine and tyrosine over 24 hours and from day to day in children with phenylketonuria
    Medical condition: Phenylketonuria (PKU) is an autosomal recessive metabolic genetic disorder by a mutation in the gene for the enzyme phenylalanine hydroxylase (PAH), rendering it nonfunctional. Left untreated, the ...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-004262-26 Sponsor Protocol Number: VBP15-002 Start Date*: 2017-02-13
    Sponsor Name:ReveraGen BioPharma Inc.
    Full Title: A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular ...
    Medical condition: Duchenne Muscular Dystrophy (DMD)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: SE (Completed) GB (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)
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: BE (Completed)
    Trial results: View results
    EudraCT Number: 2015-004333-27 Sponsor Protocol Number: SMTC11005 Start Date*: 2016-01-19
    Sponsor Name:Summit (Oxford) Limited
    Full Title: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005)
    Medical condition: Duchenne Muscular Dystrophy
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10013801 Duchenne muscular dystrophy PT
    Population Age: Children, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2015-003670-32 Sponsor Protocol Number: REFLECT Start Date*: Information not available in EudraCT
    Sponsor Name:Medical Center - University of Freiburg
    Full Title: A dual-center prospective phase I/II trial to establish safety, tolerability and to obtain first data on efficacy of losartan in children with recessive dystrophic epidermolysis bullosa (RDEB)
    Medical condition: Recessive dystrophic epidermolysis bullosa (RDEB)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10014989 Epidermolysis bullosa PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) AT (Completed)
    Trial results: View results
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