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Clinical trials for Adeno-associated virus

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
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • 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   43977   clinical trials with a EudraCT protocol, of which   7312   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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    50 result(s) found for: Adeno-associated virus. Displaying page 1 of 3.
    1  2  3  Next»
    EudraCT Number: 2015-003958-41 Sponsor Protocol Number: NSR-REP-01 Start Date*: 2016-10-31
    Sponsor Name:NightstaRx Ltd
    Full Title: A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial Of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (...
    Medical condition: Choroideremia
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004853 10008791 Choroideremia LLT
    Population Age: Adults Gender: Male
    Trial protocol: DE (Completed) FI (Completed) DK (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2017-003104-42 Sponsor Protocol Number: NSR-CHM-OS2(273CH201) Start Date*: 2018-01-05
    Sponsor Name:NightstaRx Ltd (A Biogen Company)
    Full Title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Prot...
    Medical condition: Choroideremia (CHM) X-Linked Retinitis Pigmentosa (XLRP)
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004853 10008791 Choroideremia LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10038914 Retinitis pigmentosa PT
    Population Age: Adults Gender: Male
    Trial protocol: DE (Ongoing) GB (GB - no longer in EU/EEA) FI (Ongoing) FR (Ongoing) DK (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-004949-32 Sponsor Protocol Number: P1-GM-101 Start Date*: 2021-03-30
    Sponsor Name:Lysogene
    Full Title: An open-label adaptive-design study of intracisternal administration of adeno-associated viral vector serotype rh.10 carrying the human β-galactosidase cDNA for the treatment of GM1 gangliosidosis
    Medical condition: GM1 gangliosidosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2018-004024-11 Sponsor Protocol Number: SHP648-101 Start Date*: 2020-05-04
    Sponsor Name:Baxalta Innovations GmbH
    Full Title: An Open-Label, Multinational, Phase 1/2 Study of the Safety and Dose Escalation of SHP648, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing FIX Padua in Hemophilia B Subjects
    Medical condition: Hemophilia B is a X-linked recessive bleeding disorder caused by mutations in the gene encoding clotting factor IX (FIX) that result in disruption of the normal clotting pathway. Hemophilia B affec...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: DE (Prematurely Ended) HU (Prematurely Ended) ES (Prematurely Ended) AT (Prematurely Ended) FR (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-000206-28 Sponsor Protocol Number: SB-318-1502 Start Date*: 2018-08-14
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase 1/2, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis I (MPS I)
    Medical condition: Mucopolysaccharidosis type I (MPS I)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056886 Mucopolysaccharidosis I PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-000192-33 Sponsor Protocol Number: SB-913-1602 Start Date*: 2018-08-17
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase 1/2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPS II)
    Medical condition: Mucopolysaccharidosis type II (MPS II)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056889 Mucopolysaccharidosis II PT
    Population Age: Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-001878-28 Sponsor Protocol Number: 307-201 Start Date*: 2019-12-17
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects...
    Medical condition: Phenylketonuria
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10034872 Phenylketonuria PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2016-000898-20 Sponsor Protocol Number: MGT004 Start Date*: 2016-10-04
    Sponsor Name:MeiraGTx UK Ltd
    Full Title: Long term follow-up study of participants following an open label, multi-centre, Phase I/II dose escalation trial of an adeno-associated virus vector (AAV2/5-OPTIRPE65) for gene therapy of adults a...
    Medical condition: Leber Congenital Amaurosis (LCA) caused by mutations in RPE65
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-003032-23 Sponsor Protocol Number: AADC-010 Start Date*: 2019-10-09
    Sponsor Name:National Taiwan University Hospital
    Full Title: A Phase I/II Clinical Trial for Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC
    Medical condition: Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2019-003072-39 Sponsor Protocol Number: NTUH-AADC-011 Start Date*: 2019-10-09
    Sponsor Name:National Taiwan University Hospital
    Full Title: A Clinical Trial for Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Using AAV2-hAADC - An Expansion
    Medical condition: Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    Disease:
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2005-005711-17 Sponsor Protocol Number: AGT4HB Start Date*: 2009-09-25
    Sponsor Name:St. Jude Children’s Research Hospital
    Full Title: AN OPEN LABEL DOSE-ESCALATION STUDY OF A SELF COMPLEMENTARY ADENO-ASSOCIATED VIRAL VECTOR (scAAV2/8-LP1-hFIXco) FOR GENE TRANSFER IN SUBJECTS WITH HEMOPHILIA B
    Medical condition: Gene therapy for Haemophilia B
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004865 10001140 Adeno-associated in vivo gene therapy LLT
    19.0 10010331 - Congenital, familial and genetic disorders 10061992 Haemophilia PT
    Population Age: Adults Gender: Male
    Trial protocol: GB (Temporarily Halted)
    Trial results: (No results available)
    EudraCT Number: 2017-001271-23 Sponsor Protocol Number: C0371004 Start Date*: 2019-02-20
    Sponsor Name:Pfizer Inc.
    Full Title: AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, MULTI-CENTER, LEAD-IN STUDY TO EVALUATE PROSPECTIVE EFFICACY AND SELECTED SAFETY DATA OF CURRENT FACTOR IX (FIX) OR FACTOR VIII (FVIII) PROPHYLAXIS REPLA...
    Medical condition: Moderately Severe To Severe Adult Hemophilia B Participants (FIX:C≤2%) Who Are Negative For Neutralizing Antibodies (NAb) To Adeno-Associated Virus Vector (AAV) Spark100. Moderately severe to sever...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    20.0 100000004850 10060613 Hemophilia A (Factor VIII) LLT
    Population Age: Adults Gender: Male
    Trial protocol: SE (Completed) GB (GB - no longer in EU/EEA) IE (Completed) DE (Completed) FR (Ongoing) ES (Ongoing) NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-004276-33 Sponsor Protocol Number: ATA-001-FKRP Start Date*: 2022-02-11
    Sponsor Name:Atamyo Therapeutics
    Full Title: A phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle ...
    Medical condition: FKRP-related limb-girdle muscular dystrophy (LGMD R9)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10028356 Muscular dystrophy PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DK (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-000156-18 Sponsor Protocol Number: 301OTC02 Start Date*: 2018-08-15
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Lat...
    Medical condition: Ornithine transcarbamylase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10071107 Ornithine transcarbamylase deficiency LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Ongoing) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-003430-25 Sponsor Protocol Number: 101HEMB02 Start Date*: 2017-05-18
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Sever...
    Medical condition: Moderate/severe to severe hemophilia B
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2006-001571-37 Sponsor Protocol Number: AAV2/2-hRPE65p-hRPE65 Start Date*: 2006-12-14
    Sponsor Name:University College London
    Full Title: AN OPEN-LABEL DOSE ESCALATION STUDY OF AN ADENO-ASSOCIATED VIRUS VECTOR (AAV2/2-hRPE65p-hRPE65) FOR GENE THERAPY OF SEVERE EARLY-ONSET RETINAL DEGENERATION
    Medical condition: The condition to be investigated is severe early-onset inherited retinal degeneration due to defects in the gene encoding RPE65
    Disease: Version SOC Term Classification Code Term Level
    13.1 10015919 - Eye disorders 10038845 Retinal degeneration PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2016-003856-59 Sponsor Protocol Number: MGT007 Start Date*: 2017-04-12
    Sponsor Name:MeiraGTX UK II Ltd
    Full Title: Long-term follow-up study of participants following an open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/8-hCARp.hCNGB3) for gene thera...
    Medical condition: Achromatopsia caused by mutations in the CNGB3 gene
    Disease: Version SOC Term Classification Code Term Level
    19.1 100000004850 10000454 Achromatopsia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2018-003431-29 Sponsor Protocol Number: MGT012 Start Date*: 2019-06-24
    Sponsor Name:MeiraGTx UK II Limited
    Full Title: An open label, multi-centre, Phase I/II dose escalation trial of an adeno-associated virus vector (AAV2/8-hG1.7p.coCNGA3) for gene therapy of children with achromatopsia owing to defects in CNGA3
    Medical condition: Achromatopsia caused by mutations in the CNGA3 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000454 Achromatopsia LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2016-002290-35 Sponsor Protocol Number: MGT006 Start Date*: 2016-12-20
    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 (AAV2/8-hCARp.hCNGB3) for gene therapy of adults and children with achromatopsia owing t...
    Medical condition: Achromatopsia caused by mutations in the CNGB3 gene
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10000454 Achromatopsia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2020-005266-34 Sponsor Protocol Number: UX701-CL301 Start Date*: 2022-02-11
    Sponsor Name:Ultragenyx Pharmaceutical Inc.
    Full Title: A Randomized, Double-blind, Placebo-controlled, Multicenter, Seamless, Adaptive, Safety, Dose-finding, and Phase 3 Clinical Study of UX701 AAV-mediated Gene Transfer for the Treatment of Wilson Dis...
    Medical condition: Wilson disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10047988 Wilson's disease LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Ongoing) PT (Ongoing)
    Trial results: (No results available)
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