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Clinical trials for VG

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   44341   clinical trials with a EudraCT protocol, of which   7370   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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    89 result(s) found for: VG. Displaying page 4 of 5.
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    EudraCT Number: 2017-000662-29 Sponsor Protocol Number: 270-203 Start Date*: 2018-02-01
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels ≤ 1...
    Medical condition: Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed)
    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) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-003474-31 Sponsor Protocol Number: COAV101B12301 Start Date*: 2023-06-01
    Sponsor Name:Novartis Pharma AG
    Full Title: A randomized, sham-controlled, double-blind study to evaluate the efficacy and safety of intrathecal (IT) OAV101 in patients with later onset Type 2 spinal muscular atrophy (SMA) who are ≥ 2 to < 1...
    Medical condition: Spinal Muscular Atrophy
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10041582 Spinal muscular atrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DK (Completed) GR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-000266-29 Sponsor Protocol Number: AVXS-101-CL-302 Start Date*: 2018-06-12
    Sponsor Name:AveXis, Inc.
    Full Title: Phase 3, Open Label, Single Arm, Single Dose Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1 with One or Two SMN2 Copies Delivering AVXS 101 by Intravenous ...
    Medical condition: Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    Disease:
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) SE (Prematurely Ended) BE (Completed) ES (Prematurely Ended) NL (Prematurely Ended) IT (Completed)
    Trial results: View results
    EudraCT Number: 2014-001411-39 Sponsor Protocol Number: ABT-002 Start Date*: 2018-09-04
    Sponsor Name:Abeona Therapeutics Inc
    Full Title: Phase I/II gene transfer clinical trial of rAAV9.CMV.hNAGLU for Mucopolysaccharidosis (MPS) IIIB
    Medical condition: MPS IIIB is a devastating lysosomal storage disease, caused by a N-α-acetylglucosaminidase (NAGLU) gene defect. Infants with MPS IIIB appear normal at birth, but the disease is relentlessly progre...
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) DE (Prematurely Ended) FR (Prematurely Ended) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2017-004087-35 Sponsor Protocol Number: AVXS-101-CL-304 Start Date*: 2018-12-21
    Sponsor Name:AveXis, Inc.
    Full Title: A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2
    Medical condition: Spinal Muscular Atrophy
    Disease:
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: ES (Prematurely Ended) IT (Completed) GB (GB - no longer in EU/EEA) BE (Completed) NL (Prematurely Ended)
    Trial results: View results
    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: 2017-001452-55 Sponsor Protocol Number: 204852 Start Date*: 2018-11-26
    Sponsor Name:GlaxoSmithKline Biologicals
    Full Title: A first-time-in human (FTIH), Phase I/II, randomized, multi-centric, single-blind, controlled dose-escalation study to evaluate the reactogenicity, safety, immunogenicity and efficacy of GSK Biolog...
    Medical condition: Chronic hepatitis B
    Disease: Version SOC Term Classification Code Term Level
    20.1 10021881 - Infections and infestations 10008910 Chronic hepatitis B PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-001283-30 Sponsor Protocol Number: SPK-3006-101 Start Date*: 2020-06-16
    Sponsor Name:Spark Therapeutics
    Full Title: Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease
    Medical condition: Pompe Disease (also known as glycogen storage disease type II)
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10053185 Glycogen storage disease type II PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Prematurely Ended) DE (Trial now transitioned) NL (Ongoing) DK (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2019-003213-34 Sponsor Protocol Number: 270-205 Start Date*: 2020-09-25
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Active or Prior Inhibitors
    Medical condition: Hemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060612 Hemophilia A LLT
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Prematurely Ended) DE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2019-003159-12 Sponsor Protocol Number: J4B-MC-OKAA Start Date*: 2021-12-24
    Sponsor Name:Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company
    Full Title: A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM)
    Medical condition: Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN)
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10068968 Frontotemporal dementia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) ES (Temporarily Halted) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-002443-38 Sponsor Protocol Number: C0371006 Start Date*: Information not available in EudraCT
    Sponsor Name:Pfizer Inc., 235 East 42nd Street, New York, NY 10017
    Full Title: Phase 3 open-label study to evaluate efficacy, safety, and tolerability of FIX gene transfer with fidanacogene elaparvovec (PF-06838435) in pediatric male participants <18 years of age with moderat...
    Medical condition: moderately severe to severe hemophilia B (FIX:C≤2%)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Ongoing) IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2022-002568-62 Sponsor Protocol Number: AVB-PGRN-001 Start Date*: 2023-03-13
    Sponsor Name:AviadoBio Ltd
    Full Title: A Phase 1/2 Open-Label, Ascending Dose, Multicenter Study to Evaluate the Safety and Preliminary Efficacy of AVB-101 Administered by Bilateral Intrathalamic Infusion in Subjects With Frontotemporal...
    Medical condition: Frontotemporal Dementia With Progranulin Mutations (FTD-GRN)
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: FR (Ongoing) IT (Trial now transitioned) ES (Ongoing) SE (Trial now transitioned) BE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-000195-15 Sponsor Protocol Number: P4-SAF-302 Start Date*: 2018-12-27
    Sponsor Name:Lysogene SA
    Full Title: An Open-Label, Single-Arm, Multicenter Study of Intracerebral Administration of Adeno-Associated Viral Vectors Serotype rh10 Carrying the Human N-sulfoglucosamine sulfohydrolase (SGSH) cDNA for the...
    Medical condition: Mucopolysaccharidosis Type IIIA
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10056890 Mucopolysaccharidosis III PT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) GB (GB - no longer in EU/EEA) NL (Temporarily Halted) DE (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2019-000667-24 Sponsor Protocol Number: ST-920-201 Start Date*: 2019-11-12
    Sponsor Name:Sangamo Therapeutics, Inc.
    Full Title: A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Dise...
    Medical condition: Fabry Disease (X-linked lysosomal storage disease)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10016016 Fabry's disease PT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Trial now transitioned) IT (Trial now transitioned)
    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: 2017-002187-40 Sponsor Protocol Number: GS-LHON-CLIN-05 Start Date*: 2018-03-15
    Sponsor Name:GENSIGHT BIOLOGICS
    Full Title: Efficacy and Safety of Bilateral Intravitreal Injection of GS010: A Randomized, Double-Masked, Placebo-Controlled Trial in Subjects Affected with G11778A ND4 Leber Hereditary Optic Neuropathy for U...
    Medical condition: Leber Hereditary Optic Neuropathy due to mutations in the mitochondrial NADH Dehydrogenase 4 gene
    Disease:
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) FR (Completed) GB (GB - no longer in EU/EEA) ES (Ongoing) NL (Ongoing) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-003086-33 Sponsor Protocol Number: C0371002 Start Date*: 2020-01-28
    Sponsor Name:Pfizer Inc., 235 East 42nd Street, New York, NY 10017
    Full Title: Phase 3, open label, single arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-Padua) in adult male participants with moderately severe to severe he...
    Medical condition: severe to moderately severe hemophilia B <=2%
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060614 Hemophilia B (Factor IX) LLT
    Population Age: Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned) DE (Trial now transitioned) GR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2020-002431-30 Sponsor Protocol Number: GT005-03 Start Date*: 2020-10-21
    Sponsor Name:Gyroscope Therapeutics
    Full Title: HORIZON: A Phase II, open-label, outcomes-assessor masked, multicentre, randomised,controlled study to evaluate the safety and efficacy of two doses of GT005 administered as a single subretinal inj...
    Medical condition: Age-related Macular Degeneration (AMD) presents as a progressive loss of vision in the centre of the retina (the macula) resulting in a blurred area or blank spot in the centre of vision. The prima...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004853 10075719 Atrophic age-related macular degeneration LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) FR (Completed) IE (Completed)
    Trial results: View results
    EudraCT Number: 2019-004451-37 Sponsor Protocol Number: C3731003 Start Date*: 2021-09-24
    Sponsor Name:Pfizer Inc., 235 East 42nd Street, New York, NY 10017
    Full Title: Phase 3, Open-Label, Single-Arm Study to Evaluate the Efficacy and Safety of PF-07055480 (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in Adult Male Participants with Moderately Severe to Sev...
    Medical condition: hemophilia A
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10060613 Hemophilia A (Factor VIII) LLT
    Population Age: Adults Gender: Male
    Trial protocol: DE (Trial now transitioned) FR (Trial now transitioned) IT (Completed) ES (Restarted)
    Trial results: (No results available)
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