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Clinical trials for Gene Drive

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   43881   clinical trials with a EudraCT protocol, of which   7295   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    169 result(s) found for: Gene Drive. Displaying page 1 of 9.
    1  2  3  4  5  6  7  8  9  Next»
    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: 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: 2016-003430-25 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: 2018-002502-31 Sponsor Protocol Number: RP-L102-0118 Start Date*: 2018-10-31
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemi...
    Medical condition: Fanconi anemia (subtype A)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10055206 Fanconi's anemia LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2016-003491-41 Sponsor Protocol Number: AG-CLI-0206-LTFU Start Date*: 2016-12-12
    Sponsor Name:AnGes Inc.
    Full Title: A Long Term Follow-up Study of AMG0001 in Subjects with Critical Limb Ischemia
    Medical condition: Critical limb ischemia
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004866 10058069 Critical limb ischemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: HU (Completed) BE (Ongoing) FR (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2009-012595-27 Sponsor Protocol Number: A8081007 Start Date*: 2010-01-20
    Sponsor Name:Pfizer Inc. 235 East 42nd Street, New York, NY 10017
    Full Title: PHASE 3, RANDOMIZED, OPEN-LABEL STUDY OF THE EFFICACY AND SAFETY OF PF 02341066 VERSUS STANDARD OF CARE CHEMOTHERAPY (PEMETREXED OR DOCETAXEL) IN PATIENTS WITH ADVANCED NON-SMALL CELL LUNG CANCER (...
    Medical condition: ADVANCED NON-SMALL CELL LUNG CANCER (NSCLC) HARBORING A TRANSLOCATION OR INVERSION EVENT INVOLVING THE ANAPLASTIC LYMPHOMA KINASE (ALK) GENE LOCUS
    Disease: Version SOC Term Classification Code Term Level
    17.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) NL (Completed) DE (Completed) PL (Completed) ES (Completed) HU (Completed) FR (Completed) GR (Completed) IT (Completed) IE (Completed) BG (Completed) SE (Completed)
    Trial results: View results
    EudraCT Number: 2005-003098-26 Sponsor Protocol Number: 3144A1-201-WW Start Date*: 2006-06-06
    Sponsor Name:Wyeth Research Division of Wyeth Pharmaceuticals Inc.
    Full Title: Phase 2 Study of HKI-272 in Subjects with Advanced Breast Cancer
    Medical condition: Breast cancer is the most frequently diagnosed malignancy and the second most common cause of cancer related deaths in women. HER2 is a member of the epidermal growth factor receptor (EGFR) family ...
    Disease:
    Population Age: Adults, Elderly Gender: Female
    Trial protocol: BE (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2010-022978-14 Sponsor Protocol Number: A8081013 Start Date*: 2011-02-10
    Sponsor Name:Pfizer Inc, 235 East 42nd Street, New York, NY 10017
    Full Title: PHASE 1B OPEN-LABEL STUDY OF THE SAFETY AND CLINICAL ACTIVITY OF CRIZOTINIB (PF-02341066) IN TUMORS WITH GENETIC EVENTS INVOLVING THE ANAPLASTIC LYMPHOMA KINASE (ALK ) GENE LOCUS
    Medical condition: Treatment of tumors with translocation, mutation, or amplification of the anaplastic lymphoma kinase (ALK)gene locus.
    Disease: Version SOC Term Classification Code Term Level
    13.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065252 Solid tumor LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2009-012504-13 Sponsor Protocol Number: A8081005 Start Date*: 2010-01-20
    Sponsor Name:Pfizer Inc 235 East 42nd Street, New York, NY10017
    Full Title: PHASE 2, OPEN-LABEL SINGLE ARM STUDY OF THE EFFICACY AND SAFETY OF PF 02341066 IN PATIENTS WITH ADVANCED NON-SMALL CELL LUNG CANCER (NSCLC) HARBORING A TRANSLOCATION OR INVERSION INVOLVING THE ANAP...
    Medical condition: metastatic non-small cell lung cancer
    Disease: Version SOC Term Classification Code Term Level
    15.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) NL (Completed) DE (Completed) ES (Completed) HU (Completed) PL (Completed) GR (Completed) IT (Completed) FR (Completed) IE (Completed) BG (Completed) SE (Completed)
    Trial results: View results
    EudraCT Number: 2016-003023-30 Sponsor Protocol Number: 401GSDIA01 Start Date*: 2018-11-27
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Dise...
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2022-001596-14 Sponsor Protocol Number: TYR300-101 Start Date*: 2023-06-26
    Sponsor Name:Tyra Biosciences, Inc.
    Full Title: A Multicenter, Open-label Phase 1/2 Study of TYRA 300 in Advanced Urothelial Carcinoma and Other Solid Tumors with Activating FGFR3 Gene Alterations (SURF 301)
    Medical condition: Advanced Urothelial Carcinoma and Other Solid Tumors with Activating FGFR3 Gene Alterations
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004864 10049280 Solid tumour LLT
    21.1 100000004864 10065143 Malignant solid tumour LLT
    21.1 100000004864 10065147 Malignant solid tumor LLT
    21.1 100000004864 10065252 Solid tumor LLT
    20.0 100000004864 10064467 Urothelial carcinoma LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-003603-18 Sponsor Protocol Number: 331-201 Start Date*: 2022-10-06
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 1/2, Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of BMN 331, an Adeno-Associated Virus (AAV) Vector-Mediated Gene Transfer of Human SERPING1, in Su...
    Medical condition: Hereditary Angioedema (HAE)
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    21.0 10010331 - Congenital, familial and genetic disorders 10080957 Hereditary angioedema C1 inhibitor deficiency LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2016-001057-40 Sponsor Protocol Number: 301OTC01 Start Date*: 2017-03-14
    Sponsor Name:Ultragenyx Pharmaceutical, Inc.
    Full Title: A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OT...
    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)
    Trial results: View results
    EudraCT Number: 2015-004486-86 Sponsor Protocol Number: SRA737-01 Start Date*: 2016-04-25
    Sponsor Name:Sierra Oncology, Inc.
    Full Title: A Phase 1/2 Trial of SRA737 (a Chk1 Inhibitor) Administered Orally in Subjects with Advanced Cancer
    Medical condition: Patients with histologically or cytologically proven advanced solid tumours, refractory to conventional treatment, or for which no conventional therapy is considered appropriate by the Investigator.
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004864 10007050 Cancer LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2016-001955-29 Sponsor Protocol Number: 024PKAN15004 Start Date*: Information not available in EudraCT
    Sponsor Name:Retrophin, Inc.
    Full Title: Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, ...
    Medical condition: Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive,...
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029205 - Nervous system disorders 10053643 Neurodegenerative disorder PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) ES (Prematurely Ended) GB (Prematurely Ended) CZ (Prematurely Ended) NO (Completed) FR (Completed) PL (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-004370-34 Sponsor Protocol Number: POM-003 Start Date*: 2018-04-09
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy
    Medical condition: Late-onset Pompe disease (LOPD) in subjects receiving standard-of-care enzyme replacement therapy (ERT)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10075702 Pompe's disease late onset LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Prematurely Ended) HU (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-004473-27 Sponsor Protocol Number: 401GSDIA02 Start Date*: 2021-08-25
    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 Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen St...
    Medical condition: Glycogen Storage Disease Type Ia (GSDIa).
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10056911 Glycogen storage disease type IA LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-021336-33 Sponsor Protocol Number: A8081014 Start Date*: 2010-12-30
    Sponsor Name:Pfizer Inc 235 East 42nd Street, New York, NY10017 US
    Full Title: PHASE 3, RANDOMIZED, OPEN-LABEL STUDY OF THE EFFICACY AND SAFETY OF CRIZOTINIB VERSUS PEMETREXED/CISPLATIN OR PEMETREXED/CARBOPLATIN IN PREVIOUSLY UNTREATED PATIENTS WITH NON-SQUAMOUS CARCINOMA OF ...
    Medical condition: non-small cell lung cancer (NSCLC)
    Disease: Version SOC Term Classification Code Term Level
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10029519 Non-small cell lung cancer stage III PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) ES (Completed) FI (Completed) IE (Completed) AT (Completed) IT (Completed) GB (Completed) BE (Completed) NL (Completed) DK (Prematurely Ended) PT (Completed) NO (Completed)
    Trial results: View results
    EudraCT Number: 2017-003573-34 Sponsor Protocol Number: 270-302 Start Date*: 2018-02-23
    Sponsor Name:BioMarin Pharmaceutical Inc.
    Full Title: A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII at a dose of 4E13 vg/kg in Hemoph...
    Medical condition: Haemophilia A
    Disease:
    Population Age: Adults, Elderly Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) ES (Prematurely Ended) FR (Completed) BE (Completed) IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2007-003359-35 Sponsor Protocol Number: GAU-CL-202 Start Date*: 2007-12-19
    Sponsor Name:Amicus Therapeutics, Inc.
    Full Title: A RANDOMIZED, OPEN-LABEL STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF AT2101 IN TREATMENT-NAIVE ADULT PATIENTS WITH TYPE 1 GAUCHER DISEASE
    Medical condition: Type 1 Gaucher Disease
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) DE (Completed)
    Trial results: View results
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