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Clinical trials for Insertional mutagenesis

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   44335   clinical trials with a EudraCT protocol, of which   7366   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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    13 result(s) found for: Insertional mutagenesis. Displaying page 1 of 1.
    EudraCT Number: 2016-004867-38 Sponsor Protocol Number: AUTO-LT1 Start Date*: 2018-05-18
    Sponsor Name:Autolus Limited
    Full Title: Long-term follow-up of patients previously treated with autologous T cells genetically modified with viral vectors.
    Medical condition: Potential malignancy in patients who received treatment with Autolus' CAR T cell therapy.
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) NL (Completed)
    Trial results: (No results available)
    EudraCT Number: 2019-004266-18 Sponsor Protocol Number: LTF-307 Start Date*: 2020-09-03
    Sponsor Name:bluebird bio, Inc.
    Full Title: Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector
    Medical condition: Subjects with sickle-cell disease treated with gene therapy drug products in a bluebird bio-sponsored study will be invited to participate in this long-term follow-up study to monitor the safety an...
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10040648 Sickle cell SC disease LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-002343-14 Sponsor Protocol Number: RAG1-2019-01 Start Date*: 2020-09-25
    Sponsor Name:Leiden University Medical Center
    Full Title: PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY
    Medical condition: Patients with severe combined immunodeficiency (SCID) based on a genetic defect in the Recombinase Activating Gene 1 (RAG1)
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) ES (Ongoing) PL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-001145-14 Sponsor Protocol Number: ALD-104 Start Date*: 2019-04-17
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects ≤17 Years of Age With Cerebral Adrenoleukodystrophy (CALD)
    Medical condition: Cerebral Adrenoleukodystrophy (CALD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10051260 Adrenoleukodystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Completed) DE (Completed) NL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2020-000517-33 Sponsor Protocol Number: RP-L201-0318 Start Date*: 2020-07-14
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I):A Phase I/II Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Len...
    Medical condition: Leukocyte Adhesion Deficiency-I (LAD-I)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018137 Genetic anomalies of leukocytes LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2019-001656-19 Sponsor Protocol Number: RP-L301-0119 Start Date*: 2019-09-19
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon O...
    Medical condition: Pyruvate kinase deficiency
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10037682 Pyruvate kinase deficiency anaemia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-002680-26 Sponsor Protocol Number: RP-L201-0218 Start Date*: 2018-11-27
    Sponsor Name:Rocket Pharmaceuticals, Inc.
    Full Title: Gene Therapy for Leukocyte Adhesion Deficiency-I (LAD-I): A Phase I Clinical Trial to Evaluate the Safety and Efficacy of the Infusion of Autologous Hematopoietic Stem Cells Transduced with a Lenti...
    Medical condition: Leukocyte Adhesion Deficiency-I (LAD-I)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10018137 Genetic anomalies of leukocytes LLT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: ES (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2020-005843-21 Sponsor Protocol Number: KT-US-982-5968 Start Date*: 2022-02-08
    Sponsor Name:KITE PHARMA INC.
    Full Title: Long-term Follow-up Study for Participants of Kite Sponsored Interventional Studies Treated With Gene-Modified Cells
    Medical condition: B-cell malignancies and Acute Myeloid Leukemia
    Disease: Version SOC Term Classification Code Term Level
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10026801 Mantle cell lymphoma refractory PT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008977 Chronic lymphocytic leukemia recurrent LLT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10008978 Chronic lymphocytic leukemia refractory LLT
    23.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10065856 Non-Hodgkin's lymphoma unspecified histology indolent PT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10012822 Diffuse large B-cell lymphoma refractory PT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10060558 Acute myeloid leukemia recurrent LLT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10081514 Acute myeloid leukemia refractory LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10063621 Acute lymphoblastic leukaemia recurrent LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10006599 Burkitt's lymphoma refractory PT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10036714 Primary mediastinal large B-cell lymphoma refractory PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) FR (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-004122-33 Sponsor Protocol Number: HGB-207 Start Date*: 2016-11-10
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, who do not have β0/β0 Genotype, by Transplantation of Autologous ...
    Medical condition: This study will enroll subjects with transfusion dependent beta-thalassemia, who do not have beta0/beta0 genotype, defined by a history of at least 100 mL/kg/year of packed red blood cells (pRBCs) ...
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10054660 Thalassemia beta LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed) DE (Completed) GR (Completed) GB (GB - no longer in EU/EEA) FR (Completed)
    Trial results: View results
    EudraCT Number: 2012-000695-42 Sponsor Protocol Number: HGB-205 Start Date*: 2012-12-31
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase I/II Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the β-Hemoglobinopathies (Sickle Cell Anemia and β-Thalassemia Major) by Transplantation of Autologous CD34+ Stem...
    Medical condition: Severe sickle cell anemia and transfusion-dependent beta-thalassemia major. This study will enroll patients who are eligible for an allogeneic hematopoietic stem cell transplant (HSCT) but do not...
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10055579 Sickle-cell beta thalassemia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2016-003611-35 Sponsor Protocol Number: HGB-212 Start Date*: 2017-07-24
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex V...
    Medical condition: This study will enroll subjects with transfusion dependent beta-thalassemia, defined by a history of at least 100 mL/kg/year of packed red blood cells (pRBCs) or ≥ 8 transfusions of pRBCs per year...
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054660 Thalassemia beta LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) GR (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-002805-13 Sponsor Protocol Number: LTF-304 Start Date*: 2015-11-27
    Sponsor Name:bluebird bio, Inc
    Full Title: Longterm Follow-up of Subjects With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product
    Medical condition: Cerebral Adrenoleukodystrophy (CALD)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10051260 Adrenoleukodystrophy PT
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-002245-11 Sponsor Protocol Number: LTF-303 Start Date*: 2013-11-12
    Sponsor Name:bluebird bio, Inc.
    Full Title: Long-term Follow-up of Subjects Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector
    Medical condition: Subjects treated with gene therapy drug products in a bluebird bio-sponsored or associated clinical study will be invited to participate in this long-term follow-up study to monitor the safety and ...
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10055579 Sickle-cell beta thalassemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) DE (Trial now transitioned) GB (GB - no longer in EU/EEA) GR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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