Found 17 clinical trials
Gene Therapy for Fanconi Anemia Complementation Group A
The objective of this study is to assess the therapeutic efficacy of a hematopoietic cell-based gene therapy for patients with Fanconi anemia, subtype A (FA-A). Hematopoietic stem cells from mobilized peripheral blood of patients with FA-A will be transduced ex vivo (outside the body) with a lentiviral vector carrying the …
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- 16 Feb, 2024
- 1 location
Gene Therapy for X Linked Severe Combined Immunodeficiency
A safety and efficacy clinical study of a lentiviral vector to transfer IL2RG complementary DNA to bone marrow stem cells in ten children with genetic diagnosed X-SCIDsevere combined immune deficiency .The ten children will be followed for 3-5 years and be evaluated by clinical characteristics, vector marking (vector copy number …
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- 16 Feb, 2024
- 1 location
AAV Gene Therapy Study for Subjects With PKU
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
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- 16 Feb, 2024
- 1 location
A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease
This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects 2 and 50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) with LentiGlobin BB305 Drug Product for SCD.
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- 16 Feb, 2024
- 2 locations
Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
This study will evaluate safety, tolerability, and preliminary efficacy of a single intravitreal (IVT) injection of a recombinant adeno-associated virus (AAV) gene therapy, 4D-110, in male patients with genetically-confirmed Choroideremia (CHM).
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- 05 Aug, 2020
Validation of Standardized Test Protocols to Assess the Impact of Visual Pathologies in Daily Life Activities
This tool can be used to measure a therapeutic benefit for new treatments (like gene therapy, retinal implants, intra vitreous injections ) for visual loss patients.
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- 05 Aug, 2020
Multi-4SCAR-T Therapy Targeting Breast Cancer
The purpose of this study is to assess the feasibility, safety and efficacy of multiple 4th generation CAR-T cells targeting Her2, GD2, and CD44v6 surface antigen in breast cancer. Another goal of the study is to learn more about the activities of the multi-CAR T cells and their persistency in …
- 2 views
- 05 Aug, 2020
Chimeric Antigen Receptor T-cells for The Treatment of AML Expressing CLL-1 Antigen
Patients eligible for this study have a type of blood cancer Acute Myeloid Leukemia (AML) which has come back or has not gone away after treatment. The body has different ways of fighting disease and infection, and this research study combines two different ways of fighting cancer with antibodies and …
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- 16 Feb, 2024
- 1 location
Heeba Test Trial
This study is looking for individuals 18 to 65 years old with PKU. Sanofi believes that everyoneshould have the opportunity to take part in clinical trials. It is important to include people whohave been historically under-represented in clinical trials. Sanofi is committed to inclusivity inour studies.If you are interested in …
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- 24 Feb, 2025
- 3 locations
Sujana-A Phase 1/Phase 2 open-label study to evaluate the safety, tolerability, and efficacy of a single intravenous administration of an investigational medication in adult participants with phenylketonuria (PKU)
This study is looking for individuals 18 to 65 years old with PKU. Sanofi believes that everyoneshould have the opportunity to take part in clinical trials. It is important to include people whohave been historically under-represented in clinical trials. Sanofi is committed to inclusivity inour studies.If you are interested in …
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- 13 Nov, 2023
- 1 location
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