- Trials with a EudraCT protocol (4)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
4 result(s) found for: Pulmonary sequestration.
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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... | |||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2019-004471-39 | Sponsor Protocol Number: IMR-SCD-301 | Start Date*: 2020-06-24 | |||||||||||
Sponsor Name:IMARA, Inc. | |||||||||||||
Full Title: A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease | |||||||||||||
Medical condition: Sickle Cell Disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) NL (Prematurely Ended) GR (Prematurely Ended) IT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2018-001320-19 | Sponsor Protocol Number: CTX001-121 | Start Date*: 2018-12-05 | |||||||||||
Sponsor Name:Vertex Pharmaceuticals Incorporated | |||||||||||||
Full Title: A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sick... | |||||||||||||
Medical condition: Severe Sickle Cell Disease (SCD) | |||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: BE (Trial now transitioned) GB (GB - no longer in EU/EEA) DE (Completed) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2021-006390-37 | Sponsor Protocol Number: VX21-CTX001-161 | Start Date*: Information not available in EudraCT | ||||||||||||||||
Sponsor Name:Vertex Pharmaceuticals Incorporated | ||||||||||||||||||
Full Title: A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Transfusion-Depend... | ||||||||||||||||||
Medical condition: Transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD) | ||||||||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: DE (Trial now transitioned) IT (Trial now transitioned) | ||||||||||||||||||
Trial results: (No results available) |
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