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Methodology study of retroviral insertion site analysis in Strimvelis-treated ADA-SCID patients
2017-001731-39Phase 4Terminated
- Sponsor
- Orchard Therapeutics
- Start
- Jul 2020
- Modality
- Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
01
Overview
Phase 4, open-label, single-arm trial of Strimvelis in adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID) and adenosine deaminase deficiency, sponsored by Orchard Therapeutics. Primary endpoint: Accuracy and precision of SLiM-PCR for retroviral insertion site analysis. Started Jul 2020; status: terminated.
02
Drugs
Strimvelis
03
Conditions
adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID)adenosine deaminase deficiency
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