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Clinical trial

Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem and Progenitor Cells Genetically Modified With IDUA Lentiviral Vector Encoding for the Human α-L-iduronidase Gene for the Treatment of Patients Affected by Mucopolysaccharidosis Type I, Hurler Variant

NCT03488394 is registered in ClinicalTrials.gov with Orchard Therapeutics as lead sponsor.

NCT NCT03488394
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Registry record

As filed in ClinicalTrials.gov.

Title
Phase I/II Study Evaluating Safety and Efficacy of Autologous Hematopoietic Stem and Progenitor Cells Genetically Modified With IDUA Lentiviral Vector Encoding for the Human α-L-iduronidase Gene for the Treatment of Patients Affected by Mucopolysaccharidosis Type I, Hurler Variant
Lead sponsor
Orchard Therapeutics
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
May 11, 2018
Completion date
March 31, 2035
Enrollment
8 (actual)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

mucopolysaccharidosis ih