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Clinical trial

A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene

NCT04525352 is registered in ClinicalTrials.gov with Rocket Pharmaceuticals Inc. as lead sponsor.

NCT NCT04525352
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene
Lead sponsor
Rocket Pharmaceuticals Inc.
Phase
Phase 1
Overall status
Terminated
Start date
November 19, 2020
Completion date
May 21, 2021
Enrollment
1 (actual)
Sites
1

Conditions

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

infantile malignant osteopetrosis