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RP-L401 for Infantile Malignant Osteopetrosis
NCT04525352Phase 1Terminated
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Nov 2020
- Primary completion
- May 2021
- Modality
- Gene Editing, Gene Silencing, Hematopoietic Stem Cells
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of RP-L401 in malignant infantile osteopetrosis (MIOP), sponsored by Rocket Pharmaceuticals, Inc. Primary endpoint: Number of participants with treatment-related adverse events as assessed by NCI CTCAE v5.0 at 2 years. Started Nov 2020; status: terminated.
02
Drugs
RP-L401
03
Conditions
malignant infantile osteopetrosis (MIOP)
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