drugset
drugset / Trial / NCT04525352

A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis

NCT04525352 ↗

Phase 1 Terminated 1 enrolled Rocket Pharmaceuticals Inc. California Institute for Regenerative Medicine (CIRM) · collab
NaSingle-groupOpen-labelTreatment

Summary

The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.

Timeline

Start
2020-11-19
Primary completion
2021-05-21
Completion
2021-05-21

Outcome

Outcome not reported

Stopped: “Study discontinued due to feasibility.”

Drugs

EvaluationDrugModalityDoseRoute
Subject RP-L401 Unknown — —

Indications

autosomal recessive osteopetrosis