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

NCT04525352 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2022-07-13

No results posted yet for this study

Summary

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

Conditions

  • Infantile Malignant Osteopetrosis

Interventions

BIOLOGICAL

RP-L401

CD34+ enriched hematopoietic stem cells from pediatric subjects with infantile malignant osteopetrosis transduced ex vivo with lentiviral vector carrying the TCIRG1 transgene

Sponsors & Collaborators

  • California Institute for Regenerative Medicine (CIRM)

    collaborator OTHER

  • Rocket Pharmaceuticals Inc.

    lead INDUSTRY

Principal Investigators

  • Donald B Kohn, MD · University of California, Los Angeles

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Month
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-11-19
Primary Completion
2021-05-21
Completion
2021-05-21
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04525352 on ClinicalTrials.gov