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Virus Therapy

RP-L401 for Malignant Osteopetrosis

Phase 1
Waitlist Available
Led By Donald B Kohn, MD
Research Sponsored by Rocket Pharmaceuticals Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights

Study Summary

This study is evaluating whether a new drug may be safe for use in humans.

Eligible Conditions
  • Malignant Osteopetrosis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with treatment-related adverse events as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0
Secondary outcome measures
Assessment of auditory status after infusion of RP-L401
Assessment of blood counts after infusion of RP-L401
Assessment of bone abnormalities after infusion of RP-L401
+5 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Experimental - RP-L401Experimental Treatment1 Intervention
RP-L401 is a gene therapy product containing autologous genetically modified CD34+ hematopoietic cells transduced with lentiviral vector carrying the TCIRG1 transgene

Find a Location

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor
14 Previous Clinical Trials
379 Total Patients Enrolled
California Institute for Regenerative Medicine (CIRM)OTHER
63 Previous Clinical Trials
3,168 Total Patients Enrolled
Donald B Kohn, MDPrincipal InvestigatorUniversity of California, Los Angeles
2 Previous Clinical Trials
55 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
2020. "A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04525352.
~0 spots leftby Apr 2025
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