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Gene Therapy

RP-L201 for Leukocyte Adhesion Defect

Phase 1 & 2

Waitlist Available

Led By Claire Booth

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 trial is testing a new gene therapy for a blood disorder called X-linked chronic granulomatous disease (X-CGD). The first part of the trial is to see if the therapy is safe. The second part of the trial is to see if the therapy improves survival without the need for a stem cell transplant.

Eligible Conditions

Leukocyte Adhesion Defect Type I

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 2 years

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

Phase I: Number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v.5.0

Phase II: Number of participants with treatment-related adverse events as assessed by CTCAE v.5.0

Phase II: Survival following infusion of RP-L201

Secondary outcome measures

Assessment of LAD-I-associated leukocytosis after infusion of RP-L201

Assessment of LAD-I-associated neutrophilia after infusion of RP-L201

Assessment of overall survival after infusion of RP-L201

+4 more

Trial Design

1Treatment groups

Experimental Treatment

Group I: RP-L201Experimental Treatment1 Intervention

RP-L201 is a gene therapy product containing autologous genetically modified CD34+ hematopoietic cells transduced with Chim-CD18-WPRE lentiviral vector administered as a single intravenous infusion

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

RP-L201

2019

Completed Phase 2

~10

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Find a Location

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor

14 Previous Clinical Trials

371 Total Patients Enrolled

California Institute for Regenerative Medicine (CIRM)OTHER

63 Previous Clinical Trials

3,160 Total Patients Enrolled

Claire BoothPrincipal InvestigatorUniversity College London Great Ormond Street Institute of Child Helath

1 Previous Clinical Trials

5 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the goal of this experiment?

"As reported by Rocket Pharmaceuticals Inc., the primary outcome measured over a 2 year period focuses on treatment-related adverse events, as assessed via CTCAE v.5.0. Secondary outcomes include overall survival beyond 24 months and gene correction with at least 10% of peripheral blood neutrophils carrying the therapeutic provirus 6 month post-infusion, as well as changes to partially normal or normal levels of LAD-I associated neutrophilia."

Answered by AI

Are investigators currently seeking new participants for this research endeavor?

"As detailed on clinicaltrials.gov, this medical trial is no longer recruiting patients. Initially posted in August 2019 and last updated November 2021, the study has ceased its search for participants; however, a total of 23 other trials are still open to prospective volunteers."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I

2019. "A Clinical Trial to Evaluate the Safety and Efficacy of RP-L201 in Subjects With Leukocyte Adhesion Deficiency-I". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03812263.