← Back to Search

Gene Therapy

RP-L102 for Fanconi Anemia

Phase 1
Waitlist Available
Led By Agnieszka Czechowicz, 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
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 years
Awards & highlights

Study Summary

This study is evaluating whether a gene therapy can be used to treat people with Fanconi anemia.

Eligible Conditions
  • Fanconi Anemia

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 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 CTCAE v.5.0
Secondary outcome measures
Engraftment of gene-corrected hematopoietic cells after infusion of RP-L102
Phenotypic correction of T-lymphocytes in peripheral blood after infusion of RP-L102
Phenotypic correction of hematopoietic cells in bone marrow after infusion of RP-L102
+1 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: RP-L102Experimental Treatment1 Intervention
RP-L102 is a self-inactivating lentiviral vector carrying the therapeutic FANCA gene

Find a Location

Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor
14 Previous Clinical Trials
378 Total Patients Enrolled
3 Trials studying Fanconi Anemia
21 Patients Enrolled for Fanconi Anemia
Agnieszka Czechowicz, MDPrincipal InvestigatorStanford University, Stem Cell Transplantation and Regenerative Medicine
1 Previous Clinical Trials
5 Total Patients Enrolled
1 Trials studying Fanconi Anemia
5 Patients Enrolled for Fanconi Anemia

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 Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
2019. "A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03814408.
All > Fanconi Anemia
~0 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top