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Gene Therapy

A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

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
No Placebo-Only Group

Summary

The objective of this study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in subjects with Fanconi anemia subtype A (FA-A).

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

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of participants with treatment-related adverse events as assessed by CTCAE v.5.0
Secondary study objectives
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

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
16 Previous Clinical Trials
424 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
~0 spots leftby Dec 2025
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