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Gene Therapy

Gene Therapy for Fanconi Anemia

Phase 2
Waitlist Available
Led By Rajni Agarwal, 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 3 years
Awards & highlights
No Placebo-Only Group

Summary

This trial will test whether gene therapy can help patients with Fanconi anemia, subtype A by correcting their hematopoietic stem cells ex vivo and then infusing them back into the patient.

Who is the study for?
This trial is for patients with Fanconi anemia subtype A, confirmed by a specific blood test. Participants must be at least 1 year old and weigh over 8 kg, have certain levels of blood cells, and women who can bear children must use effective contraception. People with stable or improving blood counts due to mosaicism, other serious health issues, pregnant or breastfeeding women, those with suitable sibling donors or current cancer are excluded.
What is being tested?
The study tests RP-L102 gene therapy in Fanconi anemia subtype A patients. It involves taking the patient's own stem cells from their blood, adding a correct version of the FANCA gene outside the body using a virus as a carrier, then infusing these corrected cells back into the patient to help prevent bone marrow failure.
What are the potential side effects?
Potential side effects aren't specified here but may include reactions related to stem cell infusion such as fever and chills; immune responses against modified cells; discomfort at injection sites; and risks associated with lentiviral vectors like insertional mutagenesis.

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
Bone Marrow (BM) Colony-Forming Cell (CFC) Mitomycin-C (MMC) resistance
Secondary study objectives
Bone Marrow Failure Free Survival
Genetic Correction
Hematologic Stability- Hemoglobin
+7 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 CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with a lentiviral vector carrying the FANCA gene

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Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor
16 Previous Clinical Trials
421 Total Patients Enrolled
3 Trials studying Fanconi Anemia
18 Patients Enrolled for Fanconi Anemia
Rajni Agarwal, MDPrincipal InvestigatorStanford University
4 Previous Clinical Trials
50 Total Patients Enrolled
1 Trials studying Fanconi Anemia
12 Patients Enrolled for Fanconi Anemia
Agnieszka Czechowicz, MDPrincipal InvestigatorStanford University
1 Previous Clinical Trials
2 Total Patients Enrolled
1 Trials studying Fanconi Anemia
2 Patients Enrolled for Fanconi Anemia
~1 spots leftby May 2026
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