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Gene Therapy

RP-L102 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

Study 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.Check my eligibility

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.See study design

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ 3 years

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

Bone Marrow (BM) Colony-Forming Cell (CFC) Mitomycin-C (MMC) resistance

Secondary outcome measures

Bone Marrow Failure Free Survival

Genetic Correction

Hematologic Stability- Hemoglobin

+7 more

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

Do I qualify?Apply below to find out

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Who is running the clinical trial?

Rocket Pharmaceuticals Inc.Lead Sponsor

14 Previous Clinical Trials

375 Total Patients Enrolled

3 Trials studying Fanconi Anemia

18 Patients Enrolled for Fanconi Anemia

Rajni Agarwal, MDPrincipal InvestigatorStanford University

4 Previous Clinical Trials

75 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

Frequently Asked Questions

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

Has RP-L102 received regulatory clearance from the Food and Drug Administration?

"RP-L102's safety is evaluated to be a 2 on our team at Power's scale, as this Phase 2 trial has some backing evidence regarding its security but none verifying its efficacy."

Answered by AI

Is recruitment still ongoing for this research endeavor?

"Affirmative. According to clinicaltrials.gov, this experiment was first posted on July 15th 2020 and is currently enrolling participants. The research is requiring 5 subjects from a single medical centre."

Answered by AI

How many participants is this trial enrolling?

"Affirmative. According to clinicaltrials.gov, this study is enlisting participants as of now; it was initially posted on July 15th 2020 and recently updated on November 23rd 2020. The research team needs 5 volunteers from a single site for the trial's completion."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Gene Therapy for Fanconi Anemia, Complementation Group A

2020. "Gene Therapy for Fanconi Anemia, Complementation Group A". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04248439.

All > Fanconi Anemia