Gene Therapy for Fanconi Anemia, Complementation Group A
Public ClinicalTrials.gov record NCT04248439. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A
Study identification
- NCT ID
- NCT04248439
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Enrollment
- 5 participants
Conditions and interventions
Conditions
Interventions
- RP-L102 Biological
Biological
Eligibility (public fields only)
- Age range
- 1 Year and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 14, 2020
- Primary completion
- May 4, 2026
- Completion
- May 4, 2026
- Last update posted
- Dec 21, 2025
2020 – 2026
United States locations
- U.S. sites
- 2
- U.S. states
- 2
- U.S. cities
- 2
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Stanford University | Stanford | California | 94304 | — |
| University of Minnesota | Minneapolis | Minnesota | 55454 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04248439, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 21, 2025 · Synced May 18, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04248439 live on ClinicalTrials.gov.