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Not listed Phase 1 Interventional

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

ClinicalTrials.gov ID: NCT03814408

Public ClinicalTrials.gov record NCT03814408. 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.

ClinicalTrials.gov public records Last synced Sep 2, 2026, 12:46 AM EDT

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 I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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).

Study identification

NCT ID
NCT03814408
Recruitment status
Not listed
Study type
Interventional
Phase
Phase 1
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Enrollment
2 participants

Conditions and interventions

Interventions

Biological

Eligibility (public fields only)

Age range
1 Year to 12 Years
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
Jan 10, 2019
Primary completion
Nov 30, 2020
Completion
Feb 28, 2022
Last update posted
Nov 23, 2020

2019 – 2022

United States locations

U.S. sites
1
U.S. states
1
U.S. cities
1
Facility City State ZIP Site status
Stanford University Institute for Stem Cell Biology and Regenerative Medicine Lucille Packard Children's Hospital, Stanford University Stanford California 94304

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 NCT03814408, 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 Nov 23, 2020 · Synced Sep 2, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT03814408 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →