Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102

April 7, 2026 updated by: Rocket Pharmaceuticals Inc.
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Detailed Description

Following the end of participation in Study(RP-L102-0418, RP-L102-0319, RP-L102-0118), patients will be offered enrollment into this LTFU protocol. Patients will be followed for up to 15 years following the RP-L102 infusion in the parent study, until the patient dies, withdraws consent, or is lost to follow-up (whichever occurs first).

For all follow-up visits, remote evaluation facilitated by local health care providers (with blood sample shipment to relevant laboratory facilities) is permitted; however, visits to the study center are required for up to 2 years post- RP-L102 infusion. Study center visits are encouraged when feasible, especially in years 2 through 5 following gene therapy administration. Blood samples will be archived and tested when clinically or scientifically indicated, as in the event of development of a second malignancy.

Study Type

Observational

Enrollment (Estimated)

14

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Spain
      • Madrid, Spain, 28009
        • Hospital Infantil Universitario Nino Jesus
  • United Kingdom
      • London, United Kingdom, WC1N 1EH
        • University College London Great Ormond Street Institute of Child Health (GOSH)
  • United States
    • California
      • Palo Alto, California, United States, 94305
        • Lucille Packard Children's Hospital, Stanford University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Subjects that have been treated with RP-L102 on the RP-L102-0418, RP-L102-0118 or RP-L102-0319 parent studies.

Description

Inclusion Criteria:

  1. Enrolled in one of the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).
  2. Received an autologous infusion of CD34+ enriched cells transduced ex vivo with LV vector carrying the FANCA gene, PGK-FANCA-WPRE (RP-L102), in the parent studies.
  3. Able to adhere to the study visit schedule and other protocol requirements.
  4. Has provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements.

Exclusion Criteria:

There are no criteria for exclusion in this study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Subjects that received RP-L102 on the RP-L102-0418, RP-L102-0118 and RP-L102-0319 parent studies
Subjects that received RP-L102 on the RP-L102-0418, RP-L102-0118 and RP-L102-0319 parent studies and either completed the study or discontinued early.
Biological: RP-L102
CD34+ enriched cells from subjects with Fanconi anemia subtype A (FA-A) transduced ex vivo with lentiviral vector carrying the FANCA gene, PGK-FANCA-WPRE

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Survival in patients treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).
Time Frame: From infusion in parent study to 15-years post-infusion.
Overall survival and allogeneic-HSCT-free survival will be summarized using Kaplan-Meier estimates. Events for allogeneic-HSCT-free survival are allogeneic-HSCT or death. In addition, event-free survival based on death and any of the following events will be summarized similarly: (1) BMF, (2) MDS/AML, and (3) BMF and MDS/AML.
From infusion in parent study to 15-years post-infusion.
Long term safety
Time Frame: From infusion in parent study to 15-years post-infusion.
To evaluate long-term safety following infusion of hematopoietic cells transduced with the therapeutic lentiviral vector (LV).
From infusion in parent study to 15-years post-infusion.
Long-term persistence of the therapeutic LV (provirus) in hematopoietic cells in the bone marrow (BM) and blood.
Time Frame: From infusion in parent study to 15-years post-infusion.
To determine long-term persistence of the therapeutic LV (provirus) in hematopoietic cells in the bone marrow (BM) and blood, and to evaluate potential correlations between provirus/transgene persistence and hematologic stability (absence of bone marrow failure [BMF] or hematologic malignancy).
From infusion in parent study to 15-years post-infusion.
Long-term clonality patterns.
Time Frame: From infusion in parent study to 15-years post-infusion.
To determine long-term clonality patterns beyond the 3-year follow-up stipulated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).
From infusion in parent study to 15-years post-infusion.
Replication-competent lentivirus (RCL) in serum and peripheral blood cells.
Time Frame: From infusion in parent study to 15-years post-infusion.
To evaluate, when relevant, replication-competent lentivirus (RCL) in serum and peripheral blood cells (this will not be considered relevant for subjects in whom no evidence of RCL was identified during the initial year following investigational autologous cell infusion).
From infusion in parent study to 15-years post-infusion.
Long-term stability and normalization of blood counts.
Time Frame: From infusion in parent study to 15-years post-infusion.
To determine the long-term stability and normalization of blood counts in patients after RP-L102 infusion on the parent studies.
From infusion in parent study to 15-years post-infusion.
Phenotypic correction of BM and peripheral blood cells
Time Frame: From infusion in parent study to 15-years post-infusion.
To determine the phenotypic correction of BM and peripheral blood (PB) cells (as evaluated by resistance to DNA-damaging agents) in long-term follow-up after gene therapy. BM CFU MMC resistance will be summarized by percentage of patients with expression of ≥20% from at each timepoint
From infusion in parent study to 15-years post-infusion.
Incidence of hematologic malignancies and solid organ tumors.
Time Frame: From infusion in parent study to 15-years post-infusion.
To enable preliminary assessment of the incidence of hematologic malignancies (including acute myeloid leukemia [AML]/myelodysplastic syndrome [MDS]) and solid organ tumors (including squamous cell carcinoma of the head and neck); occurrence of these events will be evaluated in the context of the underlying rates of these malignancies in Fanconi anemia (FA) patient populations (both those who have not undergone allogeneic stem cell transplant and FA patients post-hematopoietic stem cell transplantation [HSCT]).
From infusion in parent study to 15-years post-infusion.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Rocket Pharmaceuticals Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2022

Primary Completion (Estimated)

May 1, 2038

Study Completion (Estimated)

May 1, 2038

Study Registration Dates

First Submitted

April 7, 2026

First Submitted That Met QC Criteria

April 7, 2026

First Posted (Actual)

April 14, 2026

Study Record Updates

Last Update Posted (Actual)

April 14, 2026

Last Update Submitted That Met QC Criteria

April 7, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RP-L102-0221-LTFU

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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