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

NCT07527975 | Enrolling By Invitation DMC FDA Drug

• 1 other identifier: RP-L102-0221-LTFU
• Study Type: observational
• Target: 14
• Locations: 3 countries
• Sites: 3

Brief Summary

Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102

Conditions

Fanconi Anemia

Outcome Measures

Primary Outcomes (8)

• Survival in patients treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).

  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

  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.

  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.

  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.

  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.

  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

  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.

  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.

Study Arms (1)

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

Interventions

RP-L102 BIOLOGICAL

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

Subjects that received RP-L102 on the RP-L102-0418, RP-L102-0118 and RP-L102-0319 parent studies

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Enrolled in one of the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).
• 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.
• Able to adhere to the study visit schedule and other protocol requirements.
• Has provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements.

Sponsors & Collaborators

• Rocket Pharmaceuticals Inc.: lead

Study Sites (3)

Lucille Packard Children's Hospital, Stanford University

Palo Alto, California, 94305, United States

Location

Hospital Infantil Universitario Niño Jesús

Madrid, 28009, Spain

Location

University College London Great Ormond Street Institute of Child Health (GOSH)

London, WC1N 1EH, United Kingdom

Location

Biospecimen

Retention: SAMPLES WITH DNA

Human Tissue (including blood and bone marrow)

MeSH Terms

Conditions

Fanconi Anemia

Condition Hierarchy (Ancestors)

Anemia, Hypoplastic, Congenital; Anemia, Aplastic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Congenital Bone Marrow Failure Syndromes; Bone Marrow Failure Disorders; Bone Marrow Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 7, 2026
• First Posted: April 14, 2026
• Study Start: February 1, 2022
• Primary Completion (Estimated): May 1, 2038
• Study Completion (Estimated): May 1, 2038
• Last Updated: April 14, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1); ES (1); GB (1)