Skip to main content
OpenTrials
Enrolling by Invitation

NCT Number: NCT07527975

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

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

Enrolling by Invitation

Interested in participating?

Request Info

Key information

Conditions

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

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital Infantil Universitario Niño Jesús, Madrid, Spain

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

There are no criteria for exclusion in this study.

Treatment and study plan

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

Primary outcomes

  1. 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.

  2. 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).

  3. 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).

  4. 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).

  5. 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).

  6. 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.

  7. 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

  8. 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]).

Sponsors and collaborators

Lead sponsor

Rocket Pharmaceuticals Inc.

Industry

Registry information

Important dates

Study start
2022
Primary completion
2038
Study completion
2038
First posted
Apr 14, 2026
Registry last updated
Apr 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.