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

Status

Enrolling by invitation

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT07527975

Enrollment

Registered

2026-04-14

Start date

2022-02-01

Completion date

2038-05-01

Last updated

2026-04-14

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fanconi Anemia

Brief summary

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

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.

Interventions

BIOLOGICALRP-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

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Survival in patients treated 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.	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.
Long term safety	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).
Long-term persistence of the therapeutic LV (provirus) in hematopoietic cells in the bone marrow (BM) and blood.	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).
Long-term clonality patterns.	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).
Replication-competent lentivirus (RCL) in serum and peripheral blood cells.	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).
Long-term stability and normalization of blood counts.	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.
Phenotypic correction of BM and peripheral blood cells	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
Incidence of hematologic malignancies and solid organ tumors.	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 \[HSCT\]).

Countries

Spain, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 15, 2026