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Enrolling by invitation NCT07527975

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

— Observational Fanconi Anemia

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: RP-L102.
Who it may be relevant to
Registry conditions: Fanconi Anemia. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Spain, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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

  • 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

Primary outcome measures

  • 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.]
  • Long term safety [Time frame: 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.]
  • Long-term clonality patterns. [Time frame: 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.]
  • Long-term stability and normalization of blood counts. [Time frame: 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.]
  • Incidence of hematologic malignancies and solid organ tumors. [Time frame: From infusion in parent study to 15-years post-infusion.]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Case-only

Study locations

United States · 1 center
  • Lucille Packard Children's Hospital, Stanford University — Palo Alto
Spain · 1 center
  • Hospital Infantil Universitario Niño Jesús — Madrid
United Kingdom · 1 center
  • University College London Great Ormond Street Institute of Child Health (GOSH) — London

Identifiers

NCT: NCT07527975 · RP-L102-0221-LTFU

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗