An observational study in Fanconi Anemia, sponsored by Rocket Pharmaceuticals Inc.. Enrolling by invitation at 3 sites in 3 countries. Per ClinicalTrials.gov, last updated 2026-04-14.
Sponsored by Rocket Pharmaceuticals Inc. · Observational
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
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.
95 studies on the registry are indexed under Fanconi Anemia; 20 are open to participants now.
This study's planned enrollment of 14 is below the median of 94 across 24 observational studies indexed under Fanconi Anemia.
Browse Fanconi Anemia studies →Rocket Pharmaceuticals Inc. is the lead sponsor of 19 studies on the registry; 7 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Subjects that have been treated with RP-L102 on the RP-L102-0418, RP-L102-0118 or RP-L102-0319 parent studies.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
There are no criteria for exclusion in this study.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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.
Time frame: 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).
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.
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).
Time frame: 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).
Time frame: 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).
Time frame: 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.
Time frame: 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
Time frame: 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 \[HSCT\]).
Time frame: From infusion in parent study to 15-years post-infusion.
Plan to share: No
No publications or documents are linked to this record.
Started Feb 2022 (actual). Primary completion is estimated for May 2038, 195 months after the start.
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Rocket Pharmaceuticals Inc.