Fanconi AnemiaNCT07527975
Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
An observational study of Fanconi Anemia, run by Rocket Pharmaceuticals Inc. at 3 sites in 3 countries.
Trial summary
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102 Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Fanconi Anemia
- What is being tested?
- RP-L102
- How many people
- 14 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 1 Feb 2022to May 2038
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Subjects that have been treated with RP-L102 on the RP-L102-0418, RP-L102-0118 or RP-L102-0319 parent studies.
Who may be able to join
- 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.
Who cannot join
- There are no criteria for exclusion in this study.
Trial locations and contacts
3 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 3 countries
United States1 site
Lucille Packard Children's Hospital, Stanford University
Palo Alto, California, 94305
United Kingdom1 site
University College London Great Ormond Street Institute of Child Health (GOSH)
London, WC1N 1EH
About this study
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
Read the 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.
- Conditions
- Fanconi Anemia
- Being tested
- RP-L102
- Medical subject headings
- Fanconi Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RP-L102Biological
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 group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: RP-L102 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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
Measured: 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]).
Measured: From infusion in parent study to 15-years post-infusion.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2026
- Study start1 Feb 2022
- Primary completionMay 2038 (estimated)
- Study completionMay 2038 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fanconi AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07527975
- Sponsor's protocol ID
- RP-L102-0221-LTFU
- Status last verified
- Apr 2026
- First submitted
- 7 Apr 2026
- Last updated
- 14 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-Term Follow-up: Phase I/II Clinical Study to Evaluate the Safety and Efficacy of the Infusion of RP-L102
- ClinicalTrials.gov ID
- NCT07527975
- Acronym / sponsor ID
- RP-L102-0221-LTFU
- Conditions
- Fanconi Anemia
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- Rocket Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Feb 2022 (actual)
- Primary completion
- May 2038 (estimated)
- Study completion
- May 2038 (estimated)
- First posted
- 14 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects that received RP-L102 on the RP-L102-0418, RP-L102-0118 and RP-L102-0319 parent studies | – | Biological: RP-L102Subjects 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Subjects that have been treated with RP-L102 on the RP-L102-0418, RP-L102-0118 or RP-L102-0319 parent studies.
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (1 listed)
- There are no criteria for exclusion in this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RP-L102CD34+ enriched cells from subjects with Fanconi anemia subtype A (FA-A) transduced ex vivo with lentiviral vector carrying the FANCA gene, PGK-FANCA-WPRE | Biological | Subjects that received RP-L102 on the RP-L102-0418, RP-L102-0118 and RP-L102-0319 parent studies |
Primary outcome
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.
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).
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).
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).
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).
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.
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
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]).
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fanconi Anemia, run by Rocket Pharmaceuticals Inc. at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 14 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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.). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2026; AzTrials last refreshed its copy on 26 Oct 2026.