By InvitationNot Applicable

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

United States, Spain, United Kingdom14 participantsStarted 2022-02-01

Plain-language summary

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

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Survival in patients treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).

Timeframe: From infusion in parent study to 15-years post-infusion.

Long term safety

Timeframe: 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.

Timeframe: From infusion in parent study to 15-years post-infusion.

Long-term clonality patterns.

Timeframe: From infusion in parent study to 15-years post-infusion.

Replication-competent lentivirus (RCL) in serum and peripheral blood cells.

Timeframe: From infusion in parent study to 15-years post-infusion.

Long-term stability and normalization of blood counts.

Timeframe: From infusion in parent study to 15-years post-infusion.

Phenotypic correction of BM and peripheral blood cells

Timeframe: From infusion in parent study to 15-years post-infusion.

Trial details

NCT IDNCT07527975

SponsorRocket Pharmaceuticals Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2038-05

View on ClinicalTrials.gov More Fanconi Anemia trials

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

What they're measuring

Survival in patients treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118).

Timeframe: From infusion in parent study to 15-years post-infusion.

Long term safety

Timeframe: 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.

Timeframe: From infusion in parent study to 15-years post-infusion.

Long-term clonality patterns.

Timeframe: From infusion in parent study to 15-years post-infusion.

Replication-competent lentivirus (RCL) in serum and peripheral blood cells.

Timeframe: From infusion in parent study to 15-years post-infusion.

Long-term stability and normalization of blood counts.

Timeframe: From infusion in parent study to 15-years post-infusion.

Phenotypic correction of BM and peripheral blood cells

Timeframe: From infusion in parent study to 15-years post-infusion.