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

Plain-language summary

This study follows the long-term safety and efficacy outcomes of patients who previously received RP-L102, a gene therapy using corrected stem cells carrying the FANCA gene. Only people who took part in and were treated in the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118) are included. This is a study with no assigned phase and it enrolls participants by invitation.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Enrolling by invitation

Sponsor

Rocket Pharmaceuticals Inc.

Intervention

RP-L102

Start date

2/1/2022

Estimated completion

5/1/2038

Countries

United States, Spain, United Kingdom

Related genes

Locations

• Palo Alto, United States
• Madrid, Spain
• London, United Kingdom

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.