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Gene therapy for rare blood disease shows promise in Long-Term Follow-Up

NCT ID NCT07527975

Disease control Sponsor: Rocket Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 14 people with Fanconi Anemia who previously received RP-L102 gene therapy. Researchers will monitor their health for years to see if the treatment safely improves blood counts and reduces the need for a bone marrow transplant. The goal is to understand long-term benefits and risks.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 14 people

The number the study aims to enrol. It can still change while the study runs.

Started

Feb 2022

Expected to finish

May 2038

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Subjects that have been treated with RP-L102 on the RP-L102-0418, RP-L102-0118 or RP-L102-0319 parent studies.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Fanconi anemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fanconi anemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    3 sites in 3 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Hospital Infantil Universitario Niño Jesús

    Madrid, 28009, Spain

  • Lucille Packard Children's Hospital, Stanford University

    Palo Alto, California, 94305, United States

  • University College London Great Ormond Street Institute of Child Health (GOSH)

    London, WC1N 1EH, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.