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Gene therapy for rare blood disease passes 15-Year safety watch

NCT ID NCT04437771

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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 9 people with Fanconi Anemia who already received a gene therapy that adds a working FANCA gene to their blood stem cells. Researchers will check their health and blood counts for 15 years to see if the treatment remains safe and keeps working. No new treatment is given during this follow-up.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
gene therapy (autologous CD34+ cells with FANCA gene)
What this could lead to
If successful, this could show that a one-time gene therapy safely stabilizes blood counts and reduces cancer risk for people with Fanconi Anemia.
What could go wrong
This is a very small, early-phase follow-up study with only 9 patients. It cannot prove the therapy works for everyone, and long-term risks like cancer or side effects from the gene insertion are still unknown.

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 9 people

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

Started

Jun 2020

Expected to finish

Jan 2034

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 with Fanconi anemia subtype A who have been treated with ex vivo gene therapy product in FANCOLEN-I study

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 the FANCOLEN-I study 2. Treated with gene therapy in the FANCOLEN-I study 3. Able to adhere to the study visit schedule and protocol requirements 4. Provided written informed consent and, as applicable, assent to participate Exclusion Criteria: * There are no exclusion criteria for this study

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Conditions

The condition(s) this trial relates to.

anemia Bone Marrow Failure Disorders Fanconi anemia Fanconi anemia complementation group A Fanconi renotubular syndrome hereditary disease

As listed by the trial registrant

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

Fanconi anemia Fanconi anemia complementation group A

Contacts and locations

Locations

  • Hospital Infantil Universitario Niño Jesús (HIUNJ)

    Madrid, 28009, Spain

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