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

NCT ID NCT04437771

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

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.

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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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