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Gene therapy aims to fix bone marrow failure in fanconi anemia

NCT ID NCT03351868

Disease control Sponsor: Shenzhen Geno-Immune Medical Institute 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
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
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This status has not been confirmed recently, so it may be out of date.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 Aug 25, 2026 · Updated 2 times

Summary

This trial tests a gene therapy for Fanconi anemia, a rare genetic disorder that causes bone marrow failure and increases cancer risk. Participants receive their own stem cells that have been genetically corrected with a lentiviral vector to fix the faulty FANCA gene. The study evaluates safety and whether the treatment can improve blood cell counts and reduce disease complications.

What this could mean

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

Active substance
gene-modified stem cells
What this could lead to
If successful, this gene therapy could restore normal blood cell production and reduce the need for transfusions or bone marrow transplants in people with Fanconi anemia.
What could go wrong
This is an early-phase trial with only 10 participants, so results may not apply to everyone. Risks include immune reactions or the gene therapy not working as expected.

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.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

10 people

The number who actually took part.

Started

Jun 2026

Finished

Jul 2026

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

2 to 20 years

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. Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide. 2. No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection. 3. Age: ≥ 4 years. 4. Karnofsky: ≥ 70%. 5. ANC ≥ 5×10\^8/L; PLT ≥ 2×10\^10/L. 6. Hemoglobin ≥ 8g/dL. 7. Proper renal and hepatic functions (ULN denotes "upper limit of normal range") with * serum creatinine ≤ 1.5×ULN; * serum bilirubin ≤ 3×ULN; * AST/ALT ≤ 5×ULN. 8. Pulmonary function is normal; DLCO \> 50%. 9. Written, informed consent obtained prior to any study-specific procedures. Exclusion Criteria: 1. Diagnosis of active malignant disease or myelodysplastic syndrome. 2. Diagnosis of myeloid leukemia. 3. Pregnant or lactating females. 4. Existence of an available HLA-identical related donor. 5. Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive. 6. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.

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Conditions

The condition(s) this trial relates to.

Fanconi anemia Fanconi anemia complementation group A

As listed by the trial registrant

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

Fanconi anemia

Contacts and locations

Locations

  • Shenzhen Geno-immune Medical Institute

    Shenzhen, Guangdong, 518000, China

More trials for these conditions

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