Small step toward gene therapy for rare anemia

NCT ID NCT02678533

Completed Knowledge-focused Sponsor: Assistance Publique - Hôpitaux de Paris Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This pilot study tested whether a combination of two drugs (G-CSF and plerixafor) could safely collect enough stem cells from the blood of children with Fanconi anemia for future gene therapy. Only 4 patients took part, and the main goal was to see if the cell collection process was feasible. The study did not test gene therapy itself, but lays groundwork for later trials.

What this could mean

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

Active substance: G-CSF and plerixafor
What this could lead to: If successful, this method could enable gene therapy for Fanconi anemia, potentially correcting the genetic defect.
What could go wrong: This was a tiny pilot study (4 patients) focused on feasibility, not treatment. Gene therapy itself was not tested here, and many steps remain before any cure is possible.

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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

Contacts and locations

Locations

Hôpital necker-Enfants malades

Paris, PARIS, 75015, France

More trials for these conditions

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