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Small step toward gene therapy for rare anemia

NCT ID NCT02678533

Knowledge-focused Sponsor: Assistance Publique - Hôpitaux de Paris 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

4 people

The number who actually took part.

Started

Feb 2017

Finished

May 2019

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 17 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: * Patient with Fanconi anemia * Patient from 2 to 17 years old * Potential indication for allogenic bone arrow graft without HLA-identical brotherhood available * Patient's weight \>10kg * Treated and followed for at least the previous two years in a specialized center where they got a full assessment of their disease * For women of childbearing age, not pregnant and use of an effective contraception during the entire participation in the research. * Affiliated or beneficiary of an health insurance regimen * Informed and signed consent Exclusion Criteria: * Patient unable to follow the visits required by the protocol * Positive serology for HIV-1/2, HTLV-1/2, HCV and HbS * Bacterial, viral, fungal or parasitic active infection with clinical signs * Personal history of cancer, myeloproliferative hematopathy or immune deficiency * Heart failure and / or heart rhythm disorder * History of allogeneic graft of hematopoietic stem cells * Patient with an HLA-identical brotherhood donor available * Myelodysplasia diagnose on myelogram * Cytogenetic abnormality on karyotype * Malignant solid tumor * Documented spontaneous genetic reversion of medullary process * Diagnosis of a psychiatric disorder that could compromise his/her ability to participate in the study * Any disorder according to the investigator, that could compromise the ability of patient to give his writing consent and/or to comply with requiring study's procedures * Current Pregnancy * Heart, kidney or liver failure * Current participation in another interventional clinical trial * Patient under Medical Assistance State * Hypersensitivity to plerixafor or any excipient contained in MOZOBIL® * Hypersensitivity to filgrastim or any of its' excipient

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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.