Tiny study tests drug to harvest stem cells in sickle cell patients

NCT ID NCT02212535

First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This early-phase study tested whether a single injection of plerixafor could safely and effectively mobilize stem cells in 3 adults with severe sickle cell disease who lacked a matched sibling donor. The goal was to see if enough stem cells could be collected for a possible future transplant. The study was very small and focused only on the collection step, not the transplant itself.

What this could mean

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

Active substance

plerixafor

What this could lead to

If successful, this could show that plerixafor safely mobilizes stem cells in sickle cell patients, potentially enabling future stem cell transplants for those without a matched donor.

What could go wrong

This is a very early, tiny study (only 3 patients) testing only the stem cell collection step, not the transplant itself. It may not lead to a treatment, and risks include complications from the procedure.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

sickle cell disease sickle cell-beta-thalassemia disease syndrome

As listed by the trial registrant

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

Contacts and locations

Locations

  • Hôpital Necker - Enfants Malades

    Paris, 75015, France