Small study tests plerixafor for gene therapy in sickle cell
NCT ID NCT03664830
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early-phase trial is testing whether the drug plerixafor can safely and effectively collect enough stem cells from people with sickle cell disease for a future gene therapy. Only 5 participants are enrolled, and the main goal is to check for side effects. If it works, it could be a step toward a treatment that controls the disease without lifelong medication.
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 pave the way for a gene therapy that helps control sickle cell disease without lifelong medication.
What could go wrong
This is a very small, early-phase safety study with only 5 participants. It may not lead to an effective treatment, and there are risks like side effects from the drug or failure to collect enough stem cells.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for SICKLE CELL DISEASE are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
City of Hope Medical Center
Duarte, California, 91010, United States