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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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
Locations
-
City of Hope Medical Center
Duarte, California, 91010, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can Co-Designed support help parents embrace sickle cell care?
- Can community hospitals in zambia master severe disease care?
- A Pocket-Sized coach for sickle cell: can an app turn Pill-Taking into a habit?
- Can a phone app keep teens on track with sickle cell treatment?
- A common amino acid may calm sickle cell pain crises — a trial puts it to the test
- A drug that protects the liver may make stem cell transplants safer for children with sickle cell disease