SCID stem cell trial pulled before it began
NCT ID NCT04370795
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study aimed to see if lower doses of chemotherapy drugs, with or without radiation, could safely prepare children and adults with SCID for a stem cell transplant. The goal was to help the donor cells take hold while avoiding severe side effects. However, the trial was withdrawn before enrolling any participants, so no results are available.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Busulfan, sirolimus, horse-anti-thymocyte globulin, G-CSF, and total body irradiation
- What this could lead to
- If successful, this could have pointed toward a safer way to prepare SCID patients for stem cell transplants, reducing complications.
- What could go wrong
- The trial was withdrawn before enrolling anyone, so no data were collected. The approach still carries risks like graft-versus-host disease and infection.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States