Plain-English translation of NCT06442761 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle Cell Disease research guide →Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This is a Phase 1 study testing a medication called to see if it helps doctors collect stem cells from people with severe sickle cell disease. The medication is given as an injection under the skin, and about 8 hours later, doctors will collect your stem cells through a process called apheresis. These cells may be used in future gene therapy treatments for sickle cell disease.
Collecting enough stem cells from sickle cell patients has been difficult with current methods. This study is testing whether this medication can make it easier and more effective to collect these cells, which could help make future gene therapy treatments possible for more people with severe sickle cell disease.
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If you join this study, you will receive one or two injections of the medication under your skin (depending on whether you enroll early or later in the study). About 8 hours after each injection, doctors will collect your stem cells through a procedure called apheresis, which takes blood from your arm, separates out the stem cells, and returns the rest of the blood to your body. You'll have a follow-up visit 7–10 days after the procedure and a phone call or clinic visit about 30 days after your last dose.
AI-generated summary from trial data · Jun 9, 2026 · Not medical advice
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