Phase 2 Sickle Cell Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- People aged 12 to 40 years old
- People with sickle cell disease who have had at least one of the following despite receiving appropriate care: four or more severe pain crises in the past 2 years, OR two or more episodes of acute chest syndrome in the past 2 years with at least one episode occurring in the last year
- People with a general health and activity level that meets a certain minimum threshold, as measured by a standard medical scoring tool (confirm with trial site)
Who may not be able to join:
- People who have a fully matched sibling donor available for a stem cell transplant
- People who have previously had a stem cell transplant or gene therapy
- People who have, or have had, a blood cancer, a bone marrow disorder, or a significant problem with blood clotting or the immune system
- People who currently have a serious active infection, whether bacterial, viral, fungal, or parasitic
- People who are pregnant or currently breastfeeding
- People who have a chromosomal abnormality or genetic mutation that the treating doctor believes may increase the risk of developing certain blood disorders
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Matthew Porteus, MD, PhD, Kamau Therapeutics
Phone: 650-442-2283
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Proportion of patients who reach neutrophil engraftment; Incidence rate of treatment-related mortality; Incidence rate of treatment-related mortality; Overall survival; Frequency and severity of AEs/SAEs
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.