Early Phase 1 Sickle Cell Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• You must be between 12 and 35 years old (inclusive); if you are under 18, a parent or legal guardian must also give written permission.
• You must have a confirmed diagnosis of sickle cell disease with a specific genetic type: βS/βS, βS/β0, or βS/β+.
• You must have experienced at least one of the following: (a) at least 2 serious sickle cell events in the past 2 years — such as a severe pain crisis needing hospital treatment, acute chest syndrome, priapism lasting more than 2 hours requiring medical attention, or a stroke or mini-stroke confirmed by a brain scan; (b) development of multiple antibodies to red blood cells (more than 2) requiring ongoing regular blood transfusions; (c) you have tried, cannot tolerate, refused, or cannot access the standard treatment for sickle cell disease; or (d) your doctor determines that a stem cell transplant is appropriate based on sickle cell treatment guidelines.
• Before any blood transfusion, your lab results must show that at least 30% of your hemoglobin is the sickle cell type (HbS).
• Your fetal hemoglobin (HbF) level at screening must be below 20%.
• You must be well enough to go through the treatment, based on a standard health and activity assessment score of 70 or above (confirm with trial site for which scale applies to your age).
• You must be willing to follow all study requirements, use contraception if needed, attend regular check-up visits, and cooperate with all tests and examinations.

Who may not be able to join:

• Women who are pregnant, breastfeeding, or planning to become pregnant during the study period.
• People who have taken part in another clinical drug trial within the past 30 days (or longer, depending on the drug being studied).
• People who have received a drug called luspatercept within the past 3 months.
• People who have previously had any form of gene therapy for sickle cell disease.
• People who already have a fully matched family donor and are scheduled for a donor stem cell transplant.
• People who have had more than 10 unplanned hospital stays or emergency visits in the past 12 months that their doctor believes are due to ongoing long-term pain rather than sudden sickle cell pain crises.
• People with severe liver problems, including certain abnormal liver blood test results above specific limits (confirm with trial site).
• People with severely reduced kidney function (confirm with trial site).
• People with active infections including HIV, certain viral infections (CMV, EBV), syphilis, active hepatitis B or C, known tuberculosis, or active bacterial, viral, or fungal infections (note: people with successfully treated hepatitis B or C may not be excluded — confirm with trial site).
• People who the doctor determines are not suitable for the stem cell collection and transplant procedure used in this study.
• Anyone else the investigator considers unsuitable for the study for other reasons.

Important: Always verify eligibility with the trial site directly before applying.