Phase 2 Sickle Cell Disease Trial, Recruiting

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

Who might be able to join this trial:

• The patient, their parent, or legal guardian must have signed a consent form agreeing to take part in the study.
• Participants must be between 5 and 40 years old at the time of signing the consent form.
• People with a diagnosis of Sickle Cell Disease (specifically the SS or Sβ0-thalassemia types) that has caused serious complications — such as frequent painful crises requiring hospital stays, repeated episodes of acute chest syndrome, stroke or brain injury, a need for regular blood transfusions, high blood flow speeds in brain arteries, signs of high pressure in lung blood vessels, or damage to the kidneys, liver, or other organs (some of these complications apply only to those aged 16 or older).
• Alternatively, people with beta-thalassemia or Diamond-Blackfan anemia who depend on regular blood transfusions and have signs of iron build-up in the body may also be considered.
• A partially matched bone marrow donor must be available, meeting specific tissue-matching requirements (confirm details with trial site), and a search for a fully matched unrelated donor or cord blood unit must have already been completed without a suitable match being found.
• Heart, lung, liver, and kidney function must each meet minimum levels as measured by specific medical tests (confirm exact thresholds with trial site).
• Cardiology and pulmonology specialists must have cleared the person to go ahead with the treatment and transplant process.
• The person must test negative for HIV.
• Females aged 10 or older, or those who have started their menstrual period, must have a negative pregnancy test (unless surgically unable to become pregnant).
• All females who could potentially become pregnant, and all sexually active males, must agree to use an approved method of birth control for up to 24 months after the transplant, or for as long as any medication that could harm a pregnancy is being taken.
• Participants and/or their guardians will be counselled about the possibility of infertility after the transplant and encouraged to discuss options such as sperm banking or egg freezing before treatment begins.
• People with sickle cell disease must have already tried the medication hydroxyurea and found that it did not work sufficiently for them.

Who may not be able to join:

• People who have a fully matched sibling donor or a fully matched unrelated donor available, as these are considered better donor options.
• People who have already had a stem cell transplant within the six months before the planned start of the preparatory treatment.
• People who have had a central nervous system (brain or spinal cord) event, such as a stroke, within the six months before the planned start of treatment — though the trial site may reassess eligibility once enough time has passed.
• People who are currently pregnant or breastfeeding.
• People who have an active infection (bacterial, viral, or fungal) that is not under control.
• People with other medical conditions, physical findings, or test results that, in the opinion of the study doctor, could increase the risks of taking part, affect the person's ability to follow the study requirements, or affect the reliability of the study results.

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