Phase 2 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:

• People aged 2 to 50 years old.
• People who have a suitable half-matched (haploidentical) donor available.
• People who have a reasonable level of physical functioning, as measured by standard medical scoring tools.
• People whose kidneys are working well enough, based on blood test results (the trial site uses specific formulas to check this based on age).
• People whose liver is producing normal or near-normal levels of certain markers in their blood.
• People whose heart is pumping effectively (at least 50% efficiency).
• People whose lungs are functioning adequately; for children under 7 who cannot do breathing tests, a blood oxygen level above 92% is used instead.
• People with sickle cell disease (specifically types HbSS, HbSC, or HbS/β°) who have experienced one or more serious complications, such as: two or more episodes of acute chest syndrome in the past two years; three or more severe pain crises in the past two years; two or more episodes of prolonged unwanted erections in the past two years; a history of bone infection or bone death; evidence of stroke or brain blood vessel disease; confirmed high blood pressure in the lungs; or a significant number of antibodies against red blood cells (more than three).
• People with thalassemia of any type who meet all of the following: needed regular blood transfusions from before age 3; have received a high volume of blood transfusions over their lifetime; have a low level of haemoglobin before transfusions; and have an enlarged liver and spleen.
• People (or their legal guardian or parent) who are able to give written consent; children aged 7 to 17 must also give their own agreement to participate.
• People who are sexually active and able to have children who agree to use an effective method of contraception during the trial.

Who may not be able to join:

• People who have previously had a full-intensity (myeloablative) bone marrow or stem cell transplant from a donor.
• People who have had an overt stroke or brain surgery (for example, for Moyamoya disease) within the six months before enrolling.
• People who have liver cirrhosis (severe scarring of the liver); mild liver fibrosis may still be permitted (confirm with trial site).
• People with a high level of iron stored in the liver (at or above a specific threshold), where this is relevant.
• People with currently active Hepatitis B or C infection.
• People with other infections that are not under control.
• People with a current or recent cancer diagnosis, unless they have been in remission for at least two years after full treatment — certain low-risk skin or cervical conditions may be exceptions (confirm with trial site).
• People who are pregnant or who have a positive pregnancy test (applies to women who have not gone through menopause or had surgical sterilisation).
• People who are unlikely to be able to follow the treatment plan or attend required follow-up appointments.
• People with a known allergy to any component of the stem cell product used in the trial, including a preservative called DMSO.

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