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:

For the person receiving the transplant (recipient):

• People aged 22 years or younger.
• People who do not have a fully matched sibling donor available, but do have a partially matched (at least half-matched) family member who is willing to donate stem cells.
• People who have been diagnosed with sickle cell disease (any type) AND meet at least one of the following:
• A history of abnormal blood flow readings in the brain (measured by a specific ultrasound test called a transcranial Doppler) on at least two separate occasions.
• A history of a stroke or silent stroke shown on a brain MRI.
• Two or more episodes of a serious lung complication called acute chest syndrome in the past two years.
• Two or more severe pain crises requiring intravenous pain medication in the past 12 months.
• Two or more episodes of a prolonged, painful erection lasting 4 hours or more, or requiring emergency medical care.
• Regular blood transfusions, meaning 8 or more transfusions in the past 12 months.
• Worsening damage to organs (such as the heart, kidneys, or lungs) that at least two independent blood specialists have agreed in writing is not responding to other treatments and may benefit from a stem cell transplant.

For the donor:

• A family member who is at least a partial (half) match to the recipient.
• Someone who has tested negative for HIV.
• Someone who is not pregnant (confirmed by a blood or urine test within 14 days before joining).
• Someone who is not breastfeeding.
• Someone who does not have a significant blood disorder affecting haemoglobin — note that having sickle cell trait (carrying one copy of the gene, not the disease itself) is acceptable.
• Someone who has completed the required donor health and safety assessment process, or for whom the lead doctor has documented an urgent medical need (confirm with trial site).

Who may not be able to join:

For the person receiving the transplant (recipient):

• People with a low general health or ability to function score (below 60 on standard medical scales).
• People who are currently pregnant (confirmed by a blood or urine test within 14 days before joining).
• People who are currently breastfeeding.
• People with an active, uncontrolled infection (bacterial, viral, or fungal) that is getting worse despite treatment, within one month before starting the pre-transplant treatment — people with a minor or suspected infection should wait until it resolves.
• People with confirmed HIV infection.
• People with significantly elevated liver function levels in the blood, specifically a type called conjugated (direct) bilirubin more than three times the normal upper limit for their age — though people whose levels are raised due to a specific reaction to blood transfusions may still be eligible if the levels are coming back down (confirm with trial site).
• People whose heart is not pumping strongly enough, based on an ultrasound of the heart.
• People whose kidneys are not filtering blood well enough (below a certain threshold on a standard kidney function measure).
• People whose lungs are not working well enough, based on breathing tests or blood oxygen levels.
• People who have developed antibodies that specifically target the donor's tissue type, if those antibodies cannot be reduced with treatment.

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