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 patients with sickle cell disease:

• People who have sickle cell disease and have had a stroke confirmed by brain MRI
• People who have an abnormal result on a special brain blood flow test called a transcranial Doppler (reading of 200 cm/s or higher)
• People who have a "silent" brain injury (a small area of damage seen on MRI but with no obvious symptoms, confirmed by a neurologist)
• People who have sickle cell-related kidney problems, based on blood or urine test results, kidney biopsy findings, or who are already on dialysis
• People who have a specific heart finding (a tricuspid regurgitant jet velocity of 2.5 m/s or more), measured at least 3 weeks after a pain crisis
• People who have had repeated severe, prolonged, painful erections (priapism) — at least two episodes lasting 4 hours or more that required medical treatment
• People who have sickle cell-related liver problems, based on specific blood test levels
• People who have been admitted to hospital more than once per year for pain crises while already taking sickle cell medication
• People who have had an acute chest syndrome episode while already taking sickle cell medication

For patients with beta-thalassemia:

• People with beta-thalassemia who have a significant level of iron build-up in the body (grade 2 or 3), shown by two or more of the following: liver scarring on biopsy, a history of not being able to maintain iron-removal treatment, or an enlarged liver detected by physical exam or imaging

For all patients (sickle cell and beta-thalassemia):

• People aged 4 years old up to and including 64 years old
• People who have a fully matched stem cell donor (matched at all required tissue-type markers)
• People who are able to understand and sign a consent form (for children, a parent or guardian provides consent, and the child provides assent where appropriate)
• People who have a negative pregnancy test result, where applicable
• People who agree to use reliable contraception throughout the study and for 3 months after certain study medications are stopped — female participants must use an accepted method such as the pill, an intrauterine device, barrier methods with spermicide, or an implant or injection; male participants must use effective contraception including condoms

For stem cell donors:

• People who are a fully matched tissue-type donor at all required markers
• People aged 4 or older and who weigh at least 15 kg (or a weight that the medical team considers acceptable for the procedures involved)
• People who are assessed as eligible and suitable to donate stem cells under standard clinic guidelines

Who may not be able to join:

For patients:

• People whose general physical functioning falls below a certain level on a standard medical scale (confirm with trial site)
• People whose lung function is significantly reduced — specifically, a breathing test result below 35% of the expected value
• People whose blood oxygen level is below 85%, or whose blood oxygen pressure reading is below 70
• People whose heart pumping function is below 35%, as measured by an ultrasound of the heart
• People whose liver enzyme levels are more than 5 times the normal range for their age
• People who have an active, uncontrolled infection (bacterial, viral, or fungal) that is not responding to treatment, within one month before starting the pre-transplant conditioning treatment
• People with a serious illness or organ failure that would make surviving a stem cell transplant unlikely
• People who are pregnant or breastfeeding

For stem cell donors:

• People who are pregnant or breastfeeding
• People who have a significant cognitive impairment

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