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:

• You have a non-cancerous condition that requires a bone marrow transplant, such as bone marrow failure, a metabolic disorder, an immune system disorder, or a blood disorder like sickle cell disease or thalassemia
• If you have sickle cell disease, you must also have at least one serious complication, such as a stroke or silent stroke, dangerously high blood flow speeds in brain vessels despite transfusion treatment, repeated severe chest crises affecting your breathing, kidney damage from sickle cell, frequent painful crises requiring long-term opioid pain relief and seriously affecting your daily life or school attendance, a reaction to blood transfusions making it hard to find compatible blood, or repeated bone or joint damage from loss of blood supply
• If you have sickle cell disease, a recent blood test (within the last 30 days before starting treatment) must show that less than 30% of your red blood cells are the sickle type
• You are 20 years old or younger at the time of joining the trial
• You are well enough to carry out some daily activities (confirm with trial site)
• Your heart is pumping well enough, as shown by a heart ultrasound
• Your lungs are working well enough, either by a breathing test or by checking your oxygen levels while breathing normal air
• Your kidneys are working well enough, based on blood test results
• Your liver's bilirubin levels (a waste product) are not too high, based on blood test results
• Your liver enzyme levels are not too high, based on blood test results
• If you have had many blood transfusions over your lifetime, you may need a liver scan to check for iron build-up; if iron levels are very high, a liver biopsy may be needed to confirm there is no serious liver damage
• If you are a woman who could become pregnant, you must agree to use two forms of contraception from the time you sign consent until 12 months after the transplant
• If you are male, you must agree to use an effective barrier contraception method or remain abstinent from signing consent until 12 months after the transplant
• You (or your parent/guardian if a minor) must be able to give written consent in line with the study institution's guidelines

Who may not be able to join:

• You have a brother or sister whose tissue type (HLA) is a perfect match and who is able and willing to donate bone marrow
• You have serious liver scarring (cirrhosis or bridging fibrosis) or active hepatitis (liver inflammation)
• You have had an uncontrolled bacterial, viral, or fungal infection within the 6 weeks before joining the trial
• You have HIV infection or have tested positive for HIV
• You have previously had a stem cell or bone marrow transplant
• You have taken an experimental drug or device, or a drug being used outside its approved purpose, within the 3 months before joining the trial
• You are currently pregnant or breastfeeding
• You have an active autoimmune disease, such as sarcoidosis, lupus, or scleroderma (conditions where the immune system attacks the body)

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