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 between 2 and 35 years old
• People diagnosed with a specific type of sickle cell disease — either homozygous hemoglobin S (HbSS), hemoglobin SC (HbSC), or hemoglobin S beta-thalassemia (HbSβ0 or HbSβ+)
• People who have given consent to participate in this specific study
• People who have had at least one serious sickle cell-related complication, such as:
  • A stroke or significant neurological (brain or nervous system) problem
  • A "silent" crisis or problems with thinking, memory, or concentration
  • Abnormal results on a specific type of brain blood vessel scan (MRI with TOF sequence)
  • Abnormal blood flow speeds in brain arteries detected on two separate tests more than one month apart
  • More than 5 painful crises in the past year, or more than 20 painful crises over a lifetime
  • Two or more episodes of acute chest syndrome ever, or one episode in the past two years
  • A long-term need for blood transfusions, or more than 8 transfusions, or one exchange transfusion over a lifetime
  • A reaction to blood transfusions where the body develops antibodies against transfused blood (confirm with trial site)
  • More than 5 hospitalisations related to sickle cell disease over a lifetime
  • Early signs of high blood pressure in the lungs
  • Bone damage (bone death) at more than two sites in the body
  • Early signs of kidney damage related to sickle cell disease
  • Repeated painful erections (more than two episodes)

Who may not be able to join:

• People whose general health and ability to function is rated below 70% on a standard medical performance scale
• People whose immune system has developed specific antibodies against a potential stem cell donor, detected through certain blood or laboratory tests
• People whose blood type is significantly incompatible with their potential stem cell donor, as defined by specific transplant guidelines (confirm with trial site)
• People with poor heart function, including a pumping strength (ejection fraction) below 45%, a shortening fraction below 27% on heart scans, or blood pressure that is difficult to control
• People whose kidneys are not working well enough to meet the minimum levels required for stem cell transplantation, based on age-specific measurements (confirm with trial site)
• People with poor lung function, including low scores on breathing tests, or children who cannot perform breathing tests and have low blood oxygen levels
• People with significantly abnormal liver function results, or who have chronic active hepatitis (a long-term liver infection caused by a virus)
• People who are pregnant or breastfeeding; women of childbearing age will need a negative pregnancy test before entering the study
• People of reproductive age who are not willing to use effective contraception during the treatment and for at least 12 months afterwards
• People with an autoimmune disease (where the immune system attacks the body) that is not well controlled, or who are currently being treated for one
• People who would not be able to follow the treatment plan required by the study
• People who have previously had a stem cell transplant (either using their own or a donor's stem cells)
• People who receive a live virus vaccine during the trial
• People with HIV infection
• People with a history of psychiatric illness, alcohol dependency, or drug addiction that could affect their ability to understand and participate in the study
• People who are unwilling or unable to follow the study requirements or give informed consent
• People with another serious or uncontrolled medical condition — such as uncontrolled diabetes, heart failure, a heart attack within the past 6 months, uncontrolled high blood pressure, chronic kidney disease, or an active uncontrolled infection — that the treating doctor considers could affect safe participation

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