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 with sickle cell disease (SCD) between the ages of 2 and 24 years old who have a healthy brother or sister with a matching immune system type (HLA-identical), where there is a major blood type mismatch between the patient and donor, OR where the patient has developed antibodies against certain markers on the donor's red blood cells.
• People whose blood test results show a minimum level of infection-fighting white blood cells (neutrophils) and clotting cells (platelets).
• People whose general health and ability to carry out daily activities meets a minimum score on a standard health scale (confirm with trial site).

For people with the sickle cell types known as hemoglobin SS or Sβ0 thalassemia, at least one of the following must also apply:

• A history of abnormal blood flow speed readings in brain arteries, measured on at least two separate occasions.
• Worsening of blood vessel disease in the brain related to SCD, seen on imaging.
• A history of a stroke or a significant silent stroke visible on a brain scan.
• A history of two or more episodes of Acute Chest Syndrome (a serious lung complication of SCD) at any point in life.
• A history of three or more severe SCD pain episodes that needed strong pain medication (by mouth or through a drip) at any point in life.
• A history of at least one hospital stay due to a complication of SCD (not counting hospital stays only for fever monitoring).
• A history of two or more episodes of prolonged, painful erections (priapism).
• Having received eight or more red blood cell transfusions in the past 12 months as a regular treatment.
• A history of at least two episodes of the spleen trapping red blood cells (splenic sequestration) that required a blood transfusion, or surgical removal of the spleen after at least one such episode.

For people with other sickle cell types (such as hemoglobin SC or Sβ+ thalassemia), at least one of the following must also apply:

• A history of a clinically significant stroke.
• Two or more episodes of Acute Chest Syndrome in the two years before joining the trial.
• Three or more severe SCD pain episodes needing strong pain medication in the one year before joining the trial.
• A history of at least one hospital stay for SCD pain or Acute Chest Syndrome while already taking hydroxyurea (a common SCD medicine).
• Two or more episodes of prolonged, painful erections lasting four or more hours or needing urgent medical care.
• Having received eight or more red blood cell transfusions in the past 12 months as a regular treatment.
• A history of at least two episodes of the spleen trapping red blood cells that required a blood transfusion, or surgical removal of the spleen after at least one such episode.

Who may not be able to join:

• People with a life expectancy of less than six months.
• People who are pregnant or breastfeeding.
• People with a bacterial, viral, or fungal infection that is not under control within the month before treatment begins, or who have a confirmed HIV diagnosis, or who have had Hepatitis B or C (confirm with trial site).
• People whose liver test results show certain elevated levels of bilirubin or liver enzymes beyond the limits set by the trial.
• People with a weakened heart function, as measured by a heart scan (ECHO), or with an uncontrolled irregular heartbeat.
• People whose kidneys are not filtering blood efficiently enough, based on a standard kidney function calculation.
• People with significantly reduced lung function, low oxygen levels at rest, or moderate-to-severe asthma that has not been fully controlled in the past two years.
• People who have a healthy, well-matched sibling donor available who does NOT have a blood type mismatch and does NOT carry the red blood cell markers the patient has developed antibodies against (meaning the specific donor mismatch that this trial is designed to address does not apply).

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