Phase 1 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:

• You are 18 years old or older at the time of signing the consent form
• You have been diagnosed with sickle cell disease, specifically the HbSC or HbSS type
• You have experienced at least one of the following sickle cell-related complications: between 1 and 10 pain crises in the past 12 months, any history of eye damage, silent brain injury, bone tissue death, or hearing loss related to sickle cell; or at least one episode of a prolonged painful erection, liver crisis, spleen crisis, or spleen injury in the last 12 months
• Your blood hemoglobin level (a measure of red blood cells) is at least 7.0 g/dL at screening (the first two participants must have a level of at least 9.0 g/dL)
• Your alpha globin gene test (a genetic test related to sickle cell) comes back normal
• Your reticulocyte count (a measure of young red blood cells) is above a certain threshold at screening (confirm with trial site)
• Your iron levels, measured in two specific ways, fall within required ranges at screening (confirm with trial site)
• If you are taking hydroxyurea, L-glutamine, or crizanlizumab, your dose has been stable for at least 2 months before screening and is not expected to change during the study
• If you are male and not vasectomised, you and your female partner(s) agree to use two forms of highly effective birth control from your first dose until 120 days after your last dose, and you agree not to donate sperm during the study
• If you are female, you are either past menopause, have had surgery that means you cannot become pregnant, or you agree to use at least one highly effective form of birth control from day one of the study until at least 120 days after your last dose
• If you are female and could become pregnant, you have a negative pregnancy test before starting the study
• You are able to understand what the study involves and can give written consent
• You are able to follow all study procedures throughout the trial

Who may not be able to join:

• You are currently receiving regular blood transfusions or blood removal (phlebotomy), or have had either procedure within the past 60 days
• You have been hospitalised for a sickle cell pain crisis or related complication within the last 14 days
• You have a significant active infection caused by bacteria, fungus, parasites, or a virus
• You have active HIV, hepatitis B, or hepatitis C
• Your kidneys are not functioning well enough, based on a specific blood test result at screening (confirm with trial site)
• Your liver is not functioning well enough, based on a specific blood test result (confirm with trial site)
• You have had an acute chest syndrome (a serious sickle cell lung complication) in the last 6 months
• You have previously had, or are planning to have, a stem cell transplant or gene therapy
• You have had unstable or worsening heart or lung disease within the last 6 months
• You have had a serious cancer diagnosis within the last 5 years (some exceptions may apply — confirm with trial site)
• You have had major surgery within 8 weeks before screening, or have not fully recovered from a previous surgery
• You have a known allergy to any ingredient in the study drug, or a history of a severe allergic reaction to any food or medicine
• You have a history of alcohol dependence or excessive alcohol use, as assessed by the study doctor
• You have another medical, psychiatric, or lab finding that the study doctor believes would put you at unacceptable risk
• You have a major psychiatric condition that has worsened or required hospitalisation in the last 6 months, or are taking a medication that could interfere with study results
• You are pregnant or breastfeeding
• You have taken part in another clinical trial involving an experimental treatment or device within the last 30 days
• You have a history of a mini-stroke (transient ischaemic attack) or stroke — this may be considered on a case-by-case basis in consultation with the study sponsor (confirm with trial site)

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