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 who have signed an informed consent form agreeing to participate.
• People aged 14 to 55 years old.
• People who have tested negative for Hepatitis B, Hepatitis C, HIV, and COVID-19 (those who test positive for COVID-19 at screening may be re-tested at least four weeks later, as long as they have no symptoms and test negative at that point).
• People who have not smoked or vaped, including tobacco or marijuana products, in the three months before screening.
• People aged 18 and over who weigh between 45 kg and 110 kg and have a BMI under 30; people aged 14 to 17 who weigh more than 35 kg and have a BMI under 30.
• People who have a confirmed diagnosis of Sickle Cell Disease (Hb-SS type or Sβ0 type), with no more than 10 pain crises per year over the past two years, and who have had at least one of the following: acute chest syndrome, stroke, priapism, bone damage from reduced blood supply (avascular necrosis), or splenic sequestration.
• People who may or may not be taking hydroxyurea for their sickle cell disease — if taking it, the dose must have been stable for at least one month before joining the study, as directed by their doctor.
• People whose heart is functioning normally, as confirmed by tests including an ECG, physical check, and blood tests.
• People with a carboxyhemoglobin level (a measure of a certain substance in the blood) of 3.5% or below before the first dose (confirm with trial site).
• People with no significant health concerns identified through a full medical review, physical examination, blood pressure and heart rate checks, ECG, and blood tests, as judged by the study doctor.
• People with blood test results within acceptable ranges, including haemoglobin above 6 g/dL, white blood cell count above 7,000/uL, neutrophil count above 3,500/uL, and platelet count above 80,000/uL, with results shown to be stable between the screening and start of the study.
• People who test negative for pregnancy (for females).
• People who are willing to use a highly effective form of contraception throughout the study and for 30 days after it ends, where applicable.

Who may not be able to join:

• People who have a blood disorder other than Sickle Cell Disease or S/beta 0 thalassaemia.
• People whose heart shows signs of not functioning properly, based on clinical checks, ECG, or blood tests, as judged by the study doctor.
• People who have had an acute chest syndrome or pain crisis within the 21 days before the first dose, or who show signs of an oncoming pain crisis immediately before starting the study drug.
• People who currently smoke.
• People who have had a significant illness or surgery unrelated to sickle cell disease within the three months before dosing.
• People who have had a blood transfusion within six weeks before the first dose.
• People who have received a live vaccine within 28 days before the first dose.
• People who have had a fever or infectious illness within 14 days before dosing.
• People who are pregnant or breastfeeding (for females).
• People who have gained or lost more than 5 kg in the three months before dosing.
• People with a history of alcohol misuse or who have regularly consumed more than 14 units of alcohol per week in the six months before dosing (where one unit equals approximately 150 mL of wine, 360 mL of beer, or 45 mL of spirits).
• People with a history of significant kidney problems, where kidney filtration capacity is below a certain level (confirm with trial site).
• People with a history of lung infiltrates or pneumonia within six months before dosing, or a lung or airway infection within two weeks before dosing.
• People who use supplemental oxygen at home.
• People with a history of cancer, except for certain treated skin cancers more than one year prior.
• People with a history of heart disease.
• People with a history of drug misuse, including the use of opioid medications for reasons other than pain relief (use for pain control is permitted).
• People who have used certain medications — including voxelotor or crizanlizumab — or other prescription drugs not related to sickle cell disease, within 7 days or 5 half-lives before dosing (whichever is longer); herbal and vitamin supplements (except L-glutamine) must have been stopped at least 14 days before dosing.
• People who are unwilling or unable to follow the requirements of the study.
• People who have taken part in another clinical drug trial within the past 30 days or five half-lives of that drug, whichever is longer.
• People with significant abnormalities on their ECG or vital signs — including blood pressure, heart rate, or heart rhythm outside specified ranges — at screening or at the start of the study, as determined by the study doctor (confirm with trial site).
• People with any other serious medical, psychiatric, or laboratory condition that the study doctor believes would make participation unsafe or affect the reliability of the results.
• People with any other illness or condition that the study doctor believes would make them unsuitable for the trial.
• People with a known allergy to any ingredient in the study drug.

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