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:

• Adults aged 18 or older
• People who have been diagnosed with homozygous SS sickle cell disease or S-β0 Thalassemia (specific types of sickle cell disease confirmed by a doctor)
• People who were unable to tolerate a medication called hydroxyurea, chose not to take it, or did not respond to it
• People whose sickle cell disease has caused significant problems, defined as at least one of the following: two or more hospital admissions in the past 12 months due to sickle cell complications; three or more painful crises in the past 12 months lasting four or more hours and requiring a visit to a medical facility for strong pain relief; a history of recurring leg ulcers; a history of Acute Chest Syndrome (a serious lung complication) within the past five years; a history of a prolonged, painful erection (priapism) requiring medical treatment within the past two years; or a history of stroke, as long as the person is not currently on a regular blood transfusion programme
• People whose thyroid levels are in a normal range (those already taking thyroid hormone supplements for an underactive thyroid may still be eligible)
• People who are able and willing to provide written consent to take part in the study before any study procedures begin

Who may not be able to join:

• People who have used certain medications that can increase a specific type of haemoglobin (including hydroxyurea, butyrates, decitabine, 5-azacytidine, certain immune-modifying drugs, or erythropoietin) within the past 60 days
• People who have had a severe sickle cell pain crisis in the past two weeks that required treatment with injected or intravenous medication
• People with stomach or bowel conditions that could significantly affect how the study drug is absorbed (such as active ulcers, uncontrolled nausea, vomiting, diarrhoea, malabsorption conditions, or prior bowel surgery removing part of the small intestine)
• People on a regular ongoing blood transfusion programme, or whose blood tests show a high level of a particular type of haemoglobin (haemoglobin A above 20%) from a recent transfusion
• People whose blood test results at the screening visit fall outside acceptable ranges for measures including white blood cell count, haemoglobin level, platelet count, kidney function, liver enzymes, bilirubin levels, protein levels, and certain minerals such as potassium, calcium, magnesium, and phosphorus (confirm specific values with trial site)
• People with certain heart conditions, including significantly reduced heart pumping function, specific abnormal heart rhythms, a pacemaker, inherited long QT syndrome, a history of dangerous fast heart rhythms, unstable irregular heartbeat, an unusually slow resting heart rate (below 50 beats per minute), an abnormal reading on a heart tracing test (ECG), certain electrical conduction problems in the heart, chest pain (angina) within the past three months, a heart attack within the past three months, or other significant heart disease such as uncontrolled high blood pressure or heart failure
• People with other serious uncontrolled medical conditions such as uncontrolled diabetes, active or uncontrolled infection, or significant lung disease
• People who are currently taking part in another clinical trial or who have taken a study drug within the past 60 days
• People who have had major surgery within the past two weeks or who have not yet recovered from a recent surgery
• Women who are pregnant, breastfeeding, or who are of childbearing potential and are not willing to use two forms of contraception (including at least one barrier method) during the study and for three months afterwards; women of childbearing potential will also be required to have negative pregnancy tests before starting treatment
• Male participants whose sexual partners could become pregnant, if those partners are not using double contraception during the study and for three months after treatment ends; male participants must also agree to use a condom during any sexual contact with a partner who could become pregnant during and after treatment
• People with a known diagnosis of HIV, Hepatitis B, or active Hepatitis C
• People who have had a cancer diagnosis within the past five years (except for certain limited skin cancers or cervical cancer in situ)
• People with a significant history of not following medical treatment plans, or who are unwilling or unable to follow the study requirements
• People who are currently taking certain medications that are not allowed during the study and who cannot stop or switch those medications before joining (confirm specific medications with trial site)

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