A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.

The study drug CTX001 is an investigational treatment for certain diseases where the body does not have enough iron in the bone marrow (the hollow inner parts of bones). Iron is an important part of red blood cells, which are made in the bone marrow. When there is not enough iron in the bone marrow, it can lead to anaemia. Conditions like chronic kidney disease, bone marrow diseases, intestinal diseases, and bleeding disorders all can cause anaemia due to low iron levels, or iron that gets trapped in other body parts like the spleen and cannot get to the bone marrow. Although anemia can sometimes be treated with iron pills or iron infusions, some patients cannot tolerate these treatments and others do not respond to them because either their intestines don't absorb the iron or because the body has improperly stored the iron where it can't be used to make red blood cells. CTX001 is a pill that is swallowed by mouth. Inside the body, CTX001 is expected to bind to iron, help the iron be absorbed by the intestine and help the iron move out of body parts where it might be stuck. If effective, CTX001 could be used to treat anemia in patients with chronic health conditions.

Inclusion criteria

• Capable of giving informed consent
• Agrees to use effective contraception
• Body Mass Index (BMI) between 18.0 and 32.0 kg/m2
• Healthy by medical evaluation and medical history
• Hematological parameters, serum iron, transferrin and ferritin are within normal range and transferrin saturation is within normal range and greater than or equal to 20%
• Can swallow tablets and has suitable venous access for blood sampling

Exclusion criteria

• Has dietary requirements that may be difficult to accommodate
• Is a regular user of cannabis or has a history of illicit drug abuse within 1 year
• Has a history of alcohol abuse or binge drinking within 6 months
• Is a regular user of tobacco or nicotine-containing products
• Unwilling or unable to comply with the lifestyle guidelines described in the protocol
• Has clinically significant history or evidence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, neurological, immunological, or psychiatric disorder(s) as determined by the Investigator
• Has any concurrent disease or condition or physical, psychological, mental, and/or social reason that, in the opinion of the Investigator, would make the participant unsuitable for participation in the clinical study
• Has received a blood transfusion within 1 year
• Has donated whole blood within 6 months or plasma within 30 days
• Requires prescription medication or regular use of non-prescription medication
• Is an employee of the Sponsor, the CRO, or of any organization or site(s) associated with this study, or any immediate family member who is in a dependent relationship with a study site employee who is involved in the conduct of the study