CTX001: a pill to help iron move where it’s needed in healthy adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is an early-phase, first-in-human study to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of CTX001 in healthy adults. Participants will receive CTX001 or placebo in various dosing regimens, including with and without iron, and with or without food, to see how the drug is absorbed, distributed, and tolerated. The study aims to understand whether CTX001 can increase usable iron and support red blood cell production while identifying any potential side effects. Eligibility requires good general health, certain iron parameters within normal ranges, and the ability to comply with study requirements. The lead sponsor is Cajal Therapeutics, with recruitment at Nucleus Network in Melbourne, Australia.

Why it matters

Who can join: the full rules

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

Where it's running

• Nucleus Network
  Melbourne, Victoria, Australia Recruiting

Study contacts

• Principal investigator: Aarthy Joseph, Dr — Nucleus Network
• Study coordinator: Anthony Johnson
• Email: a.johnson@nucleusnetwork.com.au
• Phone: +61 07 3707 2787

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.