AnemiaEarly Phase 1NCT07577817
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.
A Early Phase 1 study testing CTX001 With and Without Iron, CTX001 With Iron and CTX001 Multiple Dose in people with Anemia and Iron Deficiencies, run by Cajal Therapeutics Inc. at 1 site in Australia.
Trial summary
This study is testing CTX001 for certain conditions where the body does not have enough available iron or has difficulty storing or moving iron properly. The purpose of this study is to investigate any side effects that may happen with CTX001, how CTX001 is absorbed by and processed in the body, and how CTX001 affects… Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Anemia, Iron Deficiencies, Chronic Kidney Disease
- What is being tested?
- CTX001 With and Without Iron, CTX001 With Iron and CTX001 Multiple Dose
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 72 participants
- Where
- 1 siteAustralia
- Study dates
- Jun 2026to 30 Dec 2026
- Phase
- Early Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Australia1 site, 1 recruiting
- Recruiting
Nucleus Network
Melbourne, Victoria, 3004
About this study
Official title: A Phase 1, 3-Part, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Oral Doses of CTX001 in Healthy Adult Participants.
This study is testing CTX001 for certain conditions where the body does not have enough available iron or has difficulty storing or moving iron properly. The purpose of this study is to investigate any side effects that may happen with CTX001, how CTX001 is absorbed by and processed in the body, and how CTX001 affects iron levels in the blood when administered with or without iron and/or food.
Read the detailed description
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.
- Conditions
- Anemia, Iron Deficiencies, Chronic Kidney Disease
- Being tested
- CTX001 With and Without Iron, CTX001 With Iron, CTX001 Multiple Dose, Placebo With and Without Iron, Placebo With Iron
- Keywords
- Red blood cells, Iron, Anemia, Chronic kidney disease
- Medical subject headings
- Anemia, Iron Deficiencies, Renal Insufficiency, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTX001 With and Without IronDrug
There are two treatment periods. In the first, CTX001 is administered as a single dose (tablet) by itself. In the second, CTX001 is administered as a single dose (tablet) with iron.
CTX001 With IronDrug
There are two treatment periods. In one, CTX001 is administered as a single dose (tablet) with iron (tablet) following a high fat, high calorie meal. In the other, CTX001 is administered as a single dose (tablet) with iron (tablet) under fasting conditions.
CTX001 Multiple DoseDrug
CTX001 (tablet) is administered daily for 7 consecutive days.
Placebo With and Without IronDrug
There are two treatment periods. In the first, placebo is administered as a single dose (tablet) by itself. In the second, placebo is administered as a single dose (tablet) with iron.
Placebo With IronDrug
There are two treatment periods. In one, placebo is administered as a single dose (tablet) with iron (tablet) following a high fat, high calorie meal. In the other, placebo is administered as a single dose (tablet) with iron (tablet) under fasting conditions.
Placebo Multiple DoseDrug
Placebo (tablet) is administered daily for 7 consecutive days.
| Study group | Role | What they receive |
|---|---|---|
| Single Ascending Dose (CTX001) | Experimental | Up to 5 sequential, single ascending dose cohorts. Receives: Drug: CTX001 With and Without Iron |
| Single Ascending Dose (Placebo) | Placebo comparator | Up to 5 sequential, single ascending dose cohorts Receives: Drug: Placebo With and Without Iron |
| Food Effect (CTX001) | Experimental | Up to 3 sequential, single ascending dose food effect cohorts Receives: Drug: CTX001 With Iron |
| Food Effect (Placebo) | Placebo comparator | Up to 3 sequential, single ascending dose food effect cohorts Receives: Drug: Placebo With Iron |
| Multiple Ascending Dose (CTX001) | Experimental | Up to 3 multiple ascending dose cohorts. Receives: Drug: CTX001 Multiple Dose |
| Multiple Ascending Dose (Placebo) | Placebo comparator | Up to 3 multiple ascending dose cohorts Receives: Drug: Placebo Multiple Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a participant, whether or not considered related to the study intervention. An AE was considered a treatment-emergent AE (TEAE) if the AE started after initial study drug administration and before the last study visit.
Measured: From first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Serious TEAEs
A serious AE (SAE) is defined as any AE that, at any dose, meets one or more of the following criteria: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital anomaly or birth defect; or any important medical event that may jeopardize the participant or may require medical intervention to prevent one of the outcomes listed above.
Measured: From first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Physical Examination
Abnormalities in physical examinations were based on investigator's discretion.
Measured: From the first dose of study drug through the last study visit; approximately 15 days.
Number of Participants with Clinically Significant Changes in Safety Laboratory Values
Safety laboratory evaluations included blood counts, serum chemistries, coagulation studies, and urinalyses. Determination of clinical significance was based on investigator discretion.
Measured: From first dose of study drug through last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Vital Signs
Vital signs included heart rate, respiratory rate, blood pressure, and temperature. Determination of clinical significance was based on investigator discretion.
Measured: From the first dose of study drug through the last study visit, approximately 15 days.
Number of Participants with Clinically Significant Changes in Electrocardiograms
Electrocardiograms are tests that measure the electrical activity of the heart. Determination of clinical significance was based on investigator discretion.
Measured: From the first dose of study drug through the last study visit, approximately 15 days.
Secondary outcomes (10)
Maximum Observed Plasma Concentration (Cmax) of CTX001
Measured: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Time to Maximum Observed Plasma Concentration (Tmax) of CTX001
Measured: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Area Under the Curve from Time Zero Extrapolated to Infinity (AUC0-inf) of CTX001
Measured: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Area Under the Curve From Time Zero to Time of Last Quantifiable Concentration (AUC0-t) of CTX001
Measured: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Apparent Clearance (CL/F) of CTX001
Measured: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Plasma Half-Life of CTX001
Measured: Pre-dose, 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 6 hours, 8 hours and 24 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Maximum Change in Serum Iron From Baseline
Measured: Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Maximum Change in Serum Transferrin From Baseline
Measured: Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Maximum Change in Serum Transferrin Saturation From Baseline
Measured: Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Maximum Change in Serum Ferritin From Baseline
Measured: Pre-dose, 30 minutes, 1 hour, 2 hours, 4 hours, and 8 hours after the Day 1 and Day 8 doses (SAD and Food Effect Cohorts) or the Day 1 and Day 7 doses (MAD Cohorts).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2026
- Study startJun 2026 (estimated)
- Primary completionOct 2026 (estimated)
- Study completion30 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cajal Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Aarthy Joseph, Dr, Principal investigator, Nucleus Network
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07577817
- Sponsor's protocol ID
- CTX001-101
- Status last verified
- May 2026
- First submitted
- 5 May 2026
- Last updated
- 12 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No