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

NCT07577817 | Not Yet Recruiting Early Phase 1

• 1 other identifier: CTX001-101
• Study Type: interventional
• Target: 72
• Locations: 1 country
• Sites: 1

Brief 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 iron levels in the blood when administered with or without iron and/or food.

Conditions

Anemia; Iron Deficiencies; Chronic Kidney Disease

Keywords

Red blood cells; Iron; Anemia; Chronic kidney disease

Outcome Measures

Primary Outcomes (6)

• 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.

  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.

  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.

  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.

  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.

  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.

  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

  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

  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

  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

  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

  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).

Study Arms (6)

Single Ascending Dose (CTX001)

EXPERIMENTAL

Up to 5 sequential, single ascending dose cohorts.

Drug: CTX001 With and Without Iron

Single Ascending Dose (Placebo)

PLACEBO COMPARATOR

Up to 5 sequential, single ascending dose cohorts

Drug: Placebo With and Without Iron

Food Effect (CTX001)

EXPERIMENTAL

Up to 3 sequential, single ascending dose food effect cohorts

Drug: CTX001 With Iron

Food Effect (Placebo)

PLACEBO COMPARATOR

Up to 3 sequential, single ascending dose food effect cohorts

Drug: Placebo With Iron

Multiple Ascending Dose (CTX001)

EXPERIMENTAL

Up to 3 multiple ascending dose cohorts.

Drug: CTX001 Multiple Dose

Multiple Ascending Dose (Placebo)

PLACEBO COMPARATOR

Up to 3 multiple ascending dose cohorts

Drug: Placebo Multiple Dose

Interventions

CTX001 With Iron DRUG

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.

Food Effect (CTX001)

Placebo With Iron DRUG

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.

Food Effect (Placebo)

CTX001 With and Without Iron DRUG

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.

Single Ascending Dose (CTX001)

CTX001 Multiple Dose DRUG

CTX001 (tablet) is administered daily for 7 consecutive days.

Multiple Ascending Dose (CTX001)

Placebo With and Without Iron DRUG

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.

Single Ascending Dose (Placebo)

Placebo Multiple Dose DRUG

Placebo (tablet) is administered daily for 7 consecutive days.

Multiple Ascending Dose (Placebo)

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Cajal Therapeutics Inc.: lead

Study Sites (1)

Nucleus Network

Melbourne, Victoria, 3004, Australia

Location

MeSH Terms

Conditions

Anemia; Iron Deficiencies; Renal Insufficiency, Chronic

Interventions

exagamglogene autotemcel; Iron

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Metals, Heavy; Elements; Inorganic Chemicals; Transition Elements; Metals

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2026
• First Posted: May 11, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): October 1, 2026
• Study Completion (Estimated): December 30, 2026
• Last Updated: May 11, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)