A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CTX001 in Healthy Adults.
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age18-55
SponsorCajal Therapeutics Inc.
About this trial
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.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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
Disqualifiers
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
Trial design
Design model
Sequential
Treatments tested in this trial
CTX001 With and Without Iron
DrugThere 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 Iron
DrugThere 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 Dose
DrugCTX001 (tablet) is administered daily for 7 consecutive days.
Placebo With and Without Iron
DrugThere 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 Iron
DrugThere 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 Dose
DrugPlacebo (tablet) is administered daily for 7 consecutive days.
Treatment groups
72 Participants
are divided into 6 treatment groups6
Treatment groupsSee each treatment group below.
Group A: Single Ascending Dose (CTX001)Experimental treatment 1 intervention
Group B: Single Ascending Dose (Placebo)Placebo comparator 1 intervention
Group C: Food Effect (CTX001)Experimental treatment 1 intervention
Group D: Food Effect (Placebo)Placebo comparator 1 intervention
Group E: Multiple Ascending Dose (CTX001)Experimental treatment 1 intervention
Group F: Multiple Ascending Dose (Placebo)Placebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
From first dose of study drug through the last study visit, approximately 15 days.2
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.
Time frame
From first dose of study drug through the last study visit, approximately 15 days.3
Number of Participants with Clinically Significant Changes in Physical Examination
Abnormalities in physical examinations were based on investigator's discretion.
Time frame
From the first dose of study drug through the last study visit; approximately 15 days.4
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.
Time frame
From first dose of study drug through last study visit, approximately 15 days.Secondary outcomes
1
Maximum Observed Plasma Concentration (Cmax) of CTX001
Time frame
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).2
Time to Maximum Observed Plasma Concentration (Tmax) of CTX001
Time frame
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).3
Area Under the Curve from Time Zero Extrapolated to Infinity (AUC0-inf) of CTX001
Time frame
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).4
Area Under the Curve From Time Zero to Time of Last Quantifiable Concentration (AUC0-t) of CTX001
Time frame
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).Sponsors and contacts
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Source ClinicalTrials.gov