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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT07901933




Registration number
Help: Registration number
NCT07901933
Ethics application status
Help: Ethics application status
Date submitted
Help: Date submitted
22/10/2026
Date registered
Help: Date registered
28/10/2026
Date last updated
Help: Date last updated
28/10/2026

Titles & IDs
Public title
Study of CTX001 in Adults With Anemia Due to Non-Dialysis-Dependent Chronic Kidney Disease
Scientific title
A Phase 1b, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Assess the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of CTX001 in Adults With Anemia Due to Non-Dialysis-Dependent Chronic Kidney Disease
Additional trial identifiers [1] 0 0
CTX001-102
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
Chronic Kidney Disease 0 0
Anemia 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Blood 0 0 0 0
Anaemia

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
Treatment: Drugs - Placebo
Treatment: Drugs - CTX001
Treatment: Drugs - Ferrous Sulfate

Experimental: CTX001 plus oral iron - Participants receive CTX001 at the assigned cohort dose together with oral iron once daily for 29 days.

Placebo comparator: Placebo plus oral iron - Participants receive matching placebo together with oral iron once daily for 29 days.


Treatment: Drugs: Placebo
Matching placebo administered orally once daily for 29 days.

Treatment: Drugs: CTX001
CTX001 administered orally at the assigned cohort dose once daily for 29 days.

Treatment: Drugs: Ferrous Sulfate
Ferrous sulfate administered orally once daily for 29 days in both treatment arms.
Intervention code [1] 0 0
Treatment: Drugs
Description of the comparator or control
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
From first administration of study intervention through the end of study participation, around Day 36.
Primary outcome [2] 0 0
Incidence of Serious TEAEs
Timepoint [2] 0 0
From first administration of study intervention through the end of study participation, around Day 36.
Primary outcome [3] 0 0
Incidence of Clinically Significant Changes in Physical Examination
Timepoint [3] 0 0
From first administration of study intervention through the end of study participation, around Day 36.
Primary outcome [4] 0 0
Incidence of Clinically Significant Changes in Safety Laboratory Values
Timepoint [4] 0 0
From first administration of study intervention through the end of study participation, around Day 36.
Primary outcome [5] 0 0
Incidence of Clinically Significant Changes in Vital Signs
Timepoint [5] 0 0
From first administration of study intervention through the end of study participation, around Day 36
Primary outcome [6] 0 0
Incidence of Clinically Significant Changes in Electrocardiograms
Timepoint [6] 0 0
From first administration of study intervention through the end of study participation, around Day 36
Secondary outcome [1] 0 0
Maximum Change From Baseline in Hemoglobin
Timepoint [1] 0 0
From first administration of study intervention through the end of study participation, around Day 36
Secondary outcome [2] 0 0
Maximum Change From Baseline in Hematocrit
Timepoint [2] 0 0
From first administration of study intervention through the end of study participation, around Day 36
Secondary outcome [3] 0 0
Maximum Change from Baseline in Serum Iron
Timepoint [3] 0 0
Baseline through the end of the treatment period, around Day 29
Secondary outcome [4] 0 0
Maximum Change from Baseline in Ferritin
Timepoint [4] 0 0
Baseline through the end of the treatment period, around Day 29
Secondary outcome [5] 0 0
Maximum Change from Baseline in Transferrin Saturation
Timepoint [5] 0 0
Baseline through the end of the treatment period, around Day 29
Secondary outcome [6] 0 0
Maximum Change from Baseline in Transferrin
Timepoint [6] 0 0
Baseline through the end of the treatment period, around Day 29.
Secondary outcome [7] 0 0
Maximum Change from Baseline in Reticulocyte Count
Timepoint [7] 0 0
From first administration of study intervention through the end of study participation, around Day 36.
Secondary outcome [8] 0 0
Maximum Change from Baseline in Red Blood Cell (RBC) Count
Timepoint [8] 0 0
From first administration of study intervention through the end of study participation, around Day 36
Secondary outcome [9] 0 0
Maximum Change from Baseline in Mean Corpuscular Hemoglobin
Timepoint [9] 0 0
From first administration of study intervention through the end of study participation, around Day 36
Secondary outcome [10] 0 0
Maximum Change From Baseline in Reticulocyte Hemoglobin Content
Timepoint [10] 0 0
From first administration of study intervention through the end of study participation, around Day 36
Secondary outcome [11] 0 0
Plasma Concentrations of CTX001
Timepoint [11] 0 0
Pre-dose through the end of the pharmacokinetic sampling period. Days 1, 15, and 29; pre-dose and 30 minutes, 1 hour, 2 hours, 4 hours, and 6 hours post-dose.
Secondary outcome [12] 0 0
Maximum Observed Plasma Concentration of CTX001 (Cmax)
Timepoint [12] 0 0
Pre-dose through the end of the pharmacokinetic sampling period. Days 1, 15, and 29; pre-dose and 30 minutes, 1 hour, 2 hours, 4 hours, and 6 hours post-dose.
Secondary outcome [13] 0 0
Time to Maximum Observed Plasma Concentration of CTX001 (Tmax)
Timepoint [13] 0 0
Pre-dose through the end of the pharmacokinetic sampling period. Days 1, 15, and 29; pre-dose and 30 minutes, 1 hour, 2 hours, 4 hours, and 6 hours post-dose.
Secondary outcome [14] 0 0
Area Under the Plasma Concentration-Time Curve of CTX001 From Time 0 to the Last Quantifiable Concentration (AUC0-t)
Timepoint [14] 0 0
Pre-dose through the end of the pharmacokinetic sampling period. Days 1, 15, and 29; pre-dose and 30 minutes, 1 hour, 2 hours, 4 hours, and 6 hours post-dose.
Secondary outcome [15] 0 0
Area Under the Plasma Concentration-Time Curve of CTX001 Extrapolated to Infinity (AUC8)
Timepoint [15] 0 0
Pre-dose through the end of the pharmacokinetic sampling period. Days 1, 15, and 29; pre-dose and 30 minutes, 1 hour, 2 hours, 4 hours, and 6 hours post-dose.
Secondary outcome [16] 0 0
Apparent Oral Clearance of CTX001 (CL/F)
Timepoint [16] 0 0
Pre-dose through the end of the pharmacokinetic sampling period. Days 1, 15, and 29; pre-dose and 30 minutes, 1 hour, 2 hours, 4 hours, and 6 hours post-dose.
Secondary outcome [17] 0 0
Terminal Elimination Half-Life of CTX001 (t½)
Timepoint [17] 0 0
Pre-dose through the end of the pharmacokinetic sampling period. Days 1, 15, and 29; pre-dose and 30 minutes, 1 hour, 2 hours, 4 hours, and 6 hours post-dose.

Eligibility
Key inclusion criteria
* Between 18 and 75 years of age
* Body mass index (BMI) between 18 and 35 kg/m2
* Estimated GFR =15 and <60 mL/min/1.73 m2
* CKD for at least 3 months
* Not currently receiving dialysis and not expected to start dialysis within 3 months of screening.
* Hemoglobin =9.0 g/dL and =11.5 g/dL
* Transferrin saturation (TSAT) =20%
* Not folate or vitamin B12 deficient
* ALT and AST =1.5 x ULN and TBL =1.5 x ULN
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
* Anemia due to a cause other than CKD, presence of active bleeding, or recent significant blood loss
* Any hemoglobinopathy, bone marrow pathology or hematological malignancy, including, but not limited to sickle cell disease, thalassemia, myelodysplastic syndrome, bone marrow fibrosis, red cell aplasia
* Known history of hemochromatosis or other iron-overload disorder, or ferritin >700 ng/mL
* RBC transfusion, intravenous iron, or ESA use within 12 weeks prior to screening
* Receiving an SGLT2 inhibitor on a dose that has not been stable for at least 12 weeks prior to screening.
* Pregnant or breastfeeding.
* Is unable or unwilling to comply with protocol-specified sun protection and contraception requirements.
* Has any disorder that would interfere with the absorption, distribution, metabolism, or excretion of drugs.
* Has a 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/11/2026
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2027
Actual
Sample size
Target
24
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,WA
Recruitment hospital [1] 0 0
Renal Research Gosford - Gosford
Recruitment hospital [2] 0 0
Royal North Shore Hospital - St Leonards
Recruitment hospital [3] 0 0
AOVO Clinical Trials - Southport
Recruitment hospital [4] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
2250 - Gosford
Recruitment postcode(s) [2] 0 0
2065 - St Leonards
Recruitment postcode(s) [3] 0 0
4215 - Southport
Recruitment postcode(s) [4] 0 0
6000 - Perth

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Cajal Therapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
A/Prof. Brendon Neuen
Address 0 0
Country 0 0
Phone 0 0
+61 2 9462 9655
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT07901933