A Phase 1 interventional study of Placebo and CTX001 in Chronic Kidney Disease and Anemia, sponsored by Cajal Therapeutics Inc.. Not yet recruiting at 4 sites in Australia. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-10-28.
Sponsored by Cajal Therapeutics Inc. · Phase 1, Interventional, and Treatment
This is a Phase 1b, randomized, double-blinded, placebo-controlled study of CTX001 in participants with anemia and chronic kidney disease who are not on dialysis.
This is a Phase 1b, randomized, double-blinded, placebo-controlled study of CTX001 in participants with anemia and chronic kidney disease who are not on dialysis. The primary objective is to assess the preliminary safety and tolerability of CTX001 administered with iron in this patient population. Secondary objectives include assessment of concentrations of CTX001 in the blood at different dose levels, and the impact of administration of CTX001 with iron on blood iron levels and other hematologic parameters.
Up to 2 different doses of CTX001 are planned to be tested in cohorts of 12 participants each, for a total planned enrollment of up to 24 participants. An additional group of up to 12 participants may be enrolled to evaluate an additional dose level. Eligible participants will be randomly assigned in a 3:1 ratio to receive CTX001 or placebo, together with an oral iron supplement, for 29 days.
3,090 studies on the registry are indexed under Renal Insufficiency, Chronic; 698 are open to participants now.
This study's planned enrollment of 24 is below the median of 73 across 2,157 interventional studies indexed under Renal Insufficiency, Chronic.
Browse Renal Insufficiency, Chronic studies →Cajal Therapeutics Inc. is the lead sponsor of 2 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants receive CTX001 at the assigned cohort dose together with oral iron once daily for 29 days.
Drug: CTX001 · Drug: Ferrous Sulfate
Participants receive matching placebo together with oral iron once daily for 29 days.
Drug: Placebo · Drug: Ferrous Sulfate
Matching placebo administered orally once daily for 29 days.
CTX001 administered orally at the assigned cohort dose once daily for 29 days.
Ferrous sulfate administered orally once daily for 29 days in both treatment arms.
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Number and percentage of participants with at least one (TEAE). An adverse event (AE) is any untoward medical occurrence in a participant, whether or not considered related to the study intervention. An AE is considered a treatment-emergent AE (TEAE) if the AE begins or worsens after the first dose of study intervention and on or before the last study visit.
Time frame: From first administration of study intervention through the end of study participation, around Day 36.
Incidence of Serious TEAEs
Number and percentage of participants with at least one serious treatment-emergent adverse event (TEAE). 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 administration of study intervention through the end of study participation, around Day 36.
Incidence of Clinically Significant Changes in Physical Examination
The investigator assesses the clinical significance of changes in physical examination findings.
Time frame: From first administration of study intervention through the end of study participation, around Day 36.
Incidence of Clinically Significant Changes in Safety Laboratory Values
Safety laboratory evaluations include blood counts, serum chemistries, coagulation studies and urinalyses. Determination of clinical significance is based on investigator discretion.
Time frame: From first administration of study intervention through the end of study participation, around Day 36.
Incidence of Clinically Significant Changes in Vital Signs
Vital signs include heart rate, respiratory rate, blood pressure, and temperature. Determination of clinical significance is based on investigator discretion.
Time frame: From first administration of study intervention through the end of study participation, around Day 36
Incidence of Clinically Significant Changes in Electrocardiograms
Electrocardiograms are tests that measure the electrical activity of the heart. Determination of clinical significance is based on investigator discretion.
Time frame: From first administration of study intervention through the end of study participation, around Day 36
Maximum Change From Baseline in Hemoglobin
Maximum observed change from baseline in hemoglobin concentration.
Time frame: From first administration of study intervention through the end of study participation, around Day 36
Maximum Change From Baseline in Hematocrit
Maximum observed change from baseline in hematocrit.
Time frame: From first administration of study intervention through the end of study participation, around Day 36
Maximum Change from Baseline in Serum Iron
Time frame: Baseline through the end of the treatment period, around Day 29
Maximum Change from Baseline in Ferritin
Time frame: Baseline through the end of the treatment period, around Day 29
Maximum Change from Baseline in Transferrin Saturation
Time frame: Baseline through the end of the treatment period, around Day 29
Maximum Change from Baseline in Transferrin
Time frame: Baseline through the end of the treatment period, around Day 29.
Maximum Change from Baseline in Reticulocyte Count
Time frame: From first administration of study intervention through the end of study participation, around Day 36.
Maximum Change from Baseline in Red Blood Cell (RBC) Count
Time frame: From first administration of study intervention through the end of study participation, around Day 36
Maximum Change from Baseline in Mean Corpuscular Hemoglobin
Time frame: From first administration of study intervention through the end of study participation, around Day 36
Maximum Change From Baseline in Reticulocyte Hemoglobin Content
Time frame: From first administration of study intervention through the end of study participation, around Day 36
Plasma Concentrations of CTX001
Plasma concentrations of CTX001 by dose level over time.
Time frame: 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.
Maximum Observed Plasma Concentration of CTX001 (Cmax)
The highest observed plasma concentration of CTX001 following dosing.
Time frame: 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.
Time to Maximum Observed Plasma Concentration of CTX001 (Tmax)
Time from dosing to the maximum observed plasma concentration of CTX001.
Time frame: 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.
Area Under the Plasma Concentration-Time Curve of CTX001 From Time 0 to the Last Quantifiable Concentration (AUC0-t)
Area under the CTX001 plasma concentration-time curve from dosing to the time of the last quantifiable concentration.
Time frame: 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.
Area Under the Plasma Concentration-Time Curve of CTX001 Extrapolated to Infinity (AUC∞)
Area under the CTX001 plasma concentration-time curve from dosing extrapolated to infinity, where reliably estimable.
Time frame: 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.
Apparent Oral Clearance of CTX001 (CL/F)
Apparent oral clearance of CTX001, estimated from the administered dose and plasma concentration-time data, where reliably estimable.
Time frame: 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.
Terminal Elimination Half-Life of CTX001 (t½)
Time required for the plasma concentration of CTX001 to decrease by half during the terminal elimination phase, where reliably estimable.
Time frame: 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.
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Nov 2026; primary completion for Oct 2027, 11 months later.
Of 77 industry-sponsored phase 1 interventional studies under Renal Insufficiency, Chronic, half reached primary completion within 7 months of starting (the median), counting those still running; a quarter within 5 and three quarters within 16. This study's planned 11 months is above that.
Counting every sponsor, half of 109 studies reached it within 12 months; a quarter within 5 and three quarters within 27.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Exagamglogene autotemcel
Renal Insufficiency, Chronic →
Cajal Therapeutics Inc.