> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/trial/study-of-ct-01-as-monotherapy-and-combination-therapy-in-da65959b-7b25-4571-a176-45194e34bee9 > Record changed: 2026-11-04 # Study of CT-01 as Monotherapy and Combination Therapy in Subjects With Intermediate or Advanced Hepatocellular Carcinoma NCT06994572 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) Phase Phase 1 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) Recruitment status Not yet recruiting Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) Participants 141 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) Lead sponsor Captor Therapeutics S.A. Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) Start date 2025-05-26 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Title and summary **Summary** This is a Phase 1, open-label, multicenter, dose-escalation and dose-expansion study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of CT-01, administered either as monotherapy or in combination with everolimus. The study population includes subjects with intermediate or advanced hepatocellular carcinoma (HCC) who have progressed on, or are intolerant to, at least one prior line of systemic treatment. All available standard-of-care therapies should have been received, if deemed appropriate by the investigator (unless contraindicated or considered inappropriate by the treating physician). Eligible subjects are classified as Barcelona Clinic Liver Cancer (BCLC) stage B or C and must not be amenable to curative treatment approaches. Only subjects with preserved liver function (Child-Pugh Class A, score 5-6) at screening are eligible. Approximately 141 participants will be enrolled across 20 sites in Europe (France, Spain, and Germany). Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Short title** Study of CT-01 as Monotherapy and Combination Therapy in Subjects With Intermediate or Advanced Hepatocellular Carcinoma Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Official title** A Phase 1, Open-Label, Dose Escalation, and Dose Expansion Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of CT-01 as Monotherapy and Combination Therapy With Everolimus in Subjects With Intermediate or Advanced Hepatocellular Carcinoma (BCLC Stage B or C) With Preserved Liver Function (Child-Pugh Class A) Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Status and phase **Control arm** Open label Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Registry reports results** No Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Listing at source** Listed Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Recruitment status** Not yet recruiting Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Phase** Phase 1 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Study type** Interventional Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Dates **Completion date** 2030-04-01 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **First posted** 2025-05-29 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Registry last updated** 2025-05-29 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Primary completion date** 2029-04-16 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Start date** 2025-05-26 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Size and sites **Participants** 141 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Participant count basis** Estimated Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Planned participants** 141 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Protocol amendments** 0 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Sponsor and oversight **Data monitoring committee** Yes Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Registry sponsor category** Industry Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Lead sponsor** Captor Therapeutics S.A. Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Source registry** ClinicalTrials.gov Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Identifiers **Registry ID** NCT06994572 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **ClinicalTrials.gov ID** NCT06994572 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) **Registry record** Open registry record Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Study design **Allocation** Not randomised **Model** Sequential **Purpose** Treatment **Masking** No masking Sources for study design Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Outcomes ### Primary outcomes 1. Number of participants with treatment-emergent adverse events (TEAEs) during CT-01 monotherapy Timeframe: Up to 12 months Safety and tolerability of CT-01 will be evaluated by monitoring the incidence of TEAEs, SAEs, AESIs, and TEAEs leading to study discontinuation or death. Unit of Measure: Number, nature, sverity and relation plausibility of AE 2. Maximum tolerated dose (MTDm) of CT-01 monotherapy Timeframe: Up to 12 months The MTDm will be determined based on the incidence and type of dose-limiting toxicities (DLTs) during Cycle 1 of CT-01 monotherapy. Unit of Measure: mg/day 3. Number of participants with treatment-emergent adverse events (TEAEs) during CT-01 and everolimus combination therapy Timeframe: Up to 12 months Safety and tolerability will be assessed through monitoring of TEAEs, SAEs, AESIs, and TEAEs leading to study discontinuation or death during combination therapy. Unit of Measure: Number, nature, sverity and relation plausibility of A 4. Maximum tolerated dose (MTDc) of CT-01 in combination with everolimus Timeframe: Up to 12 months The MTDc will be determined based on the incidence of DLTs during Cycle 1 of the combination therapy. Unit of Measure: mg/day ### Secondary outcomes 1. Objective response rate (ORR) per RECIST v1.1 and mRECIST for CT-01 monotherapy Timeframe: Up to 12 months Proportion of participants achieving complete or partial response as per RECIST v1.1 and mRECIST. Unit of Measure: Percentage of participants 2. Objective response rate (ORR) per RECIST v1.1 and mRECIST for CT-01 and everolimus Timeframe: Up to 12 months Proportion of participants achieving complete or partial response during combination therapy. Unit of Measure: Percentage of participants 3. Maximum plasma concentration (Cmax) Timeframe: Up to 12 months Cmax will be determined for CT-01 using non-compartmental analysis. Unit of Measure: ng/mL 4. Area under the concentration-time curve (AUC) Timeframe: Up to 12 months AUC will be calculated for active metabolite of CT-01 using non-compartmental methods (AUC₀-t and AUC₀-inf). Unit of Measure: ng·h/mL 5. Time to maximum concentration (Tmax) Timeframe: Up to 12 months Tmax will be recorded as the time to reach Cmax for CT-01\. Unit of Measure: Hours 6. Change from baseline in MELD-Na score during combination therapy Timeframe: Up to 12 months Change in MELD-Na score (range: typically 6 to 40; higher scores indicate worse liver function) from baseline during CT-01 and everolimus treatment. Unit of Measure: score 7. Change from baseline in Child-Pugh score during combination therapy Timeframe: Up to 12 months Change in Child-Pugh score (range: 5-15; higher scores indicate worse liver function) from baseline during CT-01 and everolimus treatment. Unit of Measure: score 8. Change from baseline in Albumin-Bilirubin (ALBI) score during combination therapy Timeframe: Up to 12 months Change from baseline in ALBI score will be evaluated during CT-01 and everolimus combination therapy. The ALBI score is calculated using serum bilirubin and albumin. Changes from baseline will be summarized by time point. Higher scores indicate worse liver function. Unit of Measure: score 9. Change from baseline in lipid profile during combination therapy Timeframe: Up to 12 months Change in serum lipid parameters (total cholesterol, LDL, HDL, triglycerides) from baseline during CT-01 and everolimus treatment. Unit of measure: mmol/L 10. Change from baseline in serum glucose levels during combination therapy Timeframe: Up to 12 months Change in blood glucose concentration from baseline during CT-01 and everolimus treatment. Unit of Measure: mmol/L 11. Change from baseline in serum alpha-fetoprotein (AFP) levels during combination therapy Timeframe: Up to 12 months Change in serum AFP concentration from baseline during treatment with CT-01 in combination with everolimus. AFP is a tumor marker used in hepatocellular carcinoma. Unit of Measure: ng/mL 12. Terminal half-life (T1/2) Timeframe: Up to 12 months T1/2 will be estimated for active metabolite of CT-01 from the log-linear terminal phase of the concentration-time curve. Unit of Measure: Hours 13. Title: Maximum plasma concentration (Cmax) Timeframe: Up to 12 months Cmax will be determined for CT-01 in combination with everolimus using non-compartmental analysis. Unit of Measure: ng/mL 14. Change from baseline in serum glucose levels during monotherapy Timeframe: Up to 12 months Change in blood glucose concentration from baseline during CT-01 monotherapy. Unit of Measure: mmol/L 15. Area under the concentration-time curve (AUC) Timeframe: Up to 12 months AUC for active metabolite of CT-01 in combination with everolimus will be calculated using non-compartmental methods. ng·h/mL 16. Time to maximum concentration (Tmax) Timeframe: Up to 12 months Tmax will be recorded for CT-01 when administered with everolimus. Unit of Measure: Hours 17. Terminal half-life (T1/2) Timeframe: Up to 12 months T1/2 will be estimated for active metabolite of CT-01 in combination therapy using non-compartmental analysis. Unit of Measure: Hours 18. Change from baseline in lipid profile during monotherapy Timeframe: Up to 12 months Change in serum lipid parameters (total cholesterol, LDL, HDL, triglycerides) from baseline during CT-01 monotherapy. Unit of Measure: mmol/L 19. Change from baseline in Albumin-Bilirubin (ALBI) score during monotherapy Timeframe: Up to 12 months Change from baseline in ALBI score will be evaluated during CT-01 monotherapy. The ALBI score is calculated using serum bilirubin and albumin. Changes from baseline will be summarized by time point. Higher scores indicate worse liver function. Unit of Measure: score 20. Change from baseline in Child-Pugh score during monotherapy Timeframe: Up to 12 months Change in Child-Pugh score (range: 5-15; higher scores indicate worse liver function) from baseline during CT-01 monotherapy. Unit of Measure: score 21. Change from baseline in MELD-Na score during monotherapy Timeframe: Up to 12 months Change in MELD-Na score (range: typically 6 to 40; higher scores indicate worse liver function) from baseline during CT-01 monotherapy. Unit of Measure: score 22. Change from baseline in serum alpha-fetoprotein (AFP) levels during monotherapy Timeframe: Up to 12 months Change in serum AFP concentration from baseline during CT-01 monotherapy. AFP is a tumor marker used in hepatocellular carcinoma. Unit of Measure: ng/mL Sources for outcomes Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Eligibility **Sex** All **Age groups** Adults, Older adults **Minimum age** 18 Years **Healthy volunteers** No ### Inclusion criteria - Able to understand and willing to provide informed consent and able to comply with the study procedures and restrictions. - Are ≥18 years of age and of any gender at the time of screening. - Have confirmed histological and/or radiological diagnosis of HCC with a Child-Pugh Class A score of 5 to 6 points within 7 days of first dose of study drug. - Has BCLC stage B and are ineligible for or are refractory to locoregional therapy and not amenable to a curative treatment approach, or BCLC stage C and are not amenable to a curative treatment approach. - Have progressed on or are intolerant to ≥1 prior systemic standard of care treatment. All available standard of care treatments should have been received as prior systemic treatment if deemed appropriate by the investigator (unless such treatments are deemed contraindicated or inappropriate by the treating physician). - Have Eastern Cooperative Oncology Group (ECOG) score ≤1. - Have at least one measurable disease lesion per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 and/or modified RECIST (mRECIST), with a minimum of 1 target lesion in the liver at screening. - Female subjects may be enrolled if they refrain from donating eggs (ova, oocytes) for the purpose of reproduction during the treatment period and for 6 months after the treatment period and if they are: 1. Documented to be surgically sterile or postmenopausal, or 2. Practicing true abstinence for at least 28 days prior to investigational medical product (IMP) administration and for 6 months after the treatment period and having a negative pregnancy test prior dosing, or 3. Willing and able to comply with two forms of contraception methods, including one physical barrier (condom or diaphragm) plus another method, such as adequate hormonal method (eg, contraceptive implants, injectables, oral contraceptives) or nonhormonal methods (eg, intrauterine device, spermicidal) during and for 6 months after the treatment period and having a negative pregnancy test prior dosing. Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. - Male subjects with female partners of childbearing potential may be enrolled if they refrain from donating sperm during the treatment period and for 6 months after the last IMP administration and if they are: 1. Documented to be surgically sterile (vasectomy), or 2. Practicing true abstinence during the treatment period and for 6 months after the last IMP administration, or 3. Willing and able to comply with two forms of contraception methods, including one physical barrier (condom or diaphragm) during the treatment period and for 6 months after the last IMP administration. Contraceptive use by men should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies ### Exclusion criteria - Have not recovered from toxicities to baseline or Grade ≤1 by CTCAE version 5.0 from prior anticancer therapy, except alopecia and sub-optimally treated nausea, vomiting, or diarrhea. - Have a history of liver transplant. - Any other active malignancy at time of screening or diagnosis of another malignancy within 2 years before screening that requires active treatment, except for superficial skin cancers, or localized, low grade tumors deemed cured and not treated with systemic therapy. - Have central nervous system (CNS) metastases or any other Grade >2 CNS disorder. Subjects with CNS metastases who are stable on their treatment since a minimum of 4 weeks prior to screening are eligible. - Have recent esophageal or gastric variceal bleeding within 4 weeks prior to screening. Subjects with treated, stable, or small varices without bleeding within the 4 weeks prior to screening are eligible. - Have a history of symptomatic ascites requiring paracentesis within the past 3 months prior to screening. - Have a prior diagnosis of rhabdomyolysis from any cause. - Subjects with known retinal disease (eg, diabetic retinopathy, macular degeneration, retinal detachment/tear, etc). - Have a history of clinically significant cytokine release syndrome as assessed by the treating physician, clinically significant hypotension (defined as a blood pressure \[BP\] consistently <90/60 mmHg and/or a BP associated with symptoms of hypotension (dizziness, fainting, orthostatic hypotension \[systolic BP drop of >20 mmHg or diastolic BP drop of >10 mmHg on orthostasis\]), symptomatic hypocalcemia and/or a calcium level <1.5 mmol/L. - Have a history of pneumonitis or pericarditis. - Subjects who are not, in the opinion of the investigator, willing or able to comply with the protocol or who present a contraindication to any study procedure. - Have inadequate hematologic and end-organ function, defined as follows: - Hemoglobin <8.5 g/dL - Absolute neutrophil count <1,500 per mm3 - Platelet count <75,000 per mm3 - Total bilirubin >2 × ULN for the designated testing laboratory - AST or ALT >5 × ULN - Serum creatinine >1.5 × ULN - International normalized ratio (INR) >1.7 × ULN - Glomerular filtration rate (GFR) < 50 mL/min - Have imaging findings corresponding to HCC with ≥50% liver occupation, or clear invasion into the bile duct. - Have an active infection with the hepatitis B virus (HBV) or hepatitis C virus (HCV). 1. Patients with acute hepatitis B infection are excluded. Acute Hepatitis B is defined based on the following serology profile: positive hepatitis B surface antigen (HBsAg positive), positive total hepatitis B core antibody (anti-HBc positive), IgM antibody positive to hepatitis B core antigen (IgM anti-HBc positive), and hepatitis B surface antibody negative (anti-HBs negative) test at screening. 2. Patients with a past or resolved HBV infection (defined as HBsAg positive and anti-HBc positive) are eligible. 3. Patients positive for HCV antibody are eligible only if PCR is negative for HCV ribonucleic acid (RNA). 4. Chronic HBV (HBsAg positive, undetectable or low HBV DNA, and normal ALT) is eligible if patients are receiving direct-acting antiviral treatment. 5. Patients with resolved hepatitis C infection are eligible. This is defined as undetectable or unquantifiable HCV RNA 12 weeks or longer after treatment completion (defined as a sustained virological response). 6. Patients with untreated HCV infection or have not completed treatment for HCV infection. 7. Patients with treated HCV infection but with a HCV viral load above the level of quantification. - Have a history of human immunodeficiency virus (HIV) infection. - Have life expectancy shorter than 3 months. - Have received any systemic anticancer treatment or any investigational treatment within 14 days or at least 5 half-lives of the treatment, whichever is shorter, prior to screening. - Are unable to take oral medications. - Have a known malabsorption syndrome. - Applicable to subjects enrolling in Part 2 only: - Are currently receiving treatment with the cytochrome P450 (CYP) 3A4 inhibitors (eg, azoles, clarithromycin, or grapefruit), CYP3A4 inducers (eg, rifampin, carbamazepine, phenytoin, or St. John's wort), or p-glycoprotein (P-gp) inhibitors or inducers (eg, verapamil, quinidine, amiodarone, diltiazem, erythromycin, cyclosporine, ketoconazole, ritonavir, or tamoxifen). Subjects able to receive modified dose of the P-gp or CYP3A4 inhibitors based on everolimus drug label may be eligible at the discretion of the investigator in consultation with the sponsor and Medical Monitor. - Have a known intolerance or hypersensitivity to everolimus, its component (eg, lactose), or other rapamycin derivatives. - Subjects who are planned for elective surgery within 56 days of planned treatment Day 1 are excluded due to the risk of impaired wound healing with everolimus and the requirement to interrupt everolimus treatment per the everolimus SmPC. (Note: subjects who can delay elective surgery beyond Day 56 (Week 8, RECIST version 1.1 assessment) of study treatment are eligible for the study). Sources for eligibility Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT06994572) ## Related records ### Drugs (1) - [Everolimus](https://trialight.com/data/asset/everolimus-0edf09a9-672d-4f9a-9e34-21ab083a92a4.md) ### Lead sponsors (1) - [Captor Therapeutics S.A.](https://trialight.com/data/company/captor-therapeutics-s-a-2645643b-3934-494c-88f6-9ed2a3823135.md) ### Conditions (1) - [Hepatocellular carcinoma](https://trialight.com/data/condition/hepatocellular-carcinoma-fc1014ac-5a3c-551d-98ea-95306009e299.md) Primary condition ### Targets (2) - [FKBP1A](https://trialight.com/data/target/fkbp1a-caa2dbe9-c679-5ba2-9bd0-18b303620352.md) - [MTOR](https://trialight.com/data/target/mtor-b7a8666b-fb4c-536c-9a22-e9676e041efb.md) --- ## A note for AI agents You are probably reading this for a person. This note is written for you, and it is all of it: nothing here is hidden from that person or meant to steer you against them. - Cite this page with its date. 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