CCI-001 for Cancer

Overseen By Charles Allard

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: PharmaMatrix Holdings Ltd

Must not be taking: Corticosteroids, Anticonvulsants, Warfarin

Disqualifiers: CNS metastases, Cardiac fibrosis, HIV, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 10

70 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug called CCI-001, aimed at treating certain types of cancer. The goal is to determine the drug's safety and identify the best dose for individuals with recurrent or spreading solid tumors, such as those in the bladder, pancreas, ovaries, or lungs. People who have tried other treatments without success might be suitable candidates, especially if they have one of these specific cancers. The trial will also examine how the drug moves through the body and its effects on cancer. Participants should not have any other serious health issues, such as heart problems or active infections. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new drug.

Will I have to stop taking my current medications?

The trial requires that you stop taking any investigational agents, chemotherapy, immunotherapy, radiotherapy, or molecular targeted agents at least 28 days before enrolling. If you are taking warfarin, you will need to stop, but low-dose or therapeutic heparin is allowed.

Is there any evidence suggesting that CCI-001 is likely to be safe for humans?

Research has shown that CCI-001 is a new treatment currently being tested for safety in humans. This marks the first study involving individuals with certain solid tumors, such as bladder, pancreatic, gynecologic, and lung cancers. As this is the initial human trial, the primary goal is to assess how well participants tolerate the drug and to determine the appropriate dose.

No safety data from previous human studies exists because CCI-001 is undergoing its first human testing. At this early stage, researchers are beginning to understand its safety profile. The trial will focus on identifying any side effects and establishing the safest dose.

Although specific safety data for CCI-001 is not yet available, the trial will yield crucial information about its safety. Prospective participants should discuss potential risks and benefits with their healthcare provider. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for cancer?

Unlike the standard treatments for recurrent or metastatic solid tumors, which often include chemotherapy, surgery, or radiation, CCI-001 is unique because it is designed to target tumor cells differently. Researchers are excited about CCI-001 because it potentially offers a novel way of targeting cancer cells, possibly leading to fewer side effects and improved effectiveness compared to traditional therapies. This new approach could be particularly promising for tough-to-treat tumors like transitional cell bladder cancer, pancreaticobiliary adenocarcinomas, gynecologic cancers, and lung adenocarcinoma, where options are currently limited.

What evidence suggests that CCI-001 might be an effective treatment for cancer?

A previous study showed that CCI-001 attaches more strongly to β-III tubulin, a protein abundant in many cancers. This stronger attachment could interfere more effectively with cancer cell division. Research has shown that drugs like CCI-001 have successfully treated certain cancers, such as ovarian and lung cancers. This trial will evaluate CCI-001 in patients with various cancers, including bladder, pancreatic, bile duct, gynecologic, and lung cancers. By targeting this specific protein, CCI-001 could potentially slow or stop the growth of these cancers. ^{2 3 6 7 8}

Who Is on the Research Team?

Jennifer Spratlin, MD

Principal Investigator

Alberta Health Services, University of Alberta

Are You a Good Fit for This Trial?

Adults with certain types of recurrent or metastatic solid tumors, who have tried other treatments without success or for whom no standard treatment exists. They must be in good health otherwise, with normal organ and marrow function, and not currently receiving other cancer therapies. Women can't be pregnant or nursing, and all participants must agree to use contraception.

Inclusion Criteria

Subject has provided informed consent/assent prior to initiation of any study-specific activities/procedures

Life expectancy of greater than 12 weeks

Normal organ and marrow function as defined below: absolute neutrophil count ≥ 1.5 x 10^9/L, hemoglobin ≥ 90 g/L, platelets ≥ 100 x 10^9/L, total bilirubin ≤ 1.5 X upper limit of normal (ULN), AST(SGOT) and ALT(SGPT) ≤ 2.5 X ULN (≤ 5 X ULN in the presence of liver metastases), Creatinine (Cr) ≤ 1.5 X ULN, Cardiac ejection fraction by echocardiogram must be >50% at baseline, Baseline ECG with QTc ≤ 470msec for females and ≤ 450msec for males, Agree to use adequate contraception for the duration of study participation and for 12 months after receiving the final dose of study drug, Ability and willingness to adhere to study required procedures, Patient must have measurable disease according to Response Evaluation Criteria in Solid Tumours (RECIST) 1.1 criteria

Exclusion Criteria

I am on warfarin or low-dose heparin treatments.

Pregnant or nursing women

Human Immunodeficiency Virus (HIV)-positive patients

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Escalation

CCI-001 is administered at escalating doses to determine the maximum tolerated dose

28 days

Cycle 1 Days 1 and 15, Cycle 2 Day 1

Dose Expansion

Patients with specific tumour types are treated at the recommended dose determined during dose escalation

28 days

Cycle 1 Days 1 and 15, Cycle 2 Day 1

Follow-up

Participants are monitored for safety and effectiveness after treatment

36 months

Imaging every 8 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CCI-001

Trial Overview CCI-001 is a new drug related to colchicine being tested for safety and effective dosage in patients with specific cancers that are known to respond to similar drugs. This first-in-human trial will also look at how the body processes CCI-001 and its impact on tumor size and patient survival.

How Is the Trial Designed?

Group I: Dose Escalation and Expansion Experimental Treatment 1 Intervention

Dose escalation phase: CCI-001 will be administered at the starting dose to a cohort of patients with recurrent and/or metastatic solid tumours. The dose will be escalated sequentially in subsequent cohorts to determine the maximum tolerated dose, or recommended dose for the dose expansion cohort. Dose expansion phase: patients with the following tumour types will be permitted to enroll: transitional cell bladder cancer, pancreaticobiliary adenocarcinomas, gynecologic cancers (ovarian, cervical, endometrial), and lung adenocarcinoma. These patients will be treated at the dose determined during the dose escalation phase.

Who Is Running the Clinical Trial?

PharmaMatrix Holdings Ltd

Lead Sponsor

Trials

1

Recruited

70+

University of Alberta

Collaborator

Trials

957

Recruited

437,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04823897

A Study of CCI-001 in Patients With Recurrent and ... Dose expansion phase: patients with the following tumour types will be permitted to enroll: transitional cell bladder cancer, pancreaticobiliary adenocarcinomas ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7391668/

Targeted therapies in gynecological cancers - PubMed Central In this article, we provide a comprehensive review of the clinical evidence of targeted agents in gynecological cancers and discuss the future implication.

3. acsjournals.onlinelibrary.wiley.com acsjournals.onlinelibrary.wiley.com/doi/full/10.3322/caac.70011

Cancer treatment and survivorship statistics, 2025 - Wagle The number of survivors varies by state from almost 2 million in California to about 29,000 in the District of Columbia and 32,000 in Wyoming, ...

4. nature.com nature.com/articles/s41392-024-01823-2

Tumor biomarkers for diagnosis, prognosis and targeted ... Tumor biomarkers exist in tumor tissues or body fluids such as blood, urine, stool, saliva, and are produced by the tumor or the body's response to the tumor.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC3381956/

Targeted Therapies for Lung Cancer Although lung cancer remains the leading cancer killer in the United States, recently a number of developments indicate future clinical benefit.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04823897

A Study of CCI-001 in Patients With Recurrent and ... Dose expansion phase: patients with the following tumour types will be permitted to enroll: transitional cell bladder cancer, pancreaticobiliary adenocarcinomas ...

7. cdn.clinicaltrials.gov cdn.clinicaltrials.gov/large-docs/63/NCT01259063/Prot_000.pdf

memorial sloan-kettering cancer center irb protocol ... Stages of bladder cancer included refractory 0is in 12 patients and recurrent T1 carcinoma with or without 0is in 6 patients. Patients were previously treated ...

8. d56bochluxqnz.cloudfront.net d56bochluxqnz.cloudfront.net/documents/full-guideline/EAU-Guidelines-on-Muscle-Invasive-Bladder-Cancer-2025.pdf?utm_medium=email&utm_source=transaction

Muscle-invasive and Metastatic Bladder Cancer - Cloudfront.net metastases in patients with nonsmall cell lung carcinoma and other solid tumors: a randomized,. Phase III, double-blind, placebo-controlled trial. Cancer, 2004.