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Cctx-001

A groundbreaking clinical trial is underway to evaluate the potential of CCTx-001, a novel chimeric antigen receptor T-cell (CAR-T) therapy, in treating patients with relapsed or refractory acute myeloid leukemia (AML). This phase 1/2 study aims to determine the optimal dosage, assess safety and tolerability, and explore the clinical effectiveness of CCTx-001 in patients who have not responded to or have relapsed after previous treatments.

Table of Contents

What is CCTX-001?

CCTX-001 is an innovative treatment being developed for patients with relapsed or refractory acute myeloid leukemia (AML)[1]. It is a type of advanced therapy known as Chimeric Antigen Receptor T Cells (CAR-T) that targets a specific protein called IL-1RAP[1]. This means that CCTX-001 is made from a patient’s own immune cells, which are modified in a laboratory to recognize and attack cancer cells more effectively.

Target Condition: Relapsed or Refractory Acute Myeloid Leukemia

Acute Myeloid Leukemia (AML) is a type of blood cancer that affects the bone marrow and blood[1]. “Relapsed” means the cancer has come back after treatment, while “refractory” means the cancer has not responded to standard treatments. CCTX-001 is being developed specifically for patients whose AML has either relapsed or become refractory to other treatments, offering hope for those who have limited options.

Clinical Trial Details

CCTX-001 is currently being studied in a clinical trial called RESOLVE AML 001[1]. This is a Phase 1/2 trial, which means it’s an early-stage study designed to:

  1. Determine the safe and effective dose of CCTX-001
  2. Assess how well patients tolerate the treatment
  3. Evaluate how effective CCTX-001 is in treating relapsed or refractory AML
The trial is “adaptive,” meaning that it can be adjusted based on early results to optimize patient outcomes[1].

How CCTX-001 Works

CCTX-001 is a type of cell therapy that uses the patient’s own immune cells. Here’s a simplified explanation of how it works:

  1. T cells (a type of immune cell) are collected from the patient’s blood through a process called leukapheresis.
  2. These T cells are then genetically modified in a laboratory to produce special receptors on their surface called chimeric antigen receptors (CARs).
  3. The CARs are designed to recognize and attach to a specific protein called IL-1RAP, which is found on some leukemia cells.
  4. The modified T cells (now called CAR-T cells) are grown in large numbers in the lab.
  5. The CAR-T cells are then infused back into the patient, where they can find and destroy cancer cells that have the IL-1RAP protein.
This personalized approach allows the patient’s own immune system to fight the cancer more effectively[1].

Eligibility Criteria

To participate in the CCTX-001 clinical trial, patients must meet certain criteria. Some key eligibility factors include:

  • Being 18 years or older
  • Having active relapsed or refractory AML
  • Having tried and not responded to standard treatments
  • Being in overall good health apart from the AML
There are also specific medical criteria that patients must meet to ensure their safety during the trial[1]. It’s important to discuss eligibility with a healthcare provider who can review all criteria in detail.

Potential Benefits

While it’s important to remember that CCTX-001 is still in the experimental stage, the potential benefits being studied include:

  • Improved response rates compared to standard treatments for relapsed/refractory AML
  • Longer-lasting remissions
  • Better quality of life for patients with this difficult-to-treat condition
The trial will measure these outcomes using various methods, including assessing the complete response rate and monitoring patients’ health-related quality of life[1].

Safety Considerations

As with any new treatment, safety is a top priority in the CCTX-001 trial. The study will closely monitor patients for side effects and complications. Some potential risks associated with CAR-T cell therapies in general include:

  • Cytokine release syndrome (a condition that can cause fever, low blood pressure, and breathing difficulties)
  • Neurological effects
  • Infections
The trial includes specific measures to manage these potential side effects and ensure patient safety[1]. Patients in the trial will be closely monitored and receive comprehensive care throughout their participation.

Aspect Details
Study Type Phase 1/2 clinical trial
Treatment CCTx-001 (CAR-T cell therapy targeting IL-1RAP)
Condition Relapsed/Refractory Acute Myeloid Leukemia (AML)
Primary Objectives Determine safe dose, assess safety and tolerability, evaluate clinical activity
Key Eligibility Criteria Adults with active r/r AML, adequate organ function, no active infections
Administration Intravenous infusion
Main Outcomes Measured Safety profile, response rates, survival outcomes, quality of life

FAQ

  • What is CCTx-001? CCTx-001 is a new type of CAR-T cell therapy that targets IL-1RAP, a protein found on certain cancer cells. It's being studied as a potential treatment for patients with relapsed or refractory acute myeloid leukemia (AML).
  • Who is eligible for this clinical trial? The trial is open to adults (18 years or older) with relapsed or refractory AML who meet specific criteria, including having active disease, adequate organ function, and no active infections or other severe medical conditions.
  • What are the main goals of this clinical trial? The primary goals are to determine the safe and effective dose of CCTx-001 (Phase 1) and to evaluate its clinical effectiveness in treating relapsed/refractory AML (Phase 2). The study will also assess safety, tolerability, and quality of life for patients.
  • How is CCTx-001 administered? CCTx-001 is given as an intravenous infusion. Before receiving the treatment, patients will undergo leukapheresis, a procedure to collect their white blood cells, which will then be modified to create the CAR-T cells.
  • What are the potential benefits and risks of participating in this trial? Potential benefits include access to a new treatment that may be effective against AML when other treatments have failed. Risks may include side effects related to the therapy, such as immune system reactions. The study team will closely monitor participants for any adverse effects throughout the trial.

Ongoing Clinical Trials on Cctx-001

  • Study on CCTx-001, Cyclophosphamide Monohydrate, and Fludarabine Phosphate for Patients with Relapsed or Refractory Acute Myeloid Leukemia

    Not yet recruiting

    2 1 1 1
    Investigated drugs:
    France Germany Spain Sweden

Glossary

  • Acute Myeloid Leukemia (AML): A type of cancer that affects the blood and bone marrow, characterized by the rapid growth of abnormal white blood cells that interfere with normal blood cell production.
  • Relapsed/Refractory: Refers to cancer that has returned after treatment (relapsed) or has not responded to initial treatment (refractory).
  • CAR-T Cell Therapy: A type of treatment in which a patient's T cells (a type of immune system cell) are changed in the laboratory to attack cancer cells.
  • IL-1RAP: Interleukin-1 Receptor Accessory Protein, a target on certain cancer cells that CCTx-001 is designed to recognize and attack.
  • Leukapheresis: A procedure to separate and collect white blood cells from the blood, used in this trial to obtain cells for creating the CAR-T therapy.
  • Intravenous Infusion: A method of delivering medications or fluids directly into a vein.
  • Phase 1/2 Trial: A clinical study that combines initial testing of a new drug's safety (Phase 1) with preliminary testing of its effectiveness (Phase 2).
  • Recommended Phase 2 Dose (RP2D): The dose of a drug determined in Phase 1 to be safe and potentially effective, which is then used in Phase 2 studies.
  • Complete Remission (CR): A treatment response where all signs of cancer have disappeared, though it does not always mean the cancer has been cured.
  • Adverse Events (AEs): Any unfavorable and unintended sign, symptom, or disease temporarily associated with the use of a medical treatment or procedure.

References

  1. http://clinicaltrials.eu/trial/study-on-cctx-001-cyclophosphamide-monohydrate-and-fludarabine-phosphate-for-patients-with-relapsed-or-refractory-acute-myeloid-leukemia/