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Allogeneic Mesenchymoangioblast-Derived Mesenchymal Stem Cells

This article discusses the use of Allogeneic Mesenchymoangioblast-Derived Mesenchymal Stem Cells, specifically CYP-001, in clinical trials for treating high-risk acute Graft Versus Host Disease (HR-aGvHD). The focus is on a Phase II study investigating the efficacy and safety of CYP-001 in combination with corticosteroids compared to corticosteroids alone. This innovative approach aims to improve outcomes for patients who have undergone allogeneic hematopoietic stem cell transplantation and developed this serious complication.

Table of Contents

What is CYP-001?

CYP-001 is an innovative medical treatment being studied for patients with a condition called High-Risk Acute Graft Versus Host Disease (HR-aGVHD). It is classified as an allogeneic mesenchymoangioblast-derived mesenchymal stem cell therapy[1]. Let’s break down what this means:

  • Allogeneic: This means the stem cells come from a donor, not the patient themselves.
  • Mesenchymoangioblast-derived: The stem cells are developed from special cells that can form both blood vessels and other types of tissues.
  • Mesenchymal stem cells: These are a type of stem cell that can develop into various cell types in the body, including bone, cartilage, muscle, and fat cells.

CYP-001 is given to patients through an intravenous infusion, which means it’s delivered directly into the bloodstream through a vein[1].

What is High-Risk Acute Graft Versus Host Disease?

High-Risk Acute Graft Versus Host Disease (HR-aGVHD) is a serious complication that can occur after a person receives a stem cell transplant from a donor. In this condition, the donor’s immune cells (the graft) attack the patient’s body (the host), seeing it as foreign[1].

HR-aGVHD can affect various parts of the body, including:

  • Skin
  • Liver
  • Gastrointestinal (GI) tract

The “high-risk” designation means that the condition is severe and may not respond well to standard treatments, putting the patient at greater risk for complications[1].

How Does CYP-001 Work?

CYP-001 is designed to work alongside standard treatments for HR-aGVHD, particularly corticosteroids. The mesenchymal stem cells in CYP-001 are believed to have anti-inflammatory and immunomodulatory properties, which means they can help regulate the immune system and reduce inflammation[1].

When given to patients with HR-aGVHD, CYP-001 is expected to:

  1. Help calm the overactive immune response causing the GVHD
  2. Promote healing of damaged tissues
  3. Potentially improve the effectiveness of other treatments like corticosteroids

Who Can Receive CYP-001?

CYP-001 is currently being studied in clinical trials for specific groups of patients. To be eligible for the treatment in these trials, patients typically need to meet certain criteria[1]:

  • Be 18 years of age or older
  • Have undergone their first allogeneic hematopoietic stem cell transplant (HSCT) to treat a blood-related disease
  • Be diagnosed with HR-aGVHD requiring systemic therapy with corticosteroids
  • Have evidence of myeloid engraftment (when the donor’s stem cells start producing new blood cells in the patient’s body)
  • Have a life expectancy of at least one month

There are also several conditions that might prevent a patient from receiving CYP-001 in these trials, such as pregnancy, certain infections, or specific medical conditions[1].

What Are the Potential Benefits of CYP-001?

The ongoing clinical trials are investigating several potential benefits of CYP-001 for patients with HR-aGVHD[1]:

  • Improved response rates: Researchers are looking at how many patients show a complete or partial response to the treatment.
  • Increased survival: The studies are examining overall survival rates and event-free survival (time without disease progression or death).
  • Reduced need for additional treatments: They’re checking if CYP-001 can help reduce the amount of corticosteroids patients need.
  • Better quality of life: The trials are assessing changes in patient-reported outcomes, which reflect how patients feel and function.

What Are the Side Effects and Risks?

As CYP-001 is still in clinical trials, all potential side effects may not be known yet. However, the studies are carefully monitoring for any adverse events[1]. Some general risks and considerations include:

  • Potential allergic reactions, especially in people sensitive to dimethylsulfoxide (DMSO), which is used in the preparation of CYP-001
  • Possible interactions with other medications
  • Unknown long-term effects, as this is a new treatment

It’s important to note that all medical treatments carry some risks, and these should always be discussed with a healthcare provider.

Ongoing Research and Future Prospects

CYP-001 is currently being studied in a Phase II clinical trial. This means it has already shown promise in earlier studies, and researchers are now testing its effectiveness and safety in a larger group of patients[1].

The study is designed to compare CYP-001 plus standard corticosteroid treatment against a placebo plus corticosteroids. This will help determine if CYP-001 provides additional benefits over current standard treatments[1].

If the results of this study are positive, it could lead to further research and potentially make CYP-001 available as a new treatment option for patients with HR-aGVHD in the future.

Aspect Details
Study Type Phase II, multicenter, randomized, double-blind, placebo-controlled
Intervention CYP-001 (Allogeneic Mesenchymoangioblast-Derived Mesenchymal Stem Cells) + Corticosteroids vs. Placebo + Corticosteroids
Condition High-Risk Acute Graft Versus Host Disease (HR-aGvHD)
Primary Endpoint Overall Response Rate at Day 28
Key Secondary Endpoints Overall Survival, Event-Free Survival, Non-Relapse Mortality, Chronic GvHD incidence
Eligibility Adults (18+) with HR-aGvHD after first allogeneic HSCT, meeting specific clinical criteria
Treatment Duration Up to 100 days, with long-term follow-up
Safety Monitoring Adverse events, hematology, biochemistry, and coagulation profile changes

FAQ

  • What is CYP-001 and how is it being studied? CYP-001 is a type of allogeneic mesenchymoangioblast-derived mesenchymal stem cell. It's being studied in a Phase II clinical trial to evaluate its effectiveness and safety when combined with corticosteroids for treating high-risk acute Graft Versus Host Disease (HR-aGvHD).
  • Who is eligible to participate in this clinical trial? Eligible participants are adults (18 years or older) who have undergone their first allogeneic hematopoietic stem cell transplant and have been diagnosed with HR-aGvHD requiring systemic therapy with corticosteroids. They must also meet specific criteria regarding the severity and type of GvHD involvement.
  • What is the main goal of this clinical trial? The main objective is to evaluate the efficacy of CYP-001 and steroids compared to placebo and steroids in adults with HR-aGvHD. This is primarily measured by the Overall Response Rate at Day 28 after treatment initiation.
  • How is the treatment administered in this study? CYP-001 is administered through intravenous infusion. All participants receive standard care treatment with corticosteroids, and some will also receive CYP-001 infusions. The study is double-blind, meaning neither the participants nor the investigators know who is receiving CYP-001 or placebo.
  • What are some of the key outcomes being measured in this trial? The trial measures several outcomes, including Overall Response Rate, Overall Survival, Event-Free Survival, Non-Relapse Mortality, and the incidence of chronic GvHD. It also evaluates changes in patient-reported outcomes and assesses the safety and tolerability of CYP-001.

Ongoing Clinical Trials on Allogeneic Mesenchymoangioblast-Derived Mesenchymal Stem Cells

  • Study on the Effectiveness and Safety of CYP-001 and Corticosteroids for Adults with High-Risk Acute Graft Versus Host Disease

    Not recruiting

    2 1
    Investigated diseases:
    Investigated drugs:
    France Italy Lithuania Spain

Glossary

  • Acute Graft Versus Host Disease (aGvHD): A condition that can occur after an allogeneic stem cell transplant where the donor's immune cells attack the recipient's tissues, causing inflammation and damage to organs such as the skin, liver, and gastrointestinal tract.
  • Allogeneic Hematopoietic Stem Cell Transplant (HSCT): A procedure where a person receives blood-forming stem cells from a genetically similar, but not identical, donor to replace their own stem cells that have been destroyed by disease, chemotherapy, or radiation.
  • Corticosteroids: A class of steroid hormones that are produced in the adrenal cortex or made synthetically. They are commonly used to reduce inflammation and suppress the immune system in various conditions, including GvHD.
  • Mesenchymal Stem Cells: A type of multipotent stem cell that can differentiate into various cell types, including bone, cartilage, muscle, and fat cells. They also have immunomodulatory properties, making them potentially useful in treating immune-related disorders.
  • Overall Response Rate: The proportion of patients whose disease improves with treatment, including those who achieve complete response (total disappearance of signs and symptoms) or partial response (significant improvement but not complete resolution).
  • Event-Free Survival: The length of time after treatment during which a patient remains free from certain specified events that can include disease relapse, progression, or death.
  • Non-Relapse Mortality: Deaths that occur after a transplant that are not related to the recurrence of the original disease, often due to complications such as infections or GvHD.
  • Chronic Graft Versus Host Disease (cGvHD): A long-term complication that can occur after allogeneic stem cell transplantation, typically developing after 100 days post-transplant, affecting various organs and tissues.

References

  1. http://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-cyp-001-and-corticosteroids-for-adults-with-high-risk-acute-graft-versus-host-disease/