Active not recruitingPHASE2INTERVENTIONAL

A Study of CYP-001 in Combination With Corticosteroids in Adults With High-risk aGvHD

This research study is for adults who have had a stem cell transplant and are now experiencing a severe complication called high-risk acute Graft Versus Host Disease (GvHD). We are testing a new treatment called CYP-001 alongside the usual steroid medication. Some participants will receive CYP-001, while others will get a placebo (a dummy treatment) along with their steroids. The aim is to see if CYP-001 can improve outcomes for patients with this challenging condition. Participants will be monitored closely for up to two years to understand the treatment's effects and safety. This is a Phase 2 study, meaning we are looking more closely at how well the new treatment works and any side effects it might have.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

Cynata Therapeutics Limited

Enrolment target

Start

04 Mar 2024

Estimated completion

01 Dec 2027

Graft Versus Host Disease, Acute

What is this study about?

When someone has a stem cell transplant, they receive healthy blood-forming cells, often from a donor. Sometimes, these new donor cells can see the patient's body as 'foreign' and attack it. This is called Graft Versus Host Disease, or GvHD. Acute GvHD is a severe form that happens soon after the transplant. It can affect different organs, such as the skin, gut, and liver, and can be very serious.

This study focuses on a type called high-risk acute GvHD, which is particularly challenging to treat. The standard treatment for GvHD involves medicines like steroids to calm down the immune system. However, these don't always work for everyone, and some people need additional options.

That's where CYP-001 comes in. This new treatment uses special cells that come from what are called induced pluripotent stem cells. The study aims to find out if adding CYP-001 to the usual steroid treatment is more effective than steroids alone in helping patients with high-risk acute GvHD. We want to see if it can reduce the severity of GvHD and improve patients' health.

Key takeaways

This study is for adults with a severe complication called high-risk acute aGvHD after a stem cell transplant.
It tests a new treatment (CYP-001) given with standard steroids, compared to steroids and a placebo.
The new treatment, CYP-001, uses special cells to help manage the GvHD.
Participants will have regular check-ups for up to two years to see how the treatment works.
Taking part is voluntary, and you can withdraw at any time.

Who may be eligible?

To join this study, you must be an adult who has recently had a stem cell transplant. You must also have been diagnosed with acute GvHD that is considered 'high-risk' and needs treatment with steroids. Your body needs to have started producing new blood cells from the transplant already, and you should be expected to live for at least another month.

There are also some reasons why you might not be able to join. For example, if you've already received other specific treatments for your GvHD apart from steroids or certain other medicines. You also can't have chronic GvHD (a longer-term form of the condition) or a mixture of both types. Certain serious infections, other significant medical problems with your heart, lungs, or kidneys, or a known allergy to a specific ingredient in the study medicine would also prevent you from taking part. You also can't have had your original cancer come back since your transplant, or have had more than one stem cell transplant.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult (18 or over)?
Have you recently had a stem cell transplant?
Have you been diagnosed with high-risk acute GvHD that needs steroid treatment?
Has your body started making new blood cells after the transplant?
Have you NOT received other GvHD treatments besides steroids (and possibly certain other specific immunosuppressants)?
Do you NOT have chronic GvHD or certain serious infections (like uncontrolled CMV, EBV, HIV, etc.)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you'll be randomly assigned to one of two groups: either you'll receive the new treatment (CYP-001) or a placebo (a dummy treatment, which looks the same but has no active medicine). Both groups will continue to receive their usual steroid medication. The CYP-001 or placebo will be given through a drip into your arm on two specific days, Day 0 and Day 4, early in the study.

You'll have regular visits to the clinic for checks and assessments for the first 100 days. These visits will involve doctors examining you, taking blood samples, and asking about your symptoms to see how the GvHD is responding. After this initial period, you'll have follow-up visits for up to two years. These longer-term visits are important to understand the full effects of the treatment and ensure your safety over time. The total time you'll be involved in the study is up to 24 months.

Potential risks and benefits

Taking part in a clinical trial might potentially offer benefits, such as gaining access to a new treatment before it's widely available, which could be more effective than current options for your condition. However, there's no guarantee the new treatment will work for you, and you might receive the placebo. All medical treatments carry potential risks, and CYP-001 may have side effects that aren't fully known yet. You will be closely monitored for any reactions or health changes. You are free to withdraw from the study at any time, for any reason, without affecting your usual medical care.

Locations (39)

Banner MD Anderson
Verified postcode
Phoenix, United States
Mayo Clinic Hospital
Verified postcode
Phoenix, United States
University of Arkansas Medical Center
Verified postcode
Little Rock, United States
Mayo Clinic Hospital
Verified postcode
Jacksonville, United States
Memorial healthcare System
Verified postcode
Pembroke Pines, United States
BMT Group of Georgia
Verified postcode
Atlanta, United States
Northwestern University
Verified postcode
Evanston, United States
Karmanos Cancer Institute
Verified postcode
Detroit, United States
University Of Nebrasaka Medical Center
Verified postcode
Omaha, United States
Weill Cornell Medicine - New York Presbyterian Hospital
Verified postcode
New York, United States
Cleveland Clinic
Verified postcode
Cleveland, United States
Penn State Health
Verified postcode
Hershey, United States

Common questions

What is Graft Versus Host Disease (GvHD)?

GvHD is a complication that can happen after a stem cell transplant when the transplanted donor cells see the patient's body as 'foreign' and start to attack it. It can affect different parts of the body like the skin, gut, and liver.

What does 'high-risk acute GvHD' mean?

Acute GvHD happens shortly after a transplant. 'High-risk' means that it is a more severe form of the disease that needs stronger treatment and careful watching.

What is CYP-001?

CYP-001 is a new investigational treatment that uses special cells derived from what are called induced pluripotent stem cells. This study is testing if it can help improve GvHD alongside standard steroid treatment.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active drug. It helps researchers compare the new medicine's effects fairly.

How long will I be in the study?

You'll have regular hospital visits for the first 100 days, followed by check-ups for up to two years (24 months) in total.