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

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What is this study about?

This clinical trial is focused on studying a condition known as High-Risk Acute Graft Versus Host Disease (HR-aGvHD). This condition can occur after a patient receives a stem cell transplant, where the donated cells attack the recipient’s body. The study is testing a new treatment called CYP-001, which is made from special cells known as allogeneic mesenchymoangioblast-derived mesenchymal stem cells. These cells are given to patients through an infusion, which means they are delivered directly into the bloodstream. The study will compare the effects of this new treatment combined with standard medications called corticosteroids against the effects of corticosteroids alone.

The purpose of the study is to see how well the new treatment works and how safe it is for patients with HR-aGvHD. Participants in the study will be randomly assigned to receive either the new treatment with corticosteroids or a placebo with corticosteroids. The study will monitor participants over a period of time to see how their condition responds to the treatment. The main goal is to observe the overall response of the disease by Day 28, which means checking if the symptoms have improved or resolved.

Throughout the study, researchers will also look at other important factors, such as how long the response lasts, overall survival, and any side effects that may occur. The study aims to provide valuable information that could lead to better treatment options for people with HR-aGvHD in the future.

1 joining the study

Upon joining the study, the participant will be randomly assigned to one of two groups: one receiving the experimental treatment CYP-001 in combination with corticosteroids, and the other receiving a placebo with corticosteroids.

The study is designed to evaluate the effectiveness and safety of the treatment for high-risk acute graft versus host disease.

2 treatment administration

Participants will receive the assigned treatment through an intravenous infusion. The frequency and dosage will be determined by the study protocol and the medical team overseeing the trial.

The treatment period will be closely monitored to assess the participant’s response and any side effects.

3 monitoring and assessment

Participants will be evaluated at specific intervals: Day 7, Day 14, Day 21, Day 28, Day 60, and Day 100. These assessments will include checking for a complete or partial response to the treatment.

The primary goal is to observe the overall response rate by Day 28, which includes the resolution or improvement of symptoms without needing additional treatments.

4 follow-up evaluations

Further evaluations will be conducted to measure the durability of the response at Day 60 and Day 100.

Additional outcomes such as overall survival, event-free survival, and failure-free survival will be tracked to understand the long-term effects of the treatment.

5 completion of the study

The study is expected to conclude by December 31, 2025. Participants will continue to be monitored for any long-term effects or changes in their condition.

The results will contribute to understanding the potential benefits and risks of the treatment for future patients.

Who Can Join the Study?

Male or female subjects who are 18 years of age or older.
Must provide written consent and agree to follow the study procedures.
Have had their first allogeneic hematopoietic stem cell transplant (HSCT), which is a procedure where stem cells are taken from a donor to treat blood-related diseases, from any donor source using bone marrow, peripheral blood stem cells, or cord blood, with any conditioning intensity.
Have been clinically diagnosed with acute Graft Versus Host Disease (GvHD), which is a condition that can occur after a stem cell transplant, requiring treatment with systemic therapy with corticosteroids (CS). A biopsy of affected organs is encouraged but not required.
Must meet specific clinical features of High-Risk acute GvHD (HR-aGvHD) within 72 hours before joining the study, such as severe skin, gastrointestinal, or liver involvement, or a combination of these.
Show evidence of myeloid engraftment, meaning the new stem cells are working, for three consecutive days with a sustained absolute neutrophil count (ANC) greater than 500 x 10⁶/L. Use of G-CSF (a medication to help increase white blood cells) and blood transfusion is allowed.
Have a life expectancy of at least one month, as judged by the study doctor.
The study doctor believes the subject or their legal representative understands the study’s nature, scope, and possible consequences.

Who Cannot Join the Study?

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Hopital Necker Enfants Malades	Paris	France
IRCCS Humanitas Research Hospital	Rozzano	Italy
Centre Hospitalier Universitaire De Lille	Lille	France
Hospital Universitario De Navarra	Pamplona	Spain

Other Sites

Site Name	City	Country
Casa Sollievo Della Sofferenza	San Giovanni Rotondo	Italy
Azienda Ulss 3 Serenissima	Venice	Italy
ASST Grande Ospedale Metropolitano Niguarda	Milan	Italy
Vilniaus universiteto ligonine Santaros klinikos VšĮ	Vilnius	Lithuania
Hospital Universitario Fundacion Jimenez Diaz	Madrid	Spain
University Clinical Hospital Virgen De La Arrixaca	Murcia	Spain
Universita’ Politecnica Delle Marche	Ancona	Italy
Hceihzaw Umiqaepakirpi Di Lf Pdpvgjwk	Madrid	Spain
Iyqveewb Cvysbl Dashqbagyriyoyhfv	L'hospitalet De Llobregat	Spain
Usluxytucz Hhuebfvxf Pejcd Swtpimrskxe Cjnsgpv Fmlk	Paris	France
Caaxdg Honzdfdzqfq Ev Ueqlotolyhvtq Dc Lzzwxfy	Limoges	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	31.01.2024
Italy	Not recruiting	31.01.2024
Lithuania	Not recruiting	31.01.2024
Spain	Not recruiting	31.01.2024

Trial locations

Investigated drugs:

Allogeneic Mesenchymoangioblast-Derived Mesenchymal Stem Cells

CYP-001 is an investigational medication being studied for its potential to treat high-risk acute graft versus host disease (HR-aGvHD). It is being tested to see if it can improve the response rate when used in combination with corticosteroids.

Corticosteroids are a type of medication commonly used to reduce inflammation and suppress the immune system. In this trial, they are used as a standard treatment for high-risk acute graft versus host disease, and their effectiveness is being compared when used alone versus in combination with CYP-001.

Investigated diseases:

Acute graft versus host disease

High-Risk Acute Graft Versus Host Disease – This condition occurs when donor immune cells attack the recipient’s body after a stem cell or bone marrow transplant. It is characterized by a rapid onset of symptoms, typically affecting the skin, liver, and gastrointestinal tract. Patients may experience skin rashes, jaundice, and diarrhea. The disease progresses as the immune response intensifies, potentially leading to more severe organ damage. The severity of symptoms can vary, and the condition is classified into different grades based on the extent of organ involvement. Management focuses on controlling the immune response to prevent further tissue damage.

Trial ID:

2022-502673-41-01

Protocol code:

CYP-GVHD-P2-01

NCT ID:

NCT05643638

Trial Phase:

Therapeutic exploratory (Phase II)

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?