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Acute-graft-versus-host DiseasePhase 1/2GVHD Cyto-MSCNCT03847844

UCMSCs as Front-line Approach of Treatment for Patients With aGVHD

A Phase 1/2 study testing Umbilical cord derived mesenchymal stem cell in people with Acute-graft-versus-host Disease, run by Cytopeutics Sdn. Bhd. at 1 site in Malaysia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Background: Graft-versus-host disease (GVHD) is a devastating complication following allogeneic hematopoietic stem cell transplantation (HSCT) mediated by stimulation of antigen presenting cells (APCs) which leads to donor T-lymphocytes activation and target tissue destruction, particularly affecting the skin… Read more about this study

Who is it for?
Ages 16 years and older
Conditions the study is for
Acute-graft-versus-host Disease
What is being tested?
Umbilical cord derived mesenchymal stem cell
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
26 participants
Where
1 siteMalaysia
Study dates
1 Feb 2019to 31 Aug 2023
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 16 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Patient age 16 and above
  • Patient who has undergone an allogeneic haematopoietic stem cell transplantation (HSCT) receiving allograft either from HLA-matched related donor (MRD), HLA-matched unrelated donor (MUD), Unrelated Cord Blood (UCB) or Haploidentical donor and developed grade II-IV acute graft versus host disease (acute GVHD) involving the skin, GI tract and/or liver based on 1994 Consensus Conference on Acute GVHD Grading
  • Patient and/or parent(s) or legal guardian(s) and assent form signed informed consent. Assent form will be obtained for patients aged less than 18 years. Investigators will obtain the permission of the parents or guardians for the participation of the minor in the research, and to solicit assent from the minor

Who cannot join

  • Patient who has enrolled in another investigational drug trial or stem cell related trial or has completed the aforesaid within (3) months
  • Patient with HIV or syphilis (Patient should be screened for HIV and VDRL up to 6 months prior to study start)
  • Patient with Hepatitis B (HBV) or Hepatitis C (HCV). All patients must be screened for HBV and HCV up to 6 months prior to study start using the routine hepatitis virus laboratory. Patients who are positive for HBsAg or HBcAb will be eligible if they are negative for HBV-DNA, these patients should receive prophylactic antiviral therapy. Patients who are positive for anti-HCV antibody will be eligible if they are negative for HCV-RNA
  • Patient has creatinine clearance of ≤50mL/min or creatinine is ≥200 µmol/L
  • Patient had undergone or on other immune-modulatory treatments such as interferon or Thalidomide over the last 12 months
  • Patient with progressive underlying disease or not in complete remission (CR) at the time of transplant
Show all 12Show fewer
  • Any other severe co-morbidities which the doctor deems as a contraindication to cell therapy
  • Adults under law protection or without ability to consent
  • The patient has previous history or on-going psychiatric illness
  • Patient has received an HSCT transplant for a solid tumor disease
  • Patient has a known hypersensitivity to dimethyl sulfoxide (DMSO)
  • Patient is a female who is pregnant, lactating, or is planning a pregnancy during study participation, or in the follow-up period

Trial locations and contacts

1 site in 1 country.

Malaysia1 site

  • Hospital Ampang

    Ampang, Selangor, 68000

Other trials in Malaysia

About this study

Official title: Cytopeutics® Umbilical Cord Mesenchymal Stem Cells (Cyto-MSC) for Patients With Grade II -IV Acute Graft-Versus-Host Disease: A Phase I/II Clinical Study

Background: Graft-versus-host disease (GVHD) is a devastating complication following allogeneic hematopoietic stem cell transplantation (HSCT) mediated by stimulation of antigen presenting cells (APCs) which leads to donor T-lymphocytes activation and target tissue destruction, particularly affecting the skin, gastrointestinal tract, and liver in acute setting. In recent years, researchers have discovered that the application of mesenchymal stromal cells (MSCs) as salvage treatment among steroid refractory GVHD patients improves outcomes without long-term risk association. On the other hand, the use of MSCs concurrently with steroids as front-line treatment for acute GVHD has yet to be researched on. The investigators hypothesize that this approach, as the MSCs will be administered at earlier stage of the disease, will increase survival rate and reduce mortality among aGVHD patients.

Objective: In this study, the investigators aim to determine the efficacy and safety of allogeneic infusion of Cytopeutics® umbilical cord-derived mesenchymal stromal cells (Cyto-MSC) in combination of standard corticosteroid therapy as front-line approach for treatment of grade II-IV acute GVHD patients.

Read more

Study design: This is a phase I/II clinical study involving patients who underwent an allogeneic HSCT for malignant or non malignant haematological disorders and developed grade II-IV acute GVHD. A total of 40 eligible patients will be recruited in this study.

For Phase I open labelled study, 5 eligible patients will be recruited to receive Cyto-MSC (5 million UC-MSCs per kg bodyweight) and standard treatment. Meanwhile, for Phase II double blinded placebo controlled study, another 35 eligible patients will be recruited and randomized into 2 study groups where 15 patients will be assigned into Group A to receive Cyto-MSC (5 million UCMSCs per kg bodyweight) and standard treatment, meanwhile another 20 patients will be assigned into Group B to receive Placebo and standard treatment.

Cyto-MSC or Placebo will be administered at Day 1 and Day 4. Another infusion of Cyto-MSC or Placebo will be given at Day 7 if the patient shows no or partial response based on GvHD grading criteria. All patients will be assessed up until 6 months follow-up which include medical history, clinical and physical evaluations, pathology investigations, biomarkers and immune cell subsets analysis, as well as quality of life questionnaires.

Being tested
Umbilical cord derived mesenchymal stem cell, Placebo

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Umbilical cord derived mesenchymal stem cellBiological

    Also called Cyto-MSC

    Umbilical cord derived mesenchymal stem cell

  • PlaceboOther

    Normal saline

Study groups in NCT03847844
Study groupRoleWhat they receive
Group AExperimentalCyto-MSC (5 million UCMSCs per kg bodyweight) and standard treatment
Receives: Biological: Umbilical cord derived mesenchymal stem cell
Group BPlacebo comparatorPlacebo (normal saline) and standard treatment
Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Rate of Complete Response (CR) at Day 28

    Measured: 28 days

  2. Rate of Partial Response (PR) at Day 28

    Measured: 28 days

  3. Rate of PR and CR at Day 14

    Measured: 14 days

  4. Percentage of patient requiring MMF rescue during treatment

    Measured: 6 months

  5. Rate of long term complication incidence

    Measured: 6 months

  6. Rate of chronic clinical response incidence

    Measured: 6 months

Secondary outcomes (10)

  1. Overall survival (OS) rate at 3 months

    Measured: 3 months

  2. Correlation between response to Cyto-MSC at Day 14 and Day 28 to survival at 90 days

    Measured: 90 days

  3. Relapse-free survival at 3 months

    Measured: 3 months

  4. Disease relapse at 3 months

    Measured: 3 months

  5. Incidence of infection

    Measured: 6 months

  6. Incidence of CMV reactivation

    Measured: 6 months

  7. Measurement quality of life of cancer patients by using EORTC QLQ-C30 questionnaire

    The final score ranges from 0 (better outcome) to 126 (worse outcome)

    Measured: 6 months

  8. Measurement quality of life in bone marrow transplantation by using FACT-BMT questionnaire

    The final score ranges from 0 (better outcome) to 164 (worse outcome)

    Measured: 6 months

  9. Measurement of health-related quality of life in children and young people by using PEDsQL questionnaire (patients age less than 18 years old)

    The final score ranges from 0 (better outcome) to 92 (worse outcome)

    Measured: 6 months

  10. Measurement of generic health status among patients by using EQ-5D questionnaire

    The final score ranges from 0 (better outcome) to 100 (worse outcome)

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted20 Feb 2019
  2. Study start1 Feb 2019
  3. Primary completion18 Jul 2023
  4. Study completion31 Aug 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
26 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03847844
Sponsor's protocol ID
POD0030/CP/R
Acronym
GVHD Cyto-MSC
Status last verified
Dec 2025
First submitted
14 Feb 2019
Last updated
2 Jan 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Umbilical cord derived mesenchymal stem cell in people with Acute-graft-versus-host Disease, run by Cytopeutics Sdn. Bhd. at 1 site in Malaysia.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Malaysia. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Rate of Complete Response (CR) at Day 28 (time frame: 28 days). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03847844.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Jan 2026.

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