Graft Vs Host DiseasePhase 3NCT04738981
Efficacy and Safety of UC-MSCs for the Treatment of Steroid-resistant aGVHD Following Allo-HSCT
A Phase 3 study testing UC-MSC and Anti-CD25 mAb in people with Graft Vs Host Disease, run by Peking University People's Hospital at 1 site in China.
Trial summary
Randomized, open-label, multicenter study to investigate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSC) for the treatment of steroid-resistant acute graft-versus-host disease (aGVHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a view to establishing… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Graft Vs Host Disease
- What is being tested?
- UC-MSC and Anti-CD25 mAb
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 130 participants
- Where
- 1 siteChina
- Study dates
- 1 Feb 2021to 2 May 2023
- Phase
- Phase 3
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1.Confirmed diagnosed steroid-resistant aGVHD after allo-HSCT. 2. Neutrophile granulocyte (ANC) ≥ 0.5 x 10^9/L. 3. Creatinine below 2 times normal upper limit . 4. Willing and able to sign written informed consent.
Who cannot join
- 1. Untoleratation of the treatment. 2. Primary disease are not effectively controlled or are progressing. 3. Active infection (Bacteria, fungi, viruses ). 4. Combined with dysfunction of multiple organ. 5. Female patients who are nursing or pregnant, or who contemplate pregnancy during the study period.
- 6. Patients who are deemed unsuitable for the study by the investigator.
Trial locations and contacts
1 site in 1 country.
China1 site
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, 100010
About this study
Official title: Efficacy and Safety of UC-MSCS for the Treatment of Steroid-resistant aGVHD Following Allo-HSCT: A Multicenter, Randomized, Open-label Trial
Randomized, open-label, multicenter study to investigate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSC) for the treatment of steroid-resistant acute graft-versus-host disease (aGVHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a view to establishing an effective treatment protocol for steroid-resistant aGVHD.
Read the detailed description
A total of 130 patients with steroid-resistant aGVHD after allo-HSCT are enrolled in this multicenter, randomized, controlled trial. Patients were randomized to UC-MSC + anti-CD25 monoclonal antibodies (mAb) group and anti-CD25 mAb group. The complete response (CR) rate after 4 weeks of treatment in the two groups will be compared. Adverse events are also recorded throughout the study.
- Conditions
- Graft Vs Host Disease
- Being tested
- UC-MSC, Anti-CD25 mAb
- Medical subject headings
- Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UC-MSCDrug
Also called Umbilical cord-derived mesenchymal stem cells
UC-MSC, iv, 1×10^6 cells/kg, once a week, for 4 weeks.
Anti-CD25 mAbDrug
Also called Basiliximab
Anti-CD25 mAb, iv, 20mg，twice in first week and once a week thereafter, for 4 weeks.
| Study group | Role | What they receive |
|---|---|---|
| UC-MSC and anti-CD25 mAb | Experimental | UC-MSC, iv, 1×10^6 cells/kg, once a week, for 4 weeks. Anti-CD25 mAb, iv, 20mg，twice in first week and once a week thereafter, 4 weeks. Other treatment would replace it according to clinical experience if aGVHD continue to progress within 3 weeks of treatment or patients are lack of response after 4 weeks of treatment. The treatment would be repeated in another 4 weeks if patients receive partial response after the first 4 weeks of treatment. Receives: Drug: UC-MSC; Drug: Anti-CD25 mAb |
| Anti-CD25 mAb | Active comparator | Anti-CD25 mAb, iv, 20mg，twice in first week and once a week thereafter, for 4 weeks. Other treatment would replace it according to clinical experience if aGVHD continue to progress within 3 weeks of treatment or patients are lack of response after 4 weeks of treatment. The treatment would be repeated in another 4 weeks if patients receive partial response after first 4 weeks of treatment. Receives: Drug: Anti-CD25 mAb |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of complete remission
Complete remission of aGVHD related symptoms and indicators
Measured: 4 weeks after treatment
Secondary outcomes (3)
Overall survival
The time from randomization to death for any reason ( the last follow-up time for missing patients; end date of follow-up for patients who are live at the end of the study).
Measured: At the end of Week 4 / 8 / 12 / 24 / 52.
Rate of partial remission
improvement of aGVHD staging in one or more organs without progression in other organs
Measured: 4 weeks after treatment
Infusion toxicity
Acute toxicity responses include impaired function of heart, kidney and liver
Measured: From the beginning of to four hours after every infusion of UC-MSC
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2021
- Study start1 Feb 2021
- Primary completion30 May 2022
- Study completion2 May 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Graft Vs Host DiseaseStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 130 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Southern Medical University, China, Third Military Medical University, Sun Yat-sen University
- Responsible party
- Principal investigator, Xiao Hui Zhang, Peking University People's Hospital
- Investigators
- Xiaohui Zhang, doctor, Principal investigator, Peking University People's Hospital, Peking University Insititute of Hematology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04738981
- Sponsor's protocol ID
- GVHD-PKU2021
- Status last verified
- May 2023
- First submitted
- 1 Feb 2021
- Last updated
- 1 Jun 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Fu H, Sun X, Lin R, Wang Y, Xuan L, Yao H, Zhang Y, Mo X, Lv M, Zheng F, Kong J, Wang F, Yan C, Han T, Chen H, Chen Y, Tang F, Sun Y, Chen Y, Xu L, Liu K, Zhang X, Liu Q, Huang X, Zhang X. Mesenchymal stromal cells plus basiliximab improve the response of steroid-refractory acute graft-versus-host disease as a second-line therapy: a multicentre, randomized, controlled trial. BMC Med. 2024 Feb 27;22(1):85. doi: 10.1186/s12916-024-03275-5.
PubMed 38413930
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of UC-MSCS for the Treatment of Steroid-resistant aGVHD Following Allo-HSCT: A Multicenter, Randomized, Open-label Trial
- ClinicalTrials.gov ID
- NCT04738981
- Acronym / sponsor ID
- GVHD-PKU2021
- Conditions
- Graft Vs Host Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 130 participants (actual)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Collaborators
- Southern Medical University, China, Third Military Medical University, Sun Yat-sen University
- Study start
- 1 Feb 2021 (actual)
- Primary completion
- 30 May 2022 (actual)
- Study completion
- 2 May 2023 (actual)
- First posted
- 4 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Randomized, open-label, multicenter study to investigate the efficacy and safety of umbilical cord-derived mesenchymal stem cells (UC-MSC) for the treatment of steroid-resistant acute graft-versus-host disease (aGVHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a view to establishing an effective treatment protocol for steroid-resistant aGVHD.
Detailed description
A total of 130 patients with steroid-resistant aGVHD after allo-HSCT are enrolled in this multicenter, randomized, controlled trial. Patients were randomized to UC-MSC + anti-CD25 monoclonal antibodies (mAb) group and anti-CD25 mAb group. The complete response (CR) rate after 4 weeks of treatment in the two groups will be compared. Adverse events are also recorded throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| UC-MSC and anti-CD25 mAb | Experimental | Drug: UC-MSC, Drug: Anti-CD25 mAbUC-MSC, iv, 1×10^6 cells/kg, once a week, for 4 weeks. Anti-CD25 mAb, iv, 20mg，twice in first week and once a week thereafter, 4 weeks. Other treatment would replace it according to clinical experience if aGVHD continue to progress within 3 weeks of treatment or patients are lack of response after 4 weeks of treatment. The treatment would be repeated in another 4 weeks if patients receive partial response after the first 4 weeks of treatment. |
| Anti-CD25 mAb | Active comparator | Drug: Anti-CD25 mAbAnti-CD25 mAb, iv, 20mg，twice in first week and once a week thereafter, for 4 weeks. Other treatment would replace it according to clinical experience if aGVHD continue to progress within 3 weeks of treatment or patients are lack of response after 4 weeks of treatment. The treatment would be repeated in another 4 weeks if patients receive partial response after first 4 weeks of treatment. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 1.Confirmed diagnosed steroid-resistant aGVHD after allo-HSCT. 2. Neutrophile granulocyte (ANC) ≥ 0.5 x 10^9/L. 3. Creatinine below 2 times normal upper limit . 4. Willing and able to sign written informed consent.
Key exclusion criteria (2 listed)
- 1. Untoleratation of the treatment. 2. Primary disease are not effectively controlled or are progressing. 3. Active infection (Bacteria, fungi, viruses ). 4. Combined with dysfunction of multiple organ. 5. Female patients who are nursing or pregnant, or who contemplate pregnancy during the study period.
- 6. Patients who are deemed unsuitable for the study by the investigator.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UC-MSCAlso: Umbilical cord-derived mesenchymal stem cellsUC-MSC, iv, 1×10^6 cells/kg, once a week, for 4 weeks. | Drug | UC-MSC and anti-CD25 mAb |
| Anti-CD25 mAbAlso: BasiliximabAnti-CD25 mAb, iv, 20mg，twice in first week and once a week thereafter, for 4 weeks. | Drug | Anti-CD25 mAb, UC-MSC and anti-CD25 mAb |
Primary outcome
Rate of complete remission
Time frame: 4 weeks after treatment
Complete remission of aGVHD related symptoms and indicators
3 secondary outcomes
Overall survival
Time frame: At the end of Week 4 / 8 / 12 / 24 / 52.
Rate of partial remission
Time frame: 4 weeks after treatment
Infusion toxicity
Time frame: From the beginning of to four hours after every infusion of UC-MSC
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2023.
- Derived from this study: PubMed 38413930Fu H, Sun X, Lin R, Wang Y, Xuan L, Yao H, Zhang Y, Mo X, Lv M, Zheng F, Kong J, Wang F, Yan C, Han T, Chen H, Chen Y, Tang F, Sun Y, Chen Y, Xu L, Liu K, Zhang X, Liu Q, Huang X, Zhang X. Mesenchymal stromal cells plus basiliximab improve the response of steroid-refractory acute graft-versus-host disease as a second-line therapy: a multicentre, randomized, controlled trial. BMC Med. 2024 Feb 27;22(1):85. doi: 10.1186/s12916-024-03275-5.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Xiaohui Zhang, doctor principal investigator
Peking University People's Hospital, Peking University Insititute of Hematology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing UC-MSC and Anti-CD25 mAb in people with Graft Vs Host Disease, run by Peking University People's Hospital at 1 site in China.
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 1 inclusion and 2 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 1 Jun 2023. 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 China. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 remission (time frame: 4 weeks after treatment). 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 NCT04738981, which also lists 1 linked publication.
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 1 Jun 2023.