Acute LeukemiaPhase 1/2NCT06279494
Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors
A Phase 1/2 study testing Sirolimus, Abatacept and MMF in people with Acute Leukemia and Myelodysplastic Syndromes, run by Peking University People's Hospital.
Trial summary
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia
- What is being tested?
- Sirolimus, Abatacept and MMF
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- Mar 2024to Mar 2025
- 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 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Primary disease: hematological malignancies (including acute leukemia, myelodysplastic syndromes), nonmalignant disorders (including severe aplastic anaemia)
- Contraindication or intolerance to CNI
- Glomerular filtration rate (GFR) 60-89 ml/min/1.73m2 before transplantation or allergy to CNI
- Intolerance to CNI during conditioning or within +45 days post transplantation, such as CNI nephrotoxicity (creatinine higher than the upper limit of normal), uncontrolled hypertension, neurological toxicity, or other conditions deemed by physician to be inappropriate for CNI
- Receiving haplo-HSCT for the first time
Who cannot join
- Allergy or intolerance to study drugs
- Active infection
- Active GVHD
- Transplantation-associated thrombotic microangiopathy
- Key organ dysfunction: liver injury (total bilirubin more than 2 upper limit of normal) or heart injury (symptomatic heart failure or ejection fraction<50%)
- Eastern Cooperative Oncology Group (ECOG) score >2
- Expected survival time <30 days
- Patients could not cooperate
Trial locations and contacts
No sites are listed yet.
About this study
Official title: Sirolimus+Abatacept+Mycophenolate Mofetil Regimen for Prophylaxis of Acute Graft-versus-host Disease (aGvHD) in Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation (Haplo-HSCT) Who Are Intolerant to Calcineurin Inhibitor
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Abatacept and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.
- Being tested
- Sirolimus, Abatacept, MMF, ATG
- Keywords
- Abatacept, Mycophenolate Mofetil, Sirolimus, Graft-versus-host-disease, Haploidentical, Calcineurin Inhibitor
- Medical subject headings
- Myelodysplastic Syndromes, Anemia, Aplastic, Graft vs Host Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SirolimusDrug
Orally (concentration 5-10ng/ml) from -1 to +100d post haplo-HSCT
AbataceptDrug
5mg/kg subcutaneous, -1/+5/+14/+28/+42/+56d post haplo-HSCT
MMFDrug
0.5g bid orally, from -3d to +60d post haplo-HSCT
ATGDrug
2.5 mg/kg, from -5d to -2d
| Study group | Role | What they receive |
|---|---|---|
| Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) | Experimental | Patients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Abatacept+MMF+ATG (SAMA) for prophylaxis of aGVHD Receives: Drug: Sirolimus; Drug: Abatacept; Drug: MMF; Drug: ATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Grade 2-4 aGVHD within 100 days post transplantation
Measured: Participants will be followed for an expected average of 100 days post transplantation
Secondary outcomes (2)
Incidence of chronic GVHD (cGVHD) within 1 year post transplantation
Measured: Participants will be followed for an expected average of 1 year
Incidence of thrombotic microangiopathy within 1 year post transplantation
Measured: Participants will be followed for an expected average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2024
- Study startMar 2024 (estimated)
- Primary completionJun 2024 (estimated)
- Study completionMar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute LeukemiaStudies 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 1/2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
- Investigators
- Xiao-Jun Huang, Principal investigator, Institute of Hematology, Peking University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06279494
- Sponsor's protocol ID
- Sirolimus for GVHD prophylaxis
- Status last verified
- Mar 2024
- First submitted
- 9 Feb 2024
- Last updated
- 21 Mar 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Sirolimus+Abatacept+Mycophenolate Mofetil Regimen for Prophylaxis of Acute Graft-versus-host Disease (aGvHD) in Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation (Haplo-HSCT) Who Are Intolerant to Calcineurin Inhibitor
- ClinicalTrials.gov ID
- NCT06279494
- Acronym / sponsor ID
- Sirolimus for GVHD prophylaxis
- Keywords
- Abatacept, Mycophenolate Mofetil, Sirolimus, Graft-versus-host-disease, Haploidentical, Calcineurin Inhibitor
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- Mar 2024 (estimated)
- Primary completion
- Jun 2024 (estimated)
- Study completion
- Mar 2025 (estimated)
- First posted
- 28 Feb 2024 (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.
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled. At present, the standard prophylaxis for GVHD is cyclosporine combined with methotrexate. However, calcineurin inhibitors (CNI) can cause some vital side effects, which are not tolerated by some patients. Therefore, this study aims to explore the safety and efficacy of Sirolimus in combination with Abatacept and Mycophenolate Mofetil for the prophylaxis of GVHD in patients with haplo-HSCT who are intolerant to calcineurin inhibitors.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) | Experimental | Drug: Sirolimus, Drug: Abatacept, Drug: MMF, Drug: ATGPatients receiving haplo-HSCT who are intolerant to calcineurin inhibitors would receive Sirolimus+Abatacept+MMF+ATG (SAMA) for prophylaxis of aGVHD |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Primary disease: hematological malignancies (including acute leukemia, myelodysplastic syndromes), nonmalignant disorders (including severe aplastic anaemia)
- Contraindication or intolerance to CNI
- Receiving haplo-HSCT for the first time
Key exclusion criteria (8 listed)
- Allergy or intolerance to study drugs
- Active infection
- Active GVHD
- Transplantation-associated thrombotic microangiopathy
- Key organ dysfunction: liver injury (total bilirubin more than 2 upper limit of normal) or heart injury (symptomatic heart failure or ejection fraction<50%)
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 |
|---|---|---|
| SirolimusOrally (concentration 5-10ng/ml) from -1 to +100d post haplo-HSCT | Drug | Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) |
| Abatacept5mg/kg subcutaneous, -1/+5/+14/+28/+42/+56d post haplo-HSCT | Drug | Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) |
| MMF0.5g bid orally, from -3d to +60d post haplo-HSCT | Drug | Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) |
| ATG2.5 mg/kg, from -5d to -2d | Drug | Sirolimus+Abatacept+Mycophenolate mofetil (MMF)+anti-thymocyte globulin (ATG) |
Primary outcome
Incidence of Grade 2-4 aGVHD within 100 days post transplantation
Time frame: Participants will be followed for an expected average of 100 days post transplantation
2 secondary outcomes
Incidence of chronic GVHD (cGVHD) within 1 year post transplantation
Time frame: Participants will be followed for an expected average of 1 year
Incidence of thrombotic microangiopathy within 1 year post transplantation
Time frame: Participants will be followed for an expected average of 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2024.
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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.
Xiao-Jun Huang principal investigator
Institute of Hematology, Peking University
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Sirolimus, Abatacept and MMF in people with Acute Leukemia and Myelodysplastic Syndromes, run by Peking University People's Hospital.
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 5 inclusion and 8 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: status not confirmed, as of the sponsor's last update on 21 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yuqian Sun, M.D. (+86-10-88324577, sunyuqian83@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Grade 2-4 aGVHD within 100 days post transplantation (time frame: Participants will be followed for an expected average of 100 days post transplantation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 21 Mar 2024.