Acute Lymphoblastic Leukemia (ALL) in Complete RemissionPhase 3PTCy-PMATNCT04314219
Comparing Post-Transplant Cyclophosphamide As GVHD Prophylaxis to Standard of Care for Acute Leukemia Patients
A Phase 3 study testing Cyclophosphamide 50mg and Methotrexate in people with Acute Lymphoblastic Leukemia (ALL) in Complete Remission and Acute Myeloid Leukemia (AML) in Remission, run by King Faisal Specialist Hospital & Research Center at 1 site in Saudi Arabia.
Trial summary
This randomized clinical trial will evaluate two approaches of GvHD prophylaxis; the standard of care GVHD prophylaxis regimen (methotrexate/calcineurin inhibitors) and post-transplant cyclophosphamide with calcineurin inhibitors for their efficacy as a new GVHD prophylaxis strategy. Read more about this study
- Who is it for?
- Ages 14 to 65 years
- Conditions the study is for
- Acute Lymphoblastic Leukemia (ALL) in Complete Remission, Acute Myeloid Leukemia (AML) in Remission
- What is being tested?
- Cyclophosphamide 50mg and Methotrexate
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 264 participants
- Where
- 1 siteSaudi Arabia
- Study dates
- 15 Aug 2021to Dec 2026
- Phase
- Phase 3
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 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with Acute Leukemias (AML, ALL) in morphologic complete remission with or without hematologic recovery
- Patients must have a fully matched (8/8) related donor willing to donate peripheral blood stem cells and must meet institutional criteria for donation
- Planned Myeloablative conditioning regimen
- Cardiac function: ejection fraction at rest ≥ 50% by MUGA or TTE
- Estimated creatinine clearance greater than 50 mL/minute
- Pulmonary function: DLCO ≥ 50% (adjusted for hemoglobin), and FVC and FEV1 ≥ 50%
- Liver function: total bilirubin < 2x the upper limit of normal (unless elevated bilirubin is attributed to Gilbert's Syndrome) and ALT/AST < 2.5x the upper normal limit
- Signed informed consent
Who cannot join
- Karnofsky or Lansky Performance Score < 70%.
- Active disease
- Patients with uncontrolled bacterial, viral, or fungal infections
- Presence of fluid collection (ascites, pleural or pericardial effusion) that interferes with methotrexate clearance or makes methotrexate use contraindicated
- Patients seropositive for HIV-1 or -2
- Patients seropositive for HTLV-I or -II
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- Patients with active Hepatitis B or C viral replication by PCR
- Women who are pregnant (positive serum or urine βHCG) or breastfeeding
- Females with childbearing potential (FCBP) or men who have sexual contact with FCBP unwilling to use effective forms of birth control or abstinence for one year after transplantation
- History of uncontrolled autoimmune disease or on active treatment
- Patients with prior malignancies, except resected non-melanoma skin cancer or treated cervical carcinoma in situ; cancer treated with curative intent ≥ 5 years previously will be allowed; cancer treated with curative intent < 5 years previously will not be allowed.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Saudi Arabia1 site, 1 recruiting
- Recruiting
King Faisal Specialist Hospital & Research Center
Riyadh
Riad O El Fakih, MD
About this study
Official title: Comparing Post-Transplant Cyclophosphamide with Calcineurin Inhibitors As a GVHD Prophylaxis to Standard Care of Methotrexate and Calcineurin Inhibitors for Acute Leukemia Incorporating Patient Pharmacogenomics Profiling
This randomized clinical trial will evaluate two approaches of GvHD prophylaxis; the standard of care GVHD prophylaxis regimen (methotrexate/calcineurin inhibitors) and post-transplant cyclophosphamide with calcineurin inhibitors for their efficacy as a new GVHD prophylaxis strategy.
Read the detailed description
An open-label randomized clinical trial will be performed. Eligible patients are females and males, between 14 and 65 years undergoing allo-HCT for treatment of Acute Leukemia (AML or ALL). Patients must have a matching related peripheral blood stem cell donor. Patients from or referred to the KFSH\&RC for allogeneic transplant consideration will be offered the opportunity to participate in this trial.
Treatment Description:
Patients will be randomized on one of the arms; an intervention or a standard of care arm.
- Conditions
- Acute Lymphoblastic Leukemia (ALL) in Complete Remission, Acute Myeloid Leukemia (AML) in Remission
- Being tested
- Cyclophosphamide 50mg, Methotrexate
- Keywords
- Sibling Donor Transplant, Allogeneic hematopoietic cell transplantation, GvHD Prophylaxis, Myeloablative regimen
- Medical subject headings
- Precursor Cell Lymphoblastic Leukemia-Lymphoma, Pathologic Complete Response, Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cyclophosphamide 50mgDrug
Cyclophosphamide as GVHD prophylaxis will be on day +3 and day +5
MethotrexateDrug
Methotrexate as GVHD prophylaxis will be on day +1, day +3, and day +6
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: Intervention | Experimental | Peripheral blood matched related donor HCT, using myeloablative conditioning regimen (IV Busulfan/IV Fludarabine for AML, IV VP16/TBI for ALL) HCT with cyclophosphamide, and oral tacrolimus (or another calcineurin inhibitor if intolerant for tacrolimus) for GVHD prophylaxis. Cyclophosphamide will be given at a dose of 50 mg/kg/day IV on Day +3 and Day +5 post stem cell infusion (only 2 doses). Receives: Drug: Cyclophosphamide 50mg |
| Arm 2: Standard of Care | Active comparator | Peripheral blood matched related donor HCT, using myeloablative conditioning regimen (IV Busulfan/IV Fludarabine for AML, IV VP16/TBI for ALL) HCT with methotrexate, and oral tacrolimus (or another calcineurin inhibitor if intolerant for tacrolimus) for GVHD prophylaxis Receives: Drug: Methotrexate |
What is being measured
The primary outcome is the main question the study is designed to answer.
GRFS (GVHD-free/relapse-free survival)
Defined as the non-occurrence of a grade 3-4 acute GVHD, or systemic therapy-requiring chronic GVHD, or relapse, or death during the first post-transplant year.
Measured: One-year post-transplant
Secondary outcomes (2)
Acute Graft versus Host Disease (aGVHD)
The probabilities of grade II-IV and III-IV acute GVHD will be determined. Acute GVHD will be graded according to Glucksberg and NIH 2014 criteria.
Measured: 100 Day post transplant
Chronic Graft versus Host Disease (cGVHD)
The cumulative incidence of systemic therapy-requiring chronic GVHD will be determined. Moderate and severe chronic GVHD will be defined per the NIH 2014 criteria.
Measured: One year Post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2020
- Study start15 Aug 2021
- Primary completionDec 2025 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Lymphoblastic Leukemia (ALL) in Complete RemissionStudies of the same condition, open and under way.
- 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
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 264 participants (estimated)
Sponsor and investigators
- Lead sponsor
- King Faisal Specialist Hospital & Research Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Riad O El Fakih, MD, Principal investigator, KFSH&RCMahmoud D Aljurf, MD, MPH, Principal investigator, KFSH&RCMarwan Y Shaheen, MD, Principal investigator, KFSH&RC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04314219
- Sponsor's protocol ID
- 2181104
- Other IDs
- Saudi FDA
- Acronym
- PTCy-PMAT
- Status last verified
- Oct 2024
- First submitted
- 17 Mar 2020
- Last updated
- 13 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No