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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04314219
- Acronym / sponsor ID
- PTCy-PMAT / 2181104
- Conditions
- Acute Lymphoblastic Leukemia (ALL) in Complete Remission, Acute Myeloid Leukemia (AML) in Remission
- Keywords
- Sibling Donor Transplant, Allogeneic hematopoietic cell transplantation, GvHD Prophylaxis, Myeloablative regimen
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 264 participants (estimated)
- Lead sponsor
- King Faisal Specialist Hospital & Research CenterUniversity, hospital or other
- Study start
- 15 Aug 2021 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 19 Mar 2020 (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.
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.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1: Intervention | Experimental | Drug: Cyclophosphamide 50mgPeripheral 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). |
| Arm 2: Standard of Care | Active comparator | Drug: MethotrexatePeripheral 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 |
Who may qualify
- Ages
- 14 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (11 listed)
- 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
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 |
|---|---|---|
| Cyclophosphamide 50mgCyclophosphamide as GVHD prophylaxis will be on day +3 and day +5 | Drug | Arm 1: Intervention |
| MethotrexateMethotrexate as GVHD prophylaxis will be on day +1, day +3, and day +6 | Drug | Arm 2: Standard of Care |
Primary outcome
GRFS (GVHD-free/relapse-free survival)
Time frame: One-year post-transplant
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.
2 secondary outcomes
Acute Graft versus Host Disease (aGVHD)
Time frame: 100 Day post transplant
Chronic Graft versus Host Disease (cGVHD)
Time frame: One year Post-transplant
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2025.
- Individual participant dataSharing planned: No
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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.
Riad O El Fakih, MD principal investigator
KFSH&RC
Mahmoud D Aljurf, MD, MPH principal investigator
KFSH&RC
Marwan Y Shaheen, MD principal investigator
KFSH&RC
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 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.
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 8 inclusion and 11 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: looking for participants now, as of the sponsor's last update on 13 Mar 2025. 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 Saudi Arabia. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Riad O El Fakih, MD (+ 966114647272 ext. 34760, relfakih1@kfshrc.edu.sa), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
GRFS (GVHD-free/relapse-free survival) (time frame: One-year post-transplant). 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 13 Mar 2025.