Patients With MDS, Acute Myeloid Leukemia (AML) Evolving From MDS, and Chronic Myelomonocytic Leukemia (CMML)Phase 2NCT06098313
HCT With PTCy in Higher-risk MDS
A Phase 2 study testing Cyclophosphamide in people with Patients With MDS, Acute Myeloid Leukemia (AML) Evolving From MDS, and Chronic Myelomonocytic Leukemia (CMML), run by Asan Medical Center at 1 site in South Korea.
Trial summary
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). Read more about this study
- Who is it for?
- Ages 15 to 75 years
- Conditions the study is for
- Patients With MDS, Acute Myeloid Leukemia (AML) Evolving From MDS, and Chronic Myelomonocytic Leukemia (CMML)
- What is being tested?
- Cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 113 participants
- Where
- 1 siteSouth Korea
- Study dates
- 1 Jul 2020to 31 May 2027
- Phase
- Phase 2
- 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 15 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- MDS defined by WHO classification, CMML, or AML evolving from MDS A. International Prognostic Scoring System (IPSS) > 1.0 or bone marrow blast ≥ 5% at any time points before HCT or B. AML progressed from MDS or C. CMML with bone marrow blast ≥ 5% at any time points before HCT
- Patients receiving first HCT
- Patients with appropriate hematopoietic cell donors A. HLA-matched sibling donor B. Unrelated donor C. HLA-mismatched familial donor
- 15 years old or older , under 75 years
- Adequate performance status (Karnofsky score of 70 or more)
- Adequate hepatic function (AST or ALT < 3 x upper normal limits and bilirubin < 1.5 x upper normal limit).
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- Adequate renal function (creatinine < 2.0 mg/dL or creatinine clearance ≥ 50 mL/min)
- Adequate cardiac function (left ventricular ejection fraction ≥ 50% on heart scan or echocardiogram)
- Adequate pulmonary function: DLCO, FEV1, and FVC ≥ 45% predicted by pulmonary function tests
- Signed and dated informed consent must be obtained from both recipient and donor.
Who cannot join
- Presence of significant active infection
- Presence of uncontrolled bleeding
- Any coexisting major illness or organ failure
- Patients with psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlike, and making informed consent impossible
- Nursing women, pregnant women, women of childbearing potential who do not want adequate contraception
- Patients with a diagnosis of prior malignancy unless disease-free for at least 5 years following therapy with curative intent (except curatively treated nonmelanoma skin cancer, in situ carcinoma, or cervical intraepithelial neoplasia)
- Presence of contraindications to cyclophosphamide (patients receiving pentostatin, symptomatic cystitis, urinary tract obstruction, with a history of hypersensitivity reactions to the component of the drug)
Trial locations and contacts
1 site in 1 country.
South Korea1 site
Asan Medical Center, University of Ulsan College of Medicine
Seoul, 05505
About this study
Official title: Efficacy and Safety of Allogeneic Hematopoietic Cell Transplantation Using Post-transplantation Cyclophosphamide for Graft-versus-host Disease Prophylaxis in Higher-risk Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia, and Secondary AML Patients
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
The secondary end points of the study include engraftment, relapse incidence, non-relapse mortality, graft-versus-host disease, donor chimerism, immune reconstitution, infections, and survivals (overall and event-free).
Read the detailed description
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
The secondary end points of the study include engraftment, relapse incidence, non-relapse mortality, graft-versus-host disease, donor chimerism, immune reconstitution, infections, and survivals (overall and event-free).
- Being tested
- Cyclophosphamide
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
-Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2 days)
| Study group | Role | What they receive |
|---|---|---|
| PTCy | Experimental | Patients who receive post-transplantation cyclophosphamide Receives: Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
GVHD-free, relapse-free survival
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
Measured: 8 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2023
- Study start1 Jul 2020
- Primary completion31 May 2027 (estimated)
- Study completion31 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Patients With MDS, Acute Myeloid Leukemia (AML) Evolving From MDS, and Chronic Myelomonocytic Leukemia (CMML)Studies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 113 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Asan Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Je-Hwan Lee, Asan Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06098313
- Sponsor's protocol ID
- HRMDS_PTCy_2019
- Status last verified
- Oct 2023
- First submitted
- 19 Oct 2023
- Last updated
- 24 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 24 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Allogeneic Hematopoietic Cell Transplantation Using Post-transplantation Cyclophosphamide for Graft-versus-host Disease Prophylaxis in Higher-risk Myelodysplastic Syndrome, Chronic Myelomonocytic Leukemia, and Secondary AML Patients
- ClinicalTrials.gov ID
- NCT06098313
- Acronym / sponsor ID
- HRMDS_PTCy_2019
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 113 participants (estimated)
- Lead sponsor
- Asan Medical CenterUniversity, hospital or other
- Study start
- 1 Jul 2020 (actual)
- Primary completion
- 31 May 2027 (estimated)
- Study completion
- 31 May 2027 (estimated)
- First posted
- 24 Oct 2023 (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 study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
The secondary end points of the study include engraftment, relapse incidence, non-relapse mortality, graft-versus-host disease, donor chimerism, immune reconstitution, infections, and survivals (overall and event-free).
Detailed description
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
The secondary end points of the study include engraftment, relapse incidence, non-relapse mortality, graft-versus-host disease, donor chimerism, immune reconstitution, infections, and survivals (overall and event-free).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PTCy | Experimental | Drug: CyclophosphamidePatients who receive post-transplantation cyclophosphamide |
Who may qualify
- Ages
- 15 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- MDS defined by WHO classification, CMML, or AML evolving from MDS A. International Prognostic Scoring System (IPSS) > 1.0 or bone marrow blast ≥ 5% at any time points before HCT or B. AML progressed from MDS or C. CMML with bone marrow blast ≥ 5% at any time points before HCT
- Patients receiving first HCT
- Patients with appropriate hematopoietic cell donors A. HLA-matched sibling donor B. Unrelated donor C. HLA-mismatched familial donor
- 15 years old or older , under 75 years
- Adequate performance status (Karnofsky score of 70 or more)
Key exclusion criteria (7 listed)
- Presence of significant active infection
- Presence of uncontrolled bleeding
- Any coexisting major illness or organ failure
- Patients with psychiatric disorder or mental deficiency severe as to make compliance with the treatment unlike, and making informed consent impossible
- Nursing women, pregnant women, women of childbearing potential who do not want adequate contraception
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-Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2 days) | Drug | PTCy |
Primary outcome
GVHD-free, relapse-free survival
Time frame: 8 years
This study is conducted to evaluate the efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with higher-risk myelodysplastic syndrome (MDS). The efficacy of the treatment will be measured in terms of the GVHD-free, relapse-free survival.
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 24 Oct 2023.
- Individual participant dataSharing planned: Undecided
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cyclophosphamide in people with Patients With MDS, Acute Myeloid Leukemia (AML) Evolving From MDS, and Chronic Myelomonocytic Leukemia (CMML), run by Asan Medical Center at 1 site in South Korea.
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 10 inclusion and 7 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: under way, not looking for participants, as of the sponsor's last update on 24 Oct 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 South Korea. 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 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?
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?
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?
GVHD-free, relapse-free survival (time frame: 8 years). 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 24 Oct 2023.