Myelodysplastic SyndromesPhase 2NCT02969980
Hematopoietic Cell Transplantation With Post-transplantation Cyclophosphamide in MDS
A Phase 2 study testing Cyclophosphamide in people with Myelodysplastic Syndromes, run by Asan Medical Center at 1 site in South Korea.
Trial summary
This study is conducted to evaluate the feasibility and efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with myelodysplastic syndrome (MDS). Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- 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
- 30 participants
- Where
- 1 siteSouth Korea
- Study dates
- Nov 2016to 16 Dec 2021
- Phase
- Phase 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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- MDS defined by WHO classification, chronic myelomonocytic leukemia (CMML), or acute myeloid leukemia (AML) evolving from MDS A. IPSS > 1.0 or bone marrow blast ≥ 5% at any time points before HCT or B. AML progressed from MDS or C. CMML
- 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
- Adequate performance status (Karnofsky score of 70 or more)
- Adequate hepatic and renal function (AST, ALT, and bilirubin < 3.0 x upper normal limit, and creatinine < 2.0 mg/dL).
- Adequate cardiac function (left ventricular ejection fraction over 40% on heart scan or echocardiogram
- 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)
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: Allogeneic Hematopoietic Cell Transplantation Using Post-transplantation Cyclophosphamide in Myelodysplastic Syndrome Patients
This study is conducted to evaluate the feasibility and efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with myelodysplastic syndrome (MDS).
Read the detailed description
- Conditioning regimen A) Busulfan 3.2 mg/kg/day i.v. daily For 4 days (days -7 to -4) in patients aged < 55 years For 2 days (days -7 and -6) in patients aged ≥ 55 years B) Fludarabine 30 mg/m2/day i.v. daily on days -7 to -2 (for 6 days)
- Harvest and infusion of donor hematopoietic cells Harvested peripheral blood mononuclear cells of donors via leukapheresis will be infused to recipients on day 0. Additional infusion on day 1 can be made based on the judgement of attending physician.
- GVHD prophylaxis A) Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2 days) B) Cyclosporine: 1.5 mg/kg i.v. every 12 h hours beginning on day 5 and changed to oral dosing when oral intake is possible. If there is no evidence of GVHD, the dosage will be tapered beginning on day 90 and discontinued on day 180. C) MMF: 15 mg/kg p.o. 3 times daily with a maximum daily dose of 3.0 g beginning on day 5
If there is no active GVHD, Cyclosporine and MMF will be discontinued without taper at day 180 and 35, respectively.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Cyclophosphamide
- Keywords
- MDS, AML progressed from MDS, CMML
- Medical subject headings
- Myelodysplastic Syndromes
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 of hematopoietic cell transplantation
| 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.
the incidence of acute and chronic graft-versus-host disease
Measured: 100 days and 2 year, respectively
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2016 (estimated)
- Study startNov 2016
- Primary completion16 Dec 2021
- Study completion16 Dec 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Asan Medical CenterThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
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
- NCT02969980
- Sponsor's protocol ID
- MDS_PTCy_2016
- Status last verified
- Oct 2023
- First submitted
- 17 Nov 2016
- Last updated
- 23 Oct 2023
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Cell Transplantation Using Post-transplantation Cyclophosphamide in Myelodysplastic Syndrome Patients
- ClinicalTrials.gov ID
- NCT02969980
- Acronym / sponsor ID
- MDS_PTCy_2016
- Conditions
- Myelodysplastic Syndromes
- Keywords
- MDS, AML progressed from MDS, CMML
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Asan Medical CenterUniversity, hospital or other
- Study start
- Nov 2016
- Primary completion
- 16 Dec 2021 (actual)
- Study completion
- 16 Dec 2021 (actual)
- First posted
- 21 Nov 2016 (estimated)
- 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 feasibility and efficacy of post-transplantation cyclophosphamide with myeloablative or reduced-intensity conditioning regimen for allogeneic hematopoietic cell transplantation (HCT) in patients with myelodysplastic syndrome (MDS).
Detailed description
- Conditioning regimen A) Busulfan 3.2 mg/kg/day i.v. daily For 4 days (days -7 to -4) in patients aged < 55 years For 2 days (days -7 and -6) in patients aged ≥ 55 years B) Fludarabine 30 mg/m2/day i.v. daily on days -7 to -2 (for 6 days)
- Harvest and infusion of donor hematopoietic cells Harvested peripheral blood mononuclear cells of donors via leukapheresis will be infused to recipients on day 0. Additional infusion on day 1 can be made based on the judgement of attending physician.
- GVHD prophylaxis A) Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2 days) B) Cyclosporine: 1.5 mg/kg i.v. every 12 h hours beginning on day 5 and changed to oral dosing when oral intake is possible. If there is no evidence of GVHD, the dosage will be tapered beginning on day 90 and discontinued on day 180. C) MMF: 15 mg/kg p.o. 3 times daily with a maximum daily dose of 3.0 g beginning on day 5
If there is no active GVHD, Cyclosporine and MMF will be discontinued without taper at day 180 and 35, respectively.
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 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- MDS defined by WHO classification, chronic myelomonocytic leukemia (CMML), or acute myeloid leukemia (AML) evolving from MDS A. IPSS > 1.0 or bone marrow blast ≥ 5% at any time points before HCT or B. AML progressed from MDS or C. CMML
- 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
- Adequate performance status (Karnofsky score of 70 or more)
Key exclusion criteria (6 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 |
|---|---|---|
| CyclophosphamideCyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 of hematopoietic cell transplantation | Drug | PTCy |
Primary outcome
the incidence of acute and chronic graft-versus-host disease
Time frame: 100 days and 2 year, respectively
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 23 Oct 2023.
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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.
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 Myelodysplastic Syndromes, 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 8 inclusion and 6 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 23 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?
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?
the incidence of acute and chronic graft-versus-host disease (time frame: 100 days and 2 year, respectively). 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 23 Oct 2023.