HCT With PTCy in Higher-risk MDS

NCT ID: NCT06098313

Last Updated: 2023-10-24

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 113 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-07-01
• Study Completion Date: 2027-05-31

Brief 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). 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).

Conditions

Patients With MDS, Acute Myeloid Leukemia (AML) Evolving From MDS, and Chronic Myelomonocytic Leukemia (CMML)

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PTCy

Patients who receive post-transplantation cyclophosphamide

Group Type: EXPERIMENTAL

Cyclophosphamide

Intervention Type: DRUG

-Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2 days)

Interventions

Cyclophosphamide

-Cyclophosphamide 50 mg/kg/day i.v. daily on days 3 and 4 (for 2 days)

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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).
• 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.

Exclusion Criteria

• 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)

• Minimum Eligible Age: 15 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Asan Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Je-Hwan Lee

M.D. Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

Asan Medical Center, University of Ulsan College of Medicine

Seoul, , South Korea

Countries

South Korea

Other Identifiers

HRMDS_PTCy_2019

Identifier Type: -

Identifier Source: org_study_id