HCT With PTCy in Higher-risk MDS

NCT06098313 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: HRMDS_PTCy_2019
• Study Type: interventional
• Target: 113
• Locations: 1 country
• Sites: 1

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).

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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.

  8 years

Study Arms (1)

PTCy

EXPERIMENTAL

Patients who receive post-transplantation cyclophosphamide

Drug: Cyclophosphamide

Interventions

Cyclophosphamide DRUG

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

PTCy

Eligibility Criteria

• Age: 15 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Asan Medical Center: lead

Study Sites (1)

Asan Medical Center, University of Ulsan College of Medicine

Seoul, 05505, South Korea

Location

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Leukemia, Myelomonocytic, Chronic

Interventions

Cyclophosphamide

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic-Myeloproliferative Diseases; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: M.D. Professor

Study Record Dates

• First Submitted: October 19, 2023
• First Posted: October 24, 2023
• Study Start: July 1, 2020
• Primary Completion (Estimated): May 31, 2027
• Study Completion (Estimated): May 31, 2027
• Last Updated: October 24, 2023

Record last verified: 2023-10

Locations

KR (1)