Actively Recruiting
Hematopoietic Stem Cell Transplantation From Haploidentical Donors for Hematological Malignancies Using Treosulfan-Based Conditioning
Led by Fred Hutchinson Cancer Center · Updated on 2026-05-29
60
Participants Needed
1
Research Sites
99 weeks
Total Duration
On this page
Sponsors
F
Fred Hutchinson Cancer Center
Lead Sponsor
M
medac GmbH
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying how well a donor stem cell transplant combined with chemotherapy drugs treosulfan and fludarabine, plus total-body irradiation, works in treating patients with various blood cancers. This phase II trial aims to understand the effects of these treatments on stopping cancer growth and supporting the bone marrow to produce healthy blood and immune cells after transplant. The study focuses on patients with hematological malignancies, including leukemia and lymphoma, and is sponsored by Fred Hutchinson Cancer Center. Participants are assigned to one of two groups: one receives a high dose of treosulfan intravenously over three days, while the other receives a lower dose over the same period. Both groups receive fludarabine intravenously for five days, total-body irradiation, and a donor stem cell transplant. After the transplant, patients receive additional medications, including cyclophosphamide, cyclosporine, mycophenolate sodium or mofetil, and filgrastim to support immune recovery and prevent complications. Treatments are carefully timed before and after the transplant, with specific dosing schedules outlined for each medication. During the study, patients undergo various assessments such as bone marrow aspiration and biopsy, echocardiography, blood sample collection, and imaging scans like CT or PET/CT. Follow-up visits occur at multiple time points up to two years post-transplant to monitor graft success, survival, disease progression, and complications like graft-versus-host disease and infections. Researchers also track platelet recovery and overall health to evaluate the treatment's effects and safety over time.
CONDITIONS
Brief Title
Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Total-Body Irradiation for the Treatment of Hematological Malignancies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of acute leukemia (acute myeloid leukemia, acute lymphoblastic leukemia, mixed phenotype leukemia) in complete morphological remission
- Diagnosis of chronic myelogenous leukemia (except refractory blast crisis) with failure or intolerance to at least one tyrosine-kinase inhibitor
- Diagnosis of chronic myelomonocytic leukemia or myelodysplastic syndromes
- Diagnosis of lymphoblastic, Burkitt's, or other high-grade lymphoma in complete or partial response
- Diagnosis of low-grade lymphoma progressed after two treatments, in complete or partial response
- Diagnosis of large cell lymphoma in second or greater complete or partial response
- Diagnosis of mantle cell lymphoma, lymphoplasmacytic lymphoma, or prolymphocytic leukemia after initial therapy in complete or partial response
- Diagnosis of Hodgkin lymphoma in second or greater complete or partial response
- Age 6 months or older
- Karnofsky performance score 70 or higher or ECOG 0-1 for adults
- Lansky score 50 or higher for children
- Adequate cardiac function without decompensated heart failure or uncontrolled arrhythmia, and left ventricular ejection fraction 40% or higher or shortening fraction over 22%
- Adequate pulmonary function without oxygen requirement, with specified lung function test results or oxygen saturation 92% or higher on room air for children
- Total bilirubin less than twice the upper limit of normal unless related to Gilbert's disease or hemolysis
- AST and ALT less than or equal to 2.5 times upper limit of normal
- Alkaline phosphatase less than or equal to 5 times upper limit of normal
- Creatinine less than 2.0 mg/dl for adults or creatinine clearance over 40 ml/min for children
- Clearance by infectious disease if recent mold infection
- Prior allogeneic hematopoietic cell transplant allowed if at least 3 months prior or in case of graft failure
- Written informed consent
- Donor must be a haploidentical relative aged 12 years or older, weighing at least 40 kg, meeting blood bank and cell therapy guidelines
- Donor-recipient compatibility with at least 5/10 HLA loci shared
- Donor vein suitability for apheresis if under 18 years old
- Donor selection prioritizes CMV serostatus and red blood cell compatibility
You will not qualify if you...
- Active, uncontrolled, life-threatening infection requiring treatment at transplant conditioning
- Presence of another malignancy with less than 75% expected 5-year survival
- Pregnant or breastfeeding
- Known allergy to treosulfan, fludarabine, or cyclophosphamide
- Use of another investigational drug within 30 days before study entry
- Central nervous system leukemia not cleared by prior treatment before conditioning
- Presence of anti-donor-specific antibodies above study thresholds
- Initial trial participants required to be negative for donor-specific antibodies
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 6 weeks
Participants receive conditioning chemotherapy with treosulfan and fludarabine, followed by total-body irradiation and allogeneic hematopoietic stem cell transplantation. After transplant, participants receive cyclophosphamide for 2 days, then cyclosporine and mycophenolate sodium or mycophenolate mofetil starting on day 5. Filgrastim is given beginning on day 5 until neutrophil recovery.
Daily visits from day -6 to day 35 with medication administration and monitoring
Duration - Up to 2 years post-transplant
Participants are followed up for monitoring of transplant outcomes and complications including graft failure, survival, and graft versus host disease.
Visits at approximately days 28, 56, 84, 365, and 730 post-transplant
Trial Site Locations
Total: 1 location
1
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States, 98109
Actively Recruiting
Research Team
P
Phuong Vo, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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