Actively Recruiting

Phase 2
Age: 0 - 50Years
All Genders
NCT03980769

Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders

Led by Fred Hutchinson Cancer Center · Updated on 2026-03-12

40

Participants Needed

1

Research Sites

373 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This phase II clinical trial studies how well treosulfan, thiotepa, fludarabine, and rabbit anti-thymocyte globulin (rATG) before donor stem cell transplantation works in treating patients with nonmalignant (non-cancerous) diseases. Hematopoietic cell transplantation has been shown to be curative for many patients with nonmalignant (non-cancerous) diseases such as primary immunodeficiency disorders, immune dysregulatory disorders, hemophagocytic lymphohistiocytosis, bone marrow failure syndromes, and hemoglobinopathies. Powerful chemotherapy drugs are often used to condition the patient before infusion of the new healthy donor cells. The purpose of the conditioning therapy is to destroy the patient's abnormal bone marrow which doesn't work properly in order to make way for the new healthy donor cells which functions normally. Although effective in curing the patient's disease, many hematopoietic cell transplantation regimens use intensive chemotherapy which can be quite toxic, have significant side effects, and can potentially be life-threatening. Investigators are investigating whether a new conditioning regimen that uses less intensive drugs (treosulfan, thiotepa, and fludarabine phosphate) results in new blood-forming cells (engraftment) of the new donor cells without increased toxicities in patients with nonmalignant (non-cancerous) diseases.

CONDITIONS

Official Title

Donor Stem Cell Transplant With Treosulfan, Fludarabine, and Thiotepa in Treating Patients With Non-malignant Disorders

Who Can Participate

Age: 0 - 50Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patient with nonmalignant disease treatable by allogeneic hematopoietic cell transplantation (HCT)
  • Patient with a nonmalignant disease not clearly defined but approved by the principal investigator
  • Age less than 50 years
  • Donor must be HLA-identical related or unrelated matched for HLA-A, B, C, DRB1 and DQB1 or mismatched by a single allele at HLA-A, B, C, or DQB1
  • Preferred donor cell source is bone marrow; peripheral blood stem cells allowed if approved
  • Recommended nucleated cell count for bone marrow grafts is at least 4.0 x 10^8 TNC/kg recipient weight
  • Recommended CD34 cell count for peripheral blood stem cell grafts is between 5 x 10^6 and 10 x 10^6 CD34/kg recipient weight
  • Use of HLA-matched sibling bone marrow and umbilical cord blood if available and matched for HLA-A, B, C, DRB1 and DQB1
Not Eligible

You will not qualify if you...

  • Patients with idiopathic aplastic anemia or Fanconi anemia
  • Patients with aplastic anemia associated with paroxysmal nocturnal hemoglobinuria or inherited marrow failure syndromes (except Fanconi anemia) are allowed
  • Impaired cardiac function with ejection fraction below 35% or shortening fraction below 26% unless cardiologist approval
  • Cardiac insufficiency requiring treatment or symptomatic coronary artery disease
  • Impaired lung function with DLCO less than 50% predicted or oxygen saturation below 92% on room air
  • Impaired kidney function with creatinine clearance below 60 mL/min/1.73m^2 or dialysis dependence
  • Severe liver dysfunction or cirrhosis requiring deferral of conditioning
  • Active infections requiring deferral of conditioning
  • Positive for HIV infection
  • Pregnant or breastfeeding females
  • Known allergy to treosulfan, fludarabine, or thiotepa
  • Donors unable to undergo marrow harvesting or peripheral blood stem cell mobilization
  • Donors positive for HIV or active infectious hepatitis
  • Female donors with positive pregnancy test
  • Donors with patient-specific HLA antibodies
  • HLA-matched sibling cord blood units that fail infectious disease screening unless waived

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, United States, 98109

Actively Recruiting

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Research Team

L

Lauri Burroughs

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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