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

NCT03980769 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2026-03-12

No results posted yet for this study

Summary

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

  • Non-Neoplastic Hematopoietic and Lymphoid Cell Disorder

Interventions

BIOLOGICAL

Rabbit Anti-Thymocyte Globulin

Given IV

PROCEDURE

Allogeneic Hematopoietic Stem Cell Transplantation

Undergo HCT via infusion

PROCEDURE

Bone Marrow Biopsy

Undergo bone marrow biopsy

PROCEDURE

Bone Marrow Aspiration

Undergo bone marrow aspiration

PROCEDURE

Magnetic Resonance Imaging

Undergo MRI

PROCEDURE

Biospecimen Collection

Undergo blood sample collection

DRUG

Thiotepa

Given IV

DRUG

Treosulfan

Given IV

DRUG

Fludarabine Phosphate

Given IV

Sponsors & Collaborators

  • Fred Hutchinson Cancer Center

    lead OTHER

Principal Investigators

  • Lauri Burroughs · Fred Hutch/University of Washington Cancer Consortium

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-05-05
Primary Completion
2028-07-01
Completion
2028-07-01
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03980769 on ClinicalTrials.gov