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

Part of paid clinical trials in Seattle, Washington.

Sponsor
Fred Hutchinson Cancer Center
Study ID
NCT03980769
Phase
PHASE2
Status
Recruiting
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Conditions

  • Non-Neoplastic Hematopoietic and Lymphoid Cell Disorder

Eligibility Criteria

Sex
ALL
Age
N/A - 50 Years
Healthy Volunteers
Not accepted

Interventions

  • Thiotepa — DRUG
    Given IV
  • Treosulfan — DRUG
    Given IV
  • Fludarabine Phosphate — DRUG
    Given IV
  • Rabbit Anti-Thymocyte Globulin — BIOLOGICAL
    Given IV
  • Allogeneic Hematopoietic Stem Cell Transplantation — PROCEDURE
    Undergo HCT via infusion
  • Bone Marrow Biopsy — PROCEDURE
    Undergo bone marrow biopsy
  • Bone Marrow Aspiration — PROCEDURE
    Undergo bone marrow aspiration
  • Magnetic Resonance Imaging — PROCEDURE
    Undergo MRI
  • Biospecimen Collection — PROCEDURE
    Undergo blood sample collection

Study Details

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.

Key Dates

Start date
May 5, 2021
Status verified
Mar 2026
Primary completion
Jul 1, 2028
Completion
Jul 1, 2028

Study Design

Enrollment
40 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment (chemotherapy, transplant)
    Patients receive thiotepa IV BID over 2 hours on day -7, treosulfan IV over 120 minutes on days -6 to -4, fludarabine phosphate IV over 60 minutes on days -6 to -2, and rabbit anti-thymocyte globulin IV over 4-6 hours on days -4 to -2. Patients then undergo allogeneic hematopoietic cell transplant via infusion on day 0. Patients may also undergo bone marrow biopsy and aspiration and MRI as clinically indicated and blood sample collection on study.

Primary Outcome Measure

Engraftment failure [ Time Frame: 1 year after transplant ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Fred Hutch/University of Washington Cancer ConsortiumSeattleWashington98109
Lauri Burroughs
[email protected]
206-667-7385
Lauri Burroughs (PRINCIPAL_INVESTIGATOR)

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By research site
Fred Hutch/University of Washington Cancer Consortium· Seattle, WA

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