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Stem Cell Transplant with Conditioning Regimen for Blood Diseases

Phase 2

Recruiting

Led By Lauri Burroughs

Research Sponsored by Fred Hutchinson Cancer Research Center

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

The recommended total nucleated cell count (TNC) for bone marrow grafts is >= 4.0 x 10^8 TNC/kg (actual recipient weight)

DONOR: Human leukocyte antigen (HLA)-identical related donor OR unrelated donor matched for HLA-A, B, C, DRB1 and DQB1 or mismatched for a single allele at HLA-A, B, C, or a single DQB1 antigen or allele mismatch by high resolution deoxyribonucleic acid (DNA) typing

Must not have

Impaired pulmonary function as evidenced by carbon monoxide diffusing capability (DLCO) corrected < 50% of predicted (or, if unable to perform pulmonary function tests, then oxygen [O2] saturation < 92% on room air)

DONOR: Donors deemed unable to undergo marrow harvesting of PBSC mobilization and leukapheresis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at 1 year post-transplant

Awards & highlights

No Placebo-Only Group

Summary

This trial will study whether a new, less intensive conditioning regimen using the drugs treosulfan, thiotepa, and fludarabine phosphate is just as effective in treating patients with nonmalignant diseases as the current standard of care, which uses more powerful and potentially more toxic chemotherapy drugs.

Who is the study for?

This trial is for patients under 50 with non-cancerous blood diseases or conditions like immune disorders, who haven't found a genetic cause for their illness. They need a matching donor for stem cell transplant and must not have severe heart, lung, kidney issues, HIV, active hepatitis or be pregnant.

What is being tested?

The study tests a less toxic conditioning regimen before stem cell transplantation using drugs treosulfan, thiotepa, fludarabine phosphate and rATG to treat non-malignant diseases by replacing faulty bone marrow with healthy donor cells.

What are the potential side effects?

Potential side effects include allergic reactions to the medications used in the treatment regimen (treosulfan, fludarabine phosphate), damage to organs from powerful chemotherapy drugs and complications related to stem cell transplantation.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My bone marrow graft meets the required cell count.

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My donor matches me closely in immune system markers or has only one mismatch.

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I am under 50 years old.

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My stem cell graft has the right number of CD34 cells.

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I have a non-cancerous condition that can be treated with a stem cell transplant from a donor.

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I have a sibling donor whose bone marrow and umbilical cord blood match mine.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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My lung function is significantly reduced, as shown by tests or oxygen levels below 92%.

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I cannot have a bone marrow or stem cell collection procedure.

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I am a donor without active infectious hepatitis.

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My kidney function is impaired, with specific test results confirming it.

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I am a female donor and my pregnancy test is positive.

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I don't have antibodies against the donor's specific HLA.

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My liver is severely damaged, as confirmed by a specialist.

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I am not pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at 1 year post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at 1 year post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and at 1 year post-transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Engraftment failure

Secondary study objectives

Event-free survival

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment (chemotherapy, transplant)Experimental Treatment9 Interventions

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Bone Marrow Biopsy

2021

Completed Phase 3

~230

Biospecimen Collection

2004

Completed Phase 3

~2030