Milder chemo may make stem cell transplants safer for Non-Cancer patients
NCT ID NCT03980769
First seen Jun 24, 2026 · Last updated Sep 17, 2026 · Updated 3 times
Summary
This phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditioning regimen can still allow the donor cells to take hold (engraft) while causing fewer toxic side effects than standard high-dose chemo. The study is currently recruiting.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- treosulfan, fludarabine, thiotepa, and rabbit anti-thymocyte globulin (rATG)
- What this could lead to
- If successful, this could offer a safer way to cure non-cancerous blood disorders using donor stem cells, with fewer severe side effects than standard high-dose chemotherapy.
- What could go wrong
- This is an early phase 2 trial with only 40 participants, so results may not apply to everyone. There are still risks of graft failure, infection, or other serious complications from the transplant.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 40 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2021
- Expected to finish
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Jul 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Up to 50 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patient with nonmalignant disease treatable by allogeneic HCT * Patient with a nonmalignant disease that is not clearly defined (a patient with a non-malignant disease for whom genetic testing has been done and a genetic mutation responsible for their non-malignant disease phenotype has not been identified) are eligible for the study following discussion with and approval by the protocol principal investigator (PI) (Dr. Lauri Burroughs) * Age \< 50 years * 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 * DONOR: Bone marrow is the preferred cell source (when feasible). However, peripheral blood stem cells (PBSC) is also allowed and the PI may determine if PBSC is preferred for certain patients * The recommended total nucleated cell count (TNC) for bone marrow grafts is \>= 4.0 x 10\^8 TNC/kg (actual recipient weight) * The recommended CD34 cell count for PBSC grafts is \>= 5 x 10\^6 CD34/kg (actual recipient weight) and the recommended maximum CD34 cell count for PBSC grafts is 10 x 10\^6 CD34/kg (actual recipient weight) * DONOR: HLA-matched sibling bone marrow in combination with HLA-matched sibling umbilical cord blood if the HLA-matched sibling umbilical cord blood was collected and stored. The HLA-matched sibling bone marrow and cord blood would be matched for HLA-A, B, C, DRB1 and DQB1 Exclusion Criteria: * Patients with idiopathic aplastic anemia and Fanconi anemia; patients with aplastic anemia associated with paroxysmal nocturnal hemoglobinuria (PNH) or inherited marrow failure syndromes (except Fanconi anemia) will be allowed * Impaired cardiac function as evidenced by ejection fraction \< 35% (or, if unable to obtain ejection fraction, shortening fraction of \< 26%) or cardiac insufficiency requiring treatment or symptomatic coronary artery disease. Patients with a shortening fraction of \< 26% may be enrolled if approved by a cardiologist * 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) * Impaired renal function as evidenced by: * Estimated creatinine clearance \< 60 mL/min/1.73m\^2 using either the Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) equation for adult patients (\>= 18 years old), or the updated Schwartz formula for pediatric patients (\< 18 years old). If the estimated creatinine clearance is \< 60 mL/min/1.73m\^2, then renal function must be measured by 24-hour creatinine clearance, Iothalamate, Iohexol or nuclear GFR and the patient is excluded if their measured creatinine clearance is \< 50 mL/min/1.73 m\^2, OR * Serum creatinine \> 2 x upper limit of normal, OR * Dialysis dependent * Evidence of synthetic dysfunction or severe cirrhosis requiring deferral of conditioning as recommended by a gastroenterology specialist * Active infectious disease requiring deferral of conditioning as recommended by an infectious disease specialist * Positive for HIV (human immunodeficiency virus) * Females who are pregnant or breast-feeding * Known hypersensitivity to treosulfan, fludarabine, and/or thiotepa * DONOR: Donors deemed unable to undergo marrow harvesting of PBSC mobilization and leukapheresis * DONOR: HIV-positive donors * DONOR: Donors with active infectious hepatitis * DONOR: Female donor with positive pregnancy test * DONOR: Donors are excluded if the patient has an identified antibody against a donor-specific HLA locus as specified in standard practice * DONOR: HLA-matched sibling cord blood units that have not passed donor screening for infectious disease markers as recommended by the National Marrow Donor Project (NMDP) will not be used unless a waiver is signed by the clinical attending allowing use of cord blood unit. Cord blood units are presumed to be cytomegalovirus (CMV) negative regardless of serologic testing due to passive transmission of maternal CMV antibodies
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Fred Hutch/University of Washington Cancer Consortium
RECRUITINGSeattle, Washington, 98109, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Chemotherapy plus immunotherapy tested against rare EBV-Driven immune storm
- Half-Matched stem cells tested as a cure for severe aplastic anemia
- New stem cell processing method offers hope for patients without a perfect donor match
- Suicide Gene-Equipped t cells aim to make stem cell transplants safer
- Engineered t cells with a kill switch aim to make stem cell transplants safer
- Half-Matched stem cell transplant offers hope for children with blood disorders