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

StatusRecruiting
PhasePhase 2
Started2021-05-05
View on ClinicalTrials.gov ↗

Amendment history

2026-03-10
Study Status v35
Primary completion date2027-07-01→2028-07-01
Completion date2027-07-01→2028-07-01
2025-09-16
2 amendments v33-v34
2 re-verifications since 2025-03-28, no change to tracked fields
2024-09-05
Study Status, Study Description, Conditions, Arms and Interventions, Eligibility, Contacts/Locations v32
InterventionsAllogeneic Hematopoietic Stem Cell Transplantation (Procedure), Fludarabine Phosphate (Drug), Rabbit Anti-Thymocyte Globulin (Biological)…→Allogeneic Hematopoietic Stem Cell Transplantation (Procedure), Biospecimen Collection (Procedure), Bone Marrow Aspiration (Procedure)…
Eligibility criteria+1 characters
Inclusion Criteria: Patient with nonmalignant disease treatable by allogenicallogeneic HCT Patient with a nonmalignant disease that is not clearly [...]
Study description+159 characters
[...] ogeneic hematopoietic cell transplant via infusion on day 0. Patients may also undergo bone marrow biopsy and aspiration and magnetic resonance imaging (MRI) as clinically indicated and blood sample collection on study. After completion of study treatment, patients are followed up at 1 year and then annually thereafter.
Study sitescontacts updated at 1 of 1 site
2024-07-19
Study Status v31
Re-verified, no change to tracked fields
2024-02-01
Study Status, Contacts/Locations v30
Study sites2→1
Show 29 earlier versions
2024-01-30
Study Status v29
Re-verified, no change to tracked fields
2023-07-10
Study Status, Study Description, Arms and Interventions, Eligibility, Contacts/Locations v28
Eligibility criteria+30 characters
[...] not clearly defined (a patient with a non-malignant disease HCT for whom genetic testing has been done and a genetic mutation responsible for their non-malignant dise [...] cipal investigator (PI) (Dr. Lauri Burroughs) Age < 50 years. DONOR: Human leukocyte antigen (HLA)-identical related dono [...]
Study description+61 characters
OUTLINE: Patients receive thiotepa intravenously (IV) twice daily (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 hematopoi [...]
Study sitesdetails revised at 1 of 2 sites
2023-05-02
Study Identification, Study Status, Contacts/Locations v27
Study sitescontacts updated at 1 of 2 sites
2023-01-26
Study Status, Eligibility v26
Eligibility criteria+15 characters
[...] protocol principal investigator (PI) (Dr. Lauri Burroughs) Age <50 years. DONOR: Human leukocyte antigen (HLA)-identical related donor [...]
2022-10-25
2 amendments v24-v25
2 re-verifications since 2022-04-14, no change to tracked fields
2021-10-07
Study Identification, Study Status, Sponsor/Collaborators, Study Description, Outcome Measures, Eligibility v23
Eligibility criteria-928 characters
[...] protocol principal investigator (PI) (Dr. Lauri Burroughs) DONOR: For the very few occasions where we identify a donor hematopoietic progenitor cell (HPC)-A from a non-NMDP source, we have procedures in place through our unrelated donor office to collect the information necessary to comply with donor testing, screening, and declaration of donor eligibility [...] DONOR: Human leukocyte antigen (HLA)-identical related donor [...] as evidenced by carbon monoxide diffusing capability (DLCO) corrected < 50% of predicted (or, if unable to perform pulmonary func [...] ology Collaboration (CKD EPI) equation for adult p [...]
Secondary endpointsdetails revised at 1 of 6 entries
Collaborators-117 characters
National Cancer Institute (NCI) National Institutes of Health (NIH) National Heart, Lung, and Blood Institute (NHLBI)
2021-07-23
Study Status v22
Re-verified, no change to tracked fields
2021-05-12
Study Status v21
Start dateestimated 2021-05-20→confirmed 2021-05-05
2021-04-20
Study Status, Conditions, Outcome Measures v20
Start date2021-05-05→2021-05-20
Secondary endpoints5 to 6 entries
Event-free survival
2021-04-05
Study Status v19
Start date2021-04-05→2021-05-05
2021-03-15
Study Status v18
Re-verified, no change to tracked fields
2021-03-05
Study Status v17
Start date2021-03-05→2021-04-05
2021-02-05
Study Status v16
Start date2021-02-04→2021-03-05
2021-01-04
Study Status v15
Start date2021-01-04→2021-02-04
2020-12-03
Study Status v14
Start date2020-12-02→2021-01-04
2020-11-02
Study Status v13
Start date2020-11-01→2020-12-02
2020-10-01
Study Status v12
Start date2020-10-01→2020-11-01
2020-09-01
Not Yet Recruiting→Recruiting Study Identification, Study Status, Sponsor/Collaborators, Study Description, Conditions, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v11
Trial statusNot Yet Recruiting→Recruiting
Start date2020-09-01→2020-10-01
InterventionsAllogeneic Hematopoietic Stem Cell Transplantation (Procedure), Fludarabine (Drug), Fludarabine Phosphate (Drug), Rabbit Anti-Thymocyte…→Allogeneic Hematopoietic Stem Cell Transplantation (Procedure), Fludarabine Phosphate (Drug), Rabbit Anti-Thymocyte Globulin (Biological)…
Eligibility criteria+991 characters
Inclusion Criteria: Patient with nonmalignant disease treatable by allogenic HCT Patient with a known nonmalignant disease that is not clearly defined, if(a discussedpatient with a non-malignant disease HCT for whom 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) to determine if they are eligible for HCT on the study DONOR: For the very few occasions where we identify a donor [...] ening, and declaration of donor eligibil [...]
Secondary endpoints1 to 5 entries
TreatmentOverall survival Transplant-related mortality Incidence of grade II-IV acute graft-versus-host disease Incidence of chronic graft-versus-host disease Donor chimerism CD3 & CD33
Study description+18 characters
OUTLINE: Patients receive thiotepa intravenously (IV) twice daily (BID) on daysday -7, treosulfan IV on days -6 to -4, fludarabine phosphate IV on days -6 to -2, and rabbit anti-thymocyte globulin IV [...]
Study sites2 entries, revised
Froedtert and the Medical College of Wisconsin
Collaborators1 to 3 entries
National Institutes of Health (NIH) National Heart, Lung, and Blood Institute (NHLBI)
2020-08-19
Study Status v10
Re-verified, no change to tracked fields
2020-07-01
Study Status v9
Start date2020-07-01→2020-09-01
2020-06-01
Study Status v8
Start date2020-06-01→2020-07-01
2020-05-01
Study Status v7
Start date2020-05-01→2020-06-01
2020-04-01
Study Status v6
Start date2020-04-01→2020-05-01
2020-01-02
Study Status v5
Start date2020-01-01→2020-04-01
2019-11-04
Study Status v4
Start date2019-11-01→2020-01-01
2019-09-24
Study Status v3
Start date2019-09-19→2019-11-01
2019-08-19
Study Status v2
Start date2019-08-01→2019-09-19
2019-07-01
Study Status v1
Start date2019-06-30→2019-08-01
2019-06-06
Original filing
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.
Trial Details
NCT Number NCT03980769
Lead Sponsor Fred Hutchinson Cancer Center
Conditions Non-Neoplastic Hematopoietic and Lymphoid Cell Disorder
Enrollment 40 participants
Start Date 2021-05-05
Primary Completion 2028-07-01 (estimated)
Study Completion 2028-07-01 (estimated)
Updated on ClinicalTrials.gov 2026-09-16