Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
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| ClinicalTrials.gov Identifier: NCT04965597 |
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Recruitment Status :
Recruiting
First Posted : July 16, 2021
Last Update Posted : September 13, 2023
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Sponsor:
Fred Hutchinson Cancer Center
Collaborators:
Blood and Marrow Transplant Clinical Trials Network
National Cancer Institute (NCI)
National Marrow Donor Program
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Fred Hutchinson Cancer Center
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Brief Summary:
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure diseases. Chemotherapy drugs, such as treosulfan, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Fludarabine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. rATG is used to decrease the body's immune response and may improve bone marrow function and increase blood cell counts. Adding treosulfan to a conditioning regimen with fludarabine and rATG may result in patients having less severe complications after a blood or bone marrow transplant.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Bone Marrow Failure Syndrome Congenital Amegakaryocytic Thrombocytopenia Congenital Pure Red Cell Aplasia Hereditary Sideroblastic Anemia Myeloid Neoplasms With Germline GATA2 Mutation Paroxysmal Nocturnal Hemoglobinuria Shwachman-Diamond Syndrome | Drug: Treosulfan Drug: Fludarabine Phosphate Drug: Tacrolimus Drug: Methotrexate Biological: Lapine T-Lymphocyte Immune Globulin Procedure: Peripheral Blood Stem Cell Transplantation Procedure: Allogeneic Bone Marrow Transplantation Other: Quality-of-Life Assessment | Phase 2 |
OUTLINE:
CONDITIONING REGIMEN: Patients receive treosulfan intravenously (IV) over 120 minutes on days -6 to -4, fludarabine phosphate IV over 60 minutes on days -6 to -2, and lapine T-lymphocyte immune globulin (rATG) IV over 4-6 hours on days -4 to -2.
TRANSPLANTATION: Patients undergo bone marrow or peripheral blood stem cell transplant on day 0.
GVHD PROPHYLAXIS: Patients receive tacrolimus IV continuously beginning on day -2 and a taper beginning on day 180. Patients may also receive tacrolimus orally (PO). Patients also receive methotrexate IV on days 1, 3, 6, and 11.
After completion of study treatment, patients are followed up at 1 year from transplant.
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 40 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases |
| Actual Study Start Date : | April 19, 2022 |
| Estimated Primary Completion Date : | December 2025 |
| Estimated Study Completion Date : | December 2026 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Shwachman-Diamond syndrome
| Arm | Intervention/treatment |
|---|---|
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Experimental: Treatment (conditioning regimen; transplant; GVHD prophylaxis)
CONDITIONING REGIMEN: Patients receive treosulfan IV over 120 minutes on days -6 to -4, fludarabine phosphate IV over 60 minutes on days -6 to -2, and rATG IV over 4-6 hours on days -4 to -2. TRANSPLANTATION: Patients undergo bone marrow or peripheral blood stem cell transplant on day 0. GVHD PROPHYLAXIS: Patients receive tacrolimus IV continuously beginning on day -2 and a taper beginning on day 180. Patients may also receive tacrolimus PO. Patients also receive methotrexate IV on days 1, 3, 6, and 11. |
Drug: Treosulfan
Given IV
Other Names:
Drug: Fludarabine Phosphate Given IV
Other Names:
Drug: Tacrolimus Given IV and PO
Other Names:
Drug: Methotrexate Given IV
Other Names:
Biological: Lapine T-Lymphocyte Immune Globulin Given IV
Other Names:
Procedure: Peripheral Blood Stem Cell Transplantation Undergo PBSC
Other Names:
Procedure: Allogeneic Bone Marrow Transplantation Undergo bone marrow transplant
Other Names:
Other: Quality-of-Life Assessment Ancillary studies
Other Name: Quality of Life Assessment |
Primary Outcome Measures :
- Graft-Versus Host-Disease (GVHD)-Free Event-Free Survival (EFS) [ Time Frame: 1 year post-HCT ]The primary endpoint is the incidence of 1-year GVHD free, EFS (GEFS). An event is defined as death due to any cause, graft rejection/failure, or 2nd HCT whichever occurs first. Grade III-IV acute GVHD and chronic GVHD (using NIH consensus criteria) requiring systemic immune suppression will be considered in this estimate
Secondary Outcome Measures :
- Overall Survival [ Time Frame: Day 100 post-HCT ]Overall survival at day 100 after HCT
- Overall Survival [ Time Frame: 6 months post-HCT ]Overall survival at 6 months after HCT
- Overall Survival [ Time Frame: 1 year post-HCT ]Overall survival at 1 year after HCT
- Event-Free Survival [ Time Frame: 1 year post-HCT ]Event-free survival will be estimated at 12 months after HCT. Grade III-IV acute GVHD and chronic GVHD requiring systemic immune suppression will be considered in this estimate. An event is defined as death due to any cause, primary or secondary graft failure/rejection, or 2nd HCT whichever occurs first
- Hematologic Recovery: Neutrophil recovery [ Time Frame: Assessed up to 1 year post-HCT ]Hematologic recovery will be assessed according to neutrophil recovery after transplant. Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) ≥ to 500/mm3 for 3 consecutive measurements on 3 different days. The first of the three days will be designated the day of neutrophil recovery. The competing event is death without neutrophil recovery.
- Hematologic Recovery: Platelet recovery [ Time Frame: Day 100 post-HCT ]Hematologic recovery will be assessed according to platelet counts recovery after transplant. Platelet recovery is defined as the first day of a minimum of 3 days that the patient has a sustained platelet count ≥ 20,000/mm3 with no platelet transfusions in the preceding 7 days. The first day of sustained platelet count above these thresholds will be designated the day of platelet engraftment.
- Donor Chimerism (CD3 and Myeloid) [ Time Frame: Day 28 post-HCT ]Proportions of patients with full (>95%), mixed (5-95%), or rejection (<5%) myeloid
- Donor Chimerism (CD3 and Myeloid) [ Time Frame: Day 100 post-HCT ]Proportions of patients with full (>95%), mixed (5-95%), or rejection (<5%) myeloid
- Donor Chimerism (CD3 and Myeloid) [ Time Frame: 1 year post-HCT ]Proportions of patients with full (>95%), mixed (5-95%), or rejection (<5%) myeloid
- Primary graft failure/rejection [ Time Frame: Day 42 post-HCT ]Defined as never achieving ANC ≥ 500/μL or never achieving ≥ 5% donor myeloid chimerism assessed by peripheral blood chimerism assays by day +42 post-HCT. Second infusion of hematopoietic cells is also considered indicative of primary graft failure by day +42 post-HCT
- Secondary graft failure/rejection post-HCT [ Time Frame: Assessed up to 1 year post-HCT ]Defined as < 5% donor myeloid chimerism in peripheral blood beyond day +42 post-HCT in patients with prior documentation of hematopoietic recovery with ≥ 5% donor cells by day +42 post-HCT. Second infusion of hematopoietic cells is also considered indicative of secondary graft failure.
- Grade II-IV and grade III-IV GVHD at day 100 [ Time Frame: Day 100 post-HCT ]The cumulative incidences of acute grade II-IV and III-IV GVHD at day 100 after HCT will be determined.
- Grade II-IV and grade III-IV GVHD at day 180 [ Time Frame: Day 180 post-HCT ]The cumulative incidences of acute grade II-IV and III-IV GVHD at day 100 after HCT will be determined.
- Chronic GVHD [ Time Frame: 1 year post-HCT ]The cumulative incidence of chronic GVHD (using NIH consensus criteria) requiring systemic immune suppression at 1 year after HCT will be determined. Data will be collected directly from providers and chart review as defined by the NIH Consensus Conference Criteria
- Incidence of grade 3-5 toxicities [ Time Frame: Day 30 post-HCT ]Grade 3-5 toxicities by day 30 after HCT
- Incidence of grade 3-5 toxicities [ Time Frame: Day 100 post-HCT ]Grade 3-5 toxicities by day 100 after HCT
- Incidence of grade 2-3 systemic infections [ Time Frame: 6 months post-HCT ]All microbiologically documented infections or significant infections requiring antibiotic/antifungal therapy occurring up to 6 months after HCT
- Incidence of Epstein Barr virus (EBV) reactivation requiring therapy [ Time Frame: Day 180 post-HCT ]The incidence of EBV reactivation requiring therapy in the first 180 days after HCT, and of EBV-associated lymphoproliferative disorder in the first 180 days after HCT will be evaluated.
- Incidence of EBV-associated lymphoproliferative disorder [ Time Frame: Day 180 post-HCT ]
- Incidence of cytomegalovirus (CMV) reactivation requiring therapy by day 180 post-HCT [ Time Frame: Up to day 180 post-HCT ]
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| Ages Eligible for Study: | 1 Year to 49 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Patient must be >= 1.0 year of age and less than 50.0 years of age at the time of enrollment (i.e. patient must have celebrated their 1st birthday when enrolled and must NOT have celebrated their 50th birthday when enrolled; 49.99 years)
- Underlying BMFD treatable by allogenic HCT
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Shwachman-Diamond syndrome
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Criteria for Diagnosis:
- A pathogenic mutation(s) for Shwachman-Diamond syndrome
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For those patients tested but lacking a genetic mutation they must meet both **** criteria below:
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Exocrine pancreatic dysfunction as defined by at least one of the following:
- Pancreatic isoamylase below normal (age >= 3 years old), OR
- Fecal elastase < 200, AND
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Bone marrow failure as evidence by at least one of the following:
- Intermittent or persistent neutropenia (absolute neutrophil count < 1,500/uL), OR
- Hypo-productive anemia with a hemoglobin concentration below the age-related adjusted norms, OR
- Unexplained macrocytosis, OR
- Platelet count < 150,000/uL without alternative etiology, OR
- Hypocellular bone marrow
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Indications for HCT:
- Severe neutropenia (absolute neutrophil count [ANC] < 500/uL), OR
- Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia, OR
- Severe thrombocytopenia (platelet count < 20,000/uL) or transfusion-dependent thrombocytopenia, OR
- Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 eligibility review committee (ERC). In addition, patients with severe or recurrent infections will be reviewed by the ERC if they do not meet the indications for transplant listed above
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Diamond Blackfan Anemia
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Criteria for Diagnosis:
- A pathogenic mutation for Diamond Blackfan anemia
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For those patients tested but lacking a genetic mutation the patient must meet the first *** criteria and at least one of the subsequent *** criteria listed below:
- History of deficiency of erythroid precursors in an otherwise cellular bone marrow AND,
- Reticulocytopenia, OR
- Elevated adenosine deaminase activity, OR
- Elevated hemoglobin F, OR
- Macrocytosis, OR
- Congenital anomalies
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Indications for HCT:
- Red blood cell (RBC) transfusion dependent anemia despite an adequate trial of steroids; OR
- Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC
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Congenital Sideroblastic anemia
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Criteria for Diagnosis:
- A pathogenic mutation(s) for sideroblastic anemia
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For those patients tested but lacking a genetic mutation:
- Presence of ringed sideroblasts in the bone marrow excluding acquired causes of ringed sideroblasts such as lead poisoning & zinc toxicity
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Indications for HCT:
- Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia OR
- Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC
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GATA2 mutation with associated marrow failure
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Criteria for Diagnosis:
** A pathogenic mutation(s) for GATA2
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Indications for HCT:
- Severe neutropenia (ANC < 500/uL), OR
- Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia, OR
- Severe thrombocytopenia (platelet count < 20,000/uL) or transfusion-dependent thrombocytopenia, OR
- Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC. In addition, patients with severe or recurrent infections will be reviewed by the ERC if they do not meet indications for transplant listed above
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SAMD9 or SAMD9L disorders
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Criteria for Diagnosis:
** A pathogenic mutation(s) for SAMD9 or SAMD9L
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Indications for HCT:
- Severe neutropenia (ANC < 500/uL), OR
- Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia, OR
- Severe thrombocytopenia (platelet count < 20,000/uL) or transfusion-dependent thrombocytopenia, OR
- Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC
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Congenital amegakaryocytic thrombocytopenia
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Criteria for Diagnosis:
- A pathogenic mutation(s) for congenital amegakaryocytic thrombocytopenia.
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For those patients tested but lacking a genetic mutation the patient must meet criteria below:
- Thrombocytopenia early in life, AND
- History of bone marrow demonstrating megakaryocyte hypoplasia
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Indications for HCT:
- Severe thrombocytopenia (platelet count < 20,000/uL) or transfusion-dependent thrombocytopenia, OR
- Neutropenia defined as an ANC < 500/uL, OR
- Severe anemia (hemoglobin < 8 g/dL) or transfusion-dependent anemia, OR
- Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC
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Paroxysmal nocturnal hemoglobinuria
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Criteria for Diagnosis:
- Paroxysmal nocturnal hemoglobinuria (PNH) clone size in granulocytes >= 10%, AND
- Complement mediated intravascular hemolysis with an elevated LDH (above institutional upper limits of normal)
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Indications for HCT:
- PNH with thrombosis despite adequate medical management, OR
- PNH with intravascular hemolysis requiring transfusion support despite adequate medical management, OR
- Additional clinical or laboratory data may be considered for protocol eligibility following review by protocol 1904 ERC. In addition, patients with PNH and cytopenias may be considered for the protocol eligibility following review by protocol 1904 ERC
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An undefined BMFD: a patient with a BMFD for whom a genetic mutation responsible for their bone marrow failure phenotype has not been identified (excluding PNH) will be eligible for this clinical trial following approval by Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 1904 ERC
* A BMFD with a known genetic mutation but not listed above will be eligible for this clinical trial following approval by BMT CTN 1904 ERC
- Patient and/or legal guardian must sign informed consent prior to initiation of conditioning for BMT CTN 1904
- Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence
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Note: The following patients MUST be reviewed by the BMT CTN 1904 ERC in order to determine if they are eligible for this trial:
- All patients with Shwachman-Diamond syndrome, Diamond Blackfan anemia, congenital sideroblastic anemia, and congenital amegakaryocytic thrombocytopenia who have had genetic testing and lack a genetic mutation
- All patients with an undefined BMFD: a patient with a BMFD for whom a genetic mutation responsible for their bone marrow failure phenotype has not been identified, excluding PNH
- All patients with a BMFD and a known genetic mutation that is not listed above
- All patients with GATA2 mutation and associated marrow failure
- All patients with SAMD9 or SAMD9L disorders
- There may be circumstances where a treating physician will consider a transplant for a patient with a BMFD who does not meet all the criteria listed under "indications for HCT". In these situations, treating physicians may submit their patient to the BMT CTN 1904 ERC for review in order to determine if the patient is eligible for this clinical trial based on additional clinical or laboratory information
- Many patients with BMFD can have bone marrow evaluations that raise concern for possible myelodysplastic syndrome (MDS) including but not limited to dysplastic bone marrow evaluations or cytogenetic abnormalities. However, in patients BMFD these findings are not necessarily diagnostic or consistent with MDS. Therefore, given the complexities of diagnosing MDS in patients with BMFD, all patients with bone marrow evaluations concerning for possible MDS should be submitted to the ERC for review to confirm or exclude MDS. This is particularly important as we do not want to exclude potentially eligible patients due to an incorrect diagnosis of MDS
- HLA-MATCHED RELATED DONOR: HLA-matched sibling: Must be a minimum HLA-6/6 matched to the recipient at HLA-A, -B (serologic typing) and DRB1 (high-resolution typing)
- HLA-MATCHED RELATED DONOR: HLA-matched related (phenotypic match): Fully matched for HLA-A, -B, -C, -DRB1, and DQB1 by high-resolution typing.
- HLA-MATCHED RELATED DONOR: If a genetic mutation is known for the patient, the HLA-matched related donor [either HLA-matched sibling or HLA-matched related (phenotypic match)] must be screened for the same genetic mutation if clinically appropriate and should be confirmed to not have the same genetic disease (this does not include patients with PNH). Consult the protocol team with questions
- HLA-MATCHED RELATED DONOR: If a patient has an undefined BMFD (a patient with a BMFD for whom a genetic mutation responsible for their bone marrow failure phenotype has not been identified), the HLA-matched related donor [either HLA-matched sibling or HLA-matched related (phenotypic match)] must have an evaluation as directed by the treating physician to confirm that the donor does not have the same underlying disease. This will include a complete blood count (CBC) with differential and potentially a bone marrow evaluation or other studies as directed by the treating physician
- UNRELATED DONOR: Fully matched for HLA-A, -B, -C, -DRB1, and DQB1 by high-resolution typing
- UNRELATED DONOR: Mismatched for a single HLA-class 1 allele (HLA-A, -B, or -C) by high-resolution typing; OR
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UNRELATED DONOR: Mismatched for a single HLA DQB1 allele or antigen by high-resolution typing
* Note: donor patient (DP) matching per institutional practice
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DONOR SELECTION RECCOMENDATIONS: in the case where there are multiple donor options, donors should be selected based on the following priority numbered below:
- Unaffected fully HLA-matched sibling
- Unaffected fully phenotypically HLA-matched related donor
- Fully HLA-matched unrelated donor
- Unrelated donor with single allele or antigen level mismatch at DQB1
- Unrelated donor with single allele level mismatch at class 1 (HLA-A, -B, or -C)
Exclusion Criteria:
- Patients with idiopathic aplastic anemia, Fanconi anemia, dyskeratosis congenita, and congenital neutropenia
- Patients with MDS as defined by the World Health Organization (WHO) or leukemia
- Prior allogeneic HCT
- Patient's weight =< 10.0 kg (actual body weight and adjusted body weight) at time of study enrollment
- Lansky (patients < 16 years of age) or Karnofsky (patients >= 16 years of age) performance < 70%
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Left ventricular ejection fraction < 50% by echocardiogram or multi-gated acquisition (MUGA) scan
* For patients unable to obtain a left ventricular ejection fraction, left ventricular shortening fraction of < 26%
- Diffusing capacity of the lungs for carbon monoxide (DLCO) (corrected/adjusted for hemoglobin) < 50%, forced expiratory volume (FEV)1 < 50% predicted, and forced vital capacity (FVC) < 50% predicted
- For patients unable to perform pulmonary function tests (PFTs) due to age or developmental delay: oxygen (O2) saturation < 92% on room air
- On supplemental oxygen
- Estimated creatinine clearance < 60 mL/minute/1.73m^2 (estimated per institutional practice)
- Dialysis dependent
- Conjugated bilirubin > 2 x ULN for age (upper limit of normal [ULN], unless attributable to Gilbert's syndrome)
- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 4 x ULN for age, or
- Fulminant liver failure or cirrhosis
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Iron overload - This exclusion criterion only applies to patients who are considered at risk for hepatic or cardiac iron overload. Therefore, not all patients enrolled on this protocol will undergo formal hepatic or cardiac iron assessment
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For patients >= 18 years with a history of significant transfusions defined as >= 8 packed red blood cell transfusions per year for >= 1 year or have received >= 20 packed red blood cell transfusions (lifetime cumulative) will require formal hepatic and cardiac iron measurement. In addition, patients with a prior history of hepatic or cardiac iron overload will also require formal assessment for iron overload. Patients are excluded if:
- Hepatic iron content >= 8 mg Fe/g dry weight by liver magnetic resonance imaging (MRI) using a validated methodology (such as T2 * MRI or ferriscan) or liver biopsy per institutional practice
- Cardiac iron content < 25 msec by cardiac T2 * MRI
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For patients < 18 years old with a history of significant transfusions defined as >= 8 packed red blood cell transfusions per year for >= 1 year or have received >= 20 packed red blood cell transfusions (lifetime cumulative) will require formal hepatic iron measurement. In addition, patients with a prior history of liver iron overload will also require formal assessment for iron overload. Patients are excluded if:
- Hepatic iron content >= 8 mg Fe/g dry weight by liver MRI using a validated methodology (such as T2 * MRI or ferriscan) or liver biopsy per institutional practice
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- Uncontrolled bacterial infection within 1 week of study enrollment. Uncontrolled is defined as currently taking medication with no clinical improvement or progression on adequate medical treatment
- Uncontrolled viral or fungal infection within 30 days of study enrollment. Uncontrolled is defined as currently taking medication with no clinical improvement or progression on adequate medical treatment
- Positive for human immunodeficiency virus (HIV)
- Presence of clinically significant anti-donor human leukocyte antigen (HLA)-antibodies per institutional practice
- Prior solid organ transplant
- Patients with prior malignancies except resected non-melanoma skin cancer or treated cervical carcinoma in situ
- Demonstrated lack of compliance with prior medical care as determined by referring physician
- Females who are pregnant or breast-feeding
- Known hypersensitivity to treosulfan or fludarabine
- Known life-threatening reaction (i.e. anaphylaxis) to Thymoglobulin that would prohibit use for the patient as this study requires use of the Thymoglobulin preparation of anti-thymocyte globulin (ATG)
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04965597
Contacts
| Contact: Megan Scott | 240-599-5648 ext 15648 | bmtctn1904@emmes.com | |
| Contact: Adam Mendizabal, PhD | amendizabal@emmes.com |
Locations
Show 24 study locations
Sponsors and Collaborators
Fred Hutchinson Cancer Center
Blood and Marrow Transplant Clinical Trials Network
National Cancer Institute (NCI)
National Marrow Donor Program
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
| Study Chair: | Lauri Burroughs, MD | Fred Hutch/University of Washington Cancer Consortium | |
| Study Chair: | Margaret MacMillan, MD | University of Minnesota |
Additional Information:
| Responsible Party: | Fred Hutchinson Cancer Center |
| ClinicalTrials.gov Identifier: | NCT04965597 |
| Other Study ID Numbers: |
BMTCTN1904 2U10HL069294-11 ( U.S. NIH Grant/Contract ) RG1121820 ( Other Identifier: Fred Hutch/University of Washington Cancer Consortium ) 10840 ( Other Identifier: Fred Hutch/University of Washington Cancer Consortium ) NCI-2021-13862 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) ) |
| First Posted: | July 16, 2021 Key Record Dates |
| Last Update Posted: | September 13, 2023 |
| Last Verified: | September 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Fred Hutchinson Cancer Center:
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Bone Marrow Failure Disorders HSCT Treosulfan Unrelated donor |
Matched donor mismatched donor transplant |
Additional relevant MeSH terms:
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Hemoglobinuria Hemoglobinuria, Paroxysmal Shwachman-Diamond Syndrome Thrombocytopenia Bone Marrow Failure Disorders Pancytopenia Red-Cell Aplasia, Pure Anemia, Diamond-Blackfan Anemia, Sideroblastic Anemia, Hypochromic Syndrome Disease Pathologic Processes Blood Platelet Disorders Hematologic Diseases |
Proteinuria Urination Disorders Urologic Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Urogenital Diseases Male Urogenital Diseases Urological Manifestations Anemia, Hemolytic Anemia Myelodysplastic Syndromes Bone Marrow Diseases Exocrine Pancreatic Insufficiency Pancreatic Diseases Digestive System Diseases |