Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
NCT ID: NCT02162420
Last Updated: 2025-05-08
Study Results
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View full resultsBasic Information
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COMPLETED
NA
61 participants
INTERVENTIONAL
2015-01-10
2025-03-11
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Arm A Dyskeratosis Congenita (DKC) (non-haploidentical donor)
Fludarabine based preparative regimen, including alemtuzumab, cyclophosphamide, fludarabine, followed by stem cell transplant for the treatment of dyskeratosis congenita.
Alemtuzumab
Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.
Fludarabine
Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.
Cyclophosphamide
Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.
Stem Cell Transplant
Stem cell transplant on day 0.
Arm B: Severe Aplastic Anemia (SAA ) (non-matched related, non-haploidentical donor)
Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.
Alemtuzumab
Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.
Fludarabine
Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.
Cyclophosphamide
Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.
Total Body Irradiation
TBI 200 cGy as a single fraction on day -1 from transplant.
Stem Cell Transplant
Stem cell transplant on day 0.
Anti-thymocyte globulin
ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant.
Arm C: Severe Aplastic Anemia (matched related donor)
Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.
Alemtuzumab
Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.
Fludarabine
Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.
Cyclophosphamide
Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.
Total Body Irradiation
TBI 200 cGy as a single fraction on day -1 from transplant.
Stem Cell Transplant
Stem cell transplant on day 0.
Anti-thymocyte globulin
ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant.
Arm D: Dyskeratosis Congenita (DKC), PTCy platform
Fludarabine based preparative regimen, including alemtuzumab, cyclophosphamide, fludarabine, followed by stem cell transplant for the treatment of dyskeratosis congenita.
Alemtuzumab
Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.
Fludarabine
Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.
Cyclophosphamide
Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.
Stem Cell Transplant
Stem cell transplant on day 0.
Arm E: Severe Aplastic Anemia (SAA), PTCy platform
Fludarabine based preparative regimen which includes: cyclophosphamide, fludarabine, rabbit ATG and total body irradiation. Followed by stem cell transplant.
Alemtuzumab
Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.
Fludarabine
Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.
Cyclophosphamide
Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.
Total Body Irradiation
TBI 200 cGy as a single fraction on day -1 from transplant.
Stem Cell Transplant
Stem cell transplant on day 0.
Anti-thymocyte globulin
ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant.
Interventions
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Alemtuzumab
Alemtuzumab 0.2 mg/kg IV over 2 hours on days -10 to -6 from transplant.
Fludarabine
Fludarabine 40 mg/m2 IV over 1 hour on days -6 to -2 from transplant.
Cyclophosphamide
Cyclophosphamide 50 mg/kg IV over 2 hours on day -7 from transplant.
Total Body Irradiation
TBI 200 cGy as a single fraction on day -1 from transplant.
Stem Cell Transplant
Stem cell transplant on day 0.
Anti-thymocyte globulin
ATG (Thymoglobulin - Rabbit ) 3 mg/kg IV on days -5 to -3 from stem cell transplant.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Acceptable hematopoeitic stem cell donor
* Dyskeratosis Congenita (DC) with evidence of BM failure defined as:
* requirement for red blood cell and/or platelet transfusions or
* requirement for G-CSF or GM-CSF or erythropoietin or
* refractory cytopenias having one of the following three
* platelets \<50,000/uL or transfusion dependent
* absolute neutrophil count \<500/uL without hematopoietic growth factor support
* hemoglobin \<9g/uL or transfusion dependent
* Diagnosis of DC with a triad of mucocutaneous features:
* oral leukoplakia
* nail dystrophy
* abnormal reticular skin hyperpigmentation, or
* Diagnosis of DC with one of the following:
* short telomeres (under a research study)
* mutation in telomerase holoenzyme (DKC1, TERT, TERC, NOP10, NHP2, TCAB1)
* mutation in shelterin complex (TINF2)
* mutation in telomere-capping complex (CTC1)
* Severe Aplastic Anemia (SAA) primary transplant with evidence of BM failure:
* Refractory cytopenia defined by bone marrow cellularity \<50% (with \< 30% residual hematopoietic cells)
* Diagnosis of SAA with refractory cytopenias having one of the following three:
* platelets \<20,000/uL or transfusion dependent
* absolute neutrophil count \<500/uL without hematopoietic growth factor support
* absolute reticulocyte count \<20,000/uL
* Severe Aplastic Anemia (SAA) requiring a 2nd transplant
* Graft failure as defined by blood/marrow chimerism of \< 5%
* Early myelodysplastic features
* With or without clonal cytogenetic abnormalities
* Adequate organ function defined as:
* cardiac: left ventricular ejection fraction ≥ 35% with no evidence of decompensated heart failure
* pulmonary: DLCO ≥30% predicted, no supplemental oxygen requirement
* renal: Glomerular filtration rate (GFR) ≥30% predicted
* Voluntary written consent
Exclusion Criteria
* Pregnant or lactating
* Uncontrolled infection
* Prior radiation therapy (applies to SAA patients only)
* Diagnosis of Fanconi anemia based on DEB
* Diagnosis of dyskeratosis congenita with advanced MDS or acute myeloid leukemia with \>30% blasts
0 Years
70 Years
ALL
No
Sponsors
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Masonic Cancer Center, University of Minnesota
OTHER
Responsible Party
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Locations
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University of Minnesota Medical Center, Fairview
Minneapolis, Minnesota, United States
Countries
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Document Type: Informed Consent Form
Other Identifiers
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MT2013-34C
Identifier Type: OTHER
Identifier Source: secondary_id
2013OC127
Identifier Type: -
Identifier Source: org_study_id
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