Status:
COMPLETED
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Dyskeratosis Congenita
Aplastic Anemia
Eligibility:
All Genders
Up to 70 years
Phase:
PHASE2
PHASE3
Brief Summary
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic ane...
Detailed Description
This is an open label, single arm, phase II clinical trial designed to evaluate the safety and efficacy of the treatment regimen. Efficacy will be measured by long-term engraftment of the transplanted...
Eligibility Criteria
Inclusion
- Patients with dyskeratosis congenita (DC) or severe aplastic anemia (SAA) 0-70 years of age with an acceptable hematopoietic stem cell (HSC) donor
- HSC source
- Human leukocyte antigen (HLA) identical or 1 antigen mismatched sibling or other relative eligible to donate bone marrow (BM), umbilical cord blood (UCB) or mobilized peripheral blood (PB) at cell doses that meet current institutional standards.
- HLA identical or up to a 1 antigen mismatched unrelated donor.
- Two units of unrelated umbilical cord blood (UCB) that are (a) up to 2 HLA antigens mismatched to the patient (b) up to 2 HLA antigens mismatched to each other, (c) minimum cell dose of ≥ 3.5 x 10\^7 nucleated cells/kg and optimal cell dose ≥ 5 x 10\^7 nucleated cells/kg.
- If two units are not available: single unrelated UCB unit selected according to Minnesota Bone Marrow Transplant (BMT) program guidelines
- Disease Characteristics for DC (both of the following):
- Evidence of BM failure:
- Requirement for red blood cell and/or platelet transfusions,
- Requirement for granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF) or erythropoietin, or
- Refractory cytopenias defined as two out of three: platelets \<40,000/microliter (uL) or transfusion dependent, Absolute neutrophil count \<500/uL without hematopoietic growth factor support, Hemoglobin \<9g/uL or transfusion dependent
- Diagnosis of DC:
- A triad of mucocutaneous features: oral leukoplakia, nail dystrophy, abnormal reticular skin hyperpigmentation.
- Or one of the following: Short telomeres (under a research study), Dyskerin mutation, Telomerase RNA (TERC) mutation
- Disease Characteristics for SAA (both of the following):
- Evidence of BM failure:
- Refractory cytopenia defined by bone marrow cellularity \<25-50% (with \< 30% residual hematopoietic cells)
- Diagnosis of SAA:
- Refractory cytopenias defined as two out of three: Platelets \<20,000/uL or transfusion dependent, Absolute neutrophil count \<500/uL without hematopoietic growth factor support, Absolute reticulocyte count \<20,000/uL
- Patients with early myelodysplastic features.
- Patients with or without clonal cytogenetic abnormalities.
- Patient
Exclusion
- Patients with one or more of the following:
- Decompensated congestive heart failure; left ventricular ejection fraction \<35%
- Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
- Carbon Monoxide Diffusing Capacity (DLCO) \<30% predicted, and oxygen requirement
- Glomerular filtration rate (GFR) \<30% predicted
- Pregnant or lactating female
- Active serious infection whereby patient has been on intravenous antibiotics for at least one week prior to study entry. Any patient with AIDS or HIV seropositivity. If recent mold infection e.g. Aspergillus - must have \>30 days of appropriate treatment before HSC transplantation and infection must be controlled and cleared by the Infectious Disease consultant.
- Cannot receive total body irradiation (TBI) due to prior radiation therapy
- Diagnosis of Fanconi anemia based on diepoxybutane (DEB).
- DC patients with advanced myelodysplastic syndrome (MDS) or acute myeloid leukemia with \>30 blasts.
- History of non hematopoietic malignancy within 2 years except resected basal cell carcinoma or treated carcinoma in situ.
Key Trial Info
Start Date :
August 1 2007
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 1 2016
Estimated Enrollment :
36 Patients enrolled
Trial Details
Trial ID
NCT00455312
Start Date
August 1 2007
End Date
June 1 2016
Last Update
December 5 2017
Active Locations (1)
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1
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455