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Trial record 12 of 16 for:    "Congenital Hypoplastic Anemia" | "Fludarabine"

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

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ClinicalTrials.gov Identifier: NCT02179359
Recruitment Status : Recruiting
First Posted : July 1, 2014
Last Update Posted : September 25, 2018
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Tracking Information
First Submitted Date  ICMJE June 27, 2014
First Posted Date  ICMJE July 1, 2014
Last Update Posted Date September 25, 2018
Actual Study Start Date  ICMJE September 2, 2014
Estimated Primary Completion Date August 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 27, 2014)
incidence of graft failure [ Time Frame: 42 days ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02179359 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 27, 2014)
  • overall survival [ Time Frame: 6 months, 1 and 2 years ]
  • disease free survival [ Time Frame: 6 months, 1 and 2 years ]
    patient no longer needing red blood cell transfusion and/or a hemoglobin S level at that of the donor ( sickle cell disease only)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
Official Title  ICMJE MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
Brief Summary This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Sickle Cell Disease
  • Transfusion Dependent Alpha- or Beta- Thalassemia
  • Diamond Blackfan Anemia
  • Paroxysmal Nocturnal Hemoglobinuria
  • Glanzmann Thrombasthenia
  • Severe Congenital Neutropenia
  • Shwachman-Diamond Syndrome
  • Non-Malignant Hematologic Disorders
Intervention  ICMJE
  • Drug: Reduced Toxicity Ablative Regimen
    • Anti-thymocyte Globulin (ATG)
    • Fludarabine
    • Busulfan
    • Stem Cell Infusion Day 0
  • Drug: Reduced Intensity Preparative Regimen
    • Alemtuzumab
    • Cyclophosphamide
    • Fludarabine
    • Total Body Irradiation (TBI)
    • Stem Cell Infusion Day 0
  • Drug: Myeloablative Preparative Regimen
    • Alemtuzumab
    • Cyclophosphamide
    • Busulfan
    • Stem Cell Infusion Day 0
Study Arms  ICMJE
  • Experimental: Reduced Toxicity Ablative Regimen
    For use in patients with a matched sibling donor or unrelated UCB donor and DBA patients who are <12 years and/or have mild/moderate iron exposure.
    Intervention: Drug: Reduced Toxicity Ablative Regimen
  • Experimental: Reduced Intensity Preparative Regimen
    For use in patients with unrelated donor bone marrow and for DBA patients who are >12 years and/or have significant iron exposure.
    Intervention: Drug: Reduced Intensity Preparative Regimen
  • Experimental: Myeloablative Preparative Regimen
    For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.
    Intervention: Drug: Myeloablative Preparative Regimen
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 27, 2014)
25
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE August 2021
Estimated Primary Completion Date August 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
  • Acceptable stem cell source identified
  • Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
  • Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
  • Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
  • Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%

Exclusion Criteria:

  • active, uncontrolled infection
  • pregnant or breastfeeding
  • HIV positive
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 55 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Kim Nelson 612-273-2925 knelso62@fairview.org
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02179359
Other Study ID Numbers  ICMJE 2014OC034
MT2014-10C ( Other Identifier: Masonic Cancer Center, University of Minnesota )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Masonic Cancer Center, University of Minnesota
Study Sponsor  ICMJE Masonic Cancer Center, University of Minnesota
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Angela R Smith, MD Masonic Cancer Center, University of Minnesota
PRS Account Masonic Cancer Center, University of Minnesota
Verification Date September 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP