- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02179359
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
February 5, 2024 updated by: Masonic Cancer Center, University of Minnesota
MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
Study Overview
Status
Recruiting
Conditions
Study Type
Interventional
Enrollment (Estimated)
25
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Lisa Burke
- Phone Number: 612-273-8482
- Email: lburke3@Fairview.org
Study Locations
-
-
Minnesota
-
Minneapolis, Minnesota, United States, 55455
- Recruiting
- University of Minnesota Medical Center, Fairview
-
Contact:
- Lisa Burke
- Phone Number: 612-273-8482
- Email: lburke3@Fairview.org
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
No older than 53 years (Child, Adult)
Accepts Healthy Volunteers
No
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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.
|
|
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.
|
|
Experimental: Myeloablative Preparative Regimen
For use in patients with a matched sibling donor, unrelated umbilical cord blood and in those with severe thalassemia.
|
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
incidence of graft failure
Time Frame: 42 days
|
42 days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
overall survival
Time Frame: 6 months, 1 and 2 years
|
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)
|
6 months, 1 and 2 years
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Principal Investigator: Ashish Gupta, MBBS, MPH, Masonic Cancer Center, University of Minnesota
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
September 2, 2014
Primary Completion (Estimated)
January 1, 2025
Study Completion (Estimated)
August 1, 2025
Study Registration Dates
First Submitted
June 27, 2014
First Submitted That Met QC Criteria
June 27, 2014
First Posted (Estimated)
July 1, 2014
Study Record Updates
Last Update Posted (Estimated)
February 6, 2024
Last Update Submitted That Met QC Criteria
February 5, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Digestive System Diseases
- Metabolic Diseases
- Urologic Diseases
- Urological Manifestations
- Bone Marrow Diseases
- Blood Coagulation Disorders, Inherited
- Hemorrhagic Disorders
- Genetic Diseases, Inborn
- Urination Disorders
- Anemia
- Blood Coagulation Disorders
- Lipid Metabolism Disorders
- Blood Platelet Disorders
- Agranulocytosis
- Leukopenia
- Leukocyte Disorders
- Pancreatic Diseases
- Proteinuria
- Anemia, Hemolytic, Congenital
- Anemia, Hemolytic
- Anemia, Hypoplastic, Congenital
- Anemia, Aplastic
- Congenital Bone Marrow Failure Syndromes
- Bone Marrow Failure Disorders
- Lipid Metabolism, Inborn Errors
- Myelodysplastic Syndromes
- Red-Cell Aplasia, Pure
- Lipomatosis
- Exocrine Pancreatic Insufficiency
- Female Urogenital Diseases
- Female Urogenital Diseases and Pregnancy Complications
- Urogenital Diseases
- Male Urogenital Diseases
- Neutropenia
- Hematologic Diseases
- Anemia, Sickle Cell
- Thalassemia
- beta-Thalassemia
- Hemoglobinopathies
- Hemoglobinuria
- Hemoglobinuria, Paroxysmal
- Anemia, Diamond-Blackfan
- Shwachman-Diamond Syndrome
- Thrombasthenia
Other Study ID Numbers
- 2014OC034
- MT2014-10C (Other Identifier: Masonic Cancer Center, University of Minnesota)
Drug and device information, study documents
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
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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-
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HemaQuest Pharmaceuticals Inc.TerminatedSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SUnited States, Lebanon, Egypt, Canada, Jamaica
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HemaQuest Pharmaceuticals Inc.CompletedSickle Cell Disease | Sickle Cell Anemia | Sickle Cell Disorders | Hemoglobin S Disease | Sickling Disorder Due to Hemoglobin SUnited States, Lebanon, Canada, Egypt, Jamaica
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Novartis PharmaceuticalsCompletedKidney TransplantationBelgium, Taiwan, Italy, Germany, Spain, Switzerland, Australia, Singapore, Norway, Netherlands, Colombia, Argentina, Brazil, Austria, Slovakia, France, Czech Republic
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Randy WindreichCompletedAcute Myeloid Leukemia (AML) | Myelodysplastic Syndrome (MDS) | Hematopoietic Stem Cell Transplant (HSCT)United States
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St. Jude Children's Research HospitalCompletedAcute Myeloid Leukemia | Leukemia | Hodgkin's Lymphoma | Juvenile Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Non-Hodgkin Lymphoma | Chronic Myeloid Leukemia | Myelodysplastic Syndrome | Paroxysmal Nocturnal HemoglobinuriaUnited States
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St. Jude Children's Research HospitalCompletedLymphoma, Non-Hodgkin | Hodgkin Disease | Chronic Myeloid Leukemia | Acute Myeloid Leukemia (AML) | Acute Lymphoblastic Leukemia (ALL) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Myelodysplastic Syndrome (MDS) | Secondary AML | Juvenile Myelomonocytic Leukemia (JMML) | Secondary MDSUnited States
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-
Xiayun He, MDCompleted