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Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

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ClinicalTrials.gov Identifier: NCT03367546
Recruitment Status : Recruiting
First Posted : December 8, 2017
Last Update Posted : May 25, 2022
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Brief Summary:
This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Thalassemia High Risk Hematologic Disorders Cerebral Adrenoleukodystrophy Inherited Metabolic Disorders Procedure: Blood and Marrow Transplant Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant Following Reducing Intensity Conditioning for Patients With Selected High Risk Non-Malignant Disease
Actual Study Start Date : July 2, 2018
Estimated Primary Completion Date : November 2025
Estimated Study Completion Date : November 2025

Arm Intervention/treatment
Experimental: rATG, FLU/CY/TBI, & Thiotepa
Anti-Thymocyte Globulin - Rabbit (rATG), Fludarabine (Fludara), Cyclophosphamide (Cytoxan, Neosar), Total Body Irradiation (TBI), & Thiotepa
Procedure: Blood and Marrow Transplant
Reduced intensity conditioning (RIC) with rabbit ATG, fludarabine, cyclophosphamide, thiotepa and low dose (2 Gy) total body irradiation followed by T-cell replete, unmanipulated, haploidentical related donor stem cell transplant (HaploHCT) and post-transplant cyclophosphamide (PTCy)

Primary Outcome Measures :
  1. Neutrophil Recovery [ Time Frame: Day 42 ]
    Incidence of neutrophil recovery by day +42

Secondary Outcome Measures :
  1. Overall Survival (OS) [ Time Frame: 1 year ]
    Incidence of overall survival at 1 year

  2. Primary Graft Failure (neutropenic and non-neutropenic) [ Time Frame: Day 42 ]
    Incidence of primary graft failure (neutropenic and non-neutropenic) by day +42

Information from the National Library of Medicine

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Ages Eligible for Study:   0 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Sickle Cell Disease (SCD)

    * If diagnosis of SCD must meet one or more of the following disease characteristics:

    • Stroke, CNS hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral MRI or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
    • Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
    • Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
    • Impaired neuropsychological function and abnormal cerebral MRI scan
    • Stage I or II sickle lung disease,
    • Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate [GFR] 30-50% of the predicted normal value)
    • Bilateral proliferative retinopathy and major visual impairment in at least one eye
    • Osteonecrosis of multiple joints with documented destructive changes
    • Requirement for chronic transfusions
    • RBC alloimmunization
  • Transfusion Dependent Alpha- or Beta-Thalassemia
  • Other Non-Malignant Hematologic Disorders:

Transfusion dependent or involve other potential life-threatening cytopenias, including but not limited to Paroxysmal Nocturnal Hemoglobinuria, Glanzmann's Thrombasthenia, Severe Congenital Neutropenia and Shwachman-Diamond Syndrome

  • cALD

    • Diagnosis of ALD by abnormal plasma very long chain fatty acid (VLCFA) profile or ABCD1 gene mutation
    • Cerebral disease on MRI
    • Absence of a Major Functional Disability (cortical blindness, loss of communication, wheelchair dependence) on the ALD Neurologic Function Scale
  • Other inherited metabolic disorders:

Any other inherited metabolic disorder for which alloHCT is indicated and for whom, in the opinion of the treating physician, the patient's best treatment option is with a haploidentical donor following non-myeloablatve conditioning.

  • Age, Performance Status, Consent

    • Age: 0-55 years
    • Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
    • Consent: voluntary written consent (adult or parental/guardian)
  • Adequate Organ Function

    • Renal: Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
    • Hepatic: Bilirubin and ALT <3 times the upper limit of institutional normal
    • Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%.

Exclusion Criteria:

  • Availability of a suitable HLA-matched related donor
  • Uncontrolled infection
  • Pregnant or breastfeeding
  • HIV positive

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): NCT03367546

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Contact: Lisa Burke, RN 612-273-8482 lburke3@Fairview.org

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United States, Minnesota
Masonic Caner Center at University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Lisa Burke    612-273-8482    lburke3@Fairview.org   
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
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Principal Investigator: Christen L Ebens, MD, MPH University of Minnesota - Pediatrics Blood and Marrow Transplant
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Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT03367546    
Other Study ID Numbers: 2017LS101
MT2017-30 ( Other Identifier: University of Minnesota Masonic Cancer Center )
First Posted: December 8, 2017    Key Record Dates
Last Update Posted: May 25, 2022
Last Verified: May 2022

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Masonic Cancer Center, University of Minnesota:
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Hematologic Diseases
Metabolic Diseases
Pathologic Processes
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Genetic Diseases, Inborn
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hereditary Central Nervous System Demyelinating Diseases
Demyelinating Diseases
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Metabolism, Inborn Errors
Peroxisomal Disorders
Adrenal Insufficiency
Adrenal Gland Diseases