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
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Condition or disease | Intervention/treatment | Phase |
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Sickle Cell Disease Thalassemia High Risk Hematologic Disorders Cerebral Adrenoleukodystrophy Inherited Metabolic Disorders | Procedure: Blood and Marrow Transplant | Phase 2 |
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 |
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Experimental: rATG, FLU/CY/TBI, & Thiotepa
Anti-Thymocyte Globulin - Rabbit (rATG), Fludarabine (Fludara), Cyclophosphamide (Cytoxan, Neosar), Total Body Irradiation (TBI), & Thiotepa
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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) |
- Neutrophil Recovery [ Time Frame: Day 42 ]Incidence of neutrophil recovery by day +42
- Overall Survival (OS) [ Time Frame: 1 year ]Incidence of overall survival at 1 year
- Primary Graft Failure (neutropenic and non-neutropenic) [ Time Frame: Day 42 ]Incidence of primary graft failure (neutropenic and non-neutropenic) by day +42

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

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
Contact: Lisa Burke, RN | 612-273-8482 | lburke3@Fairview.org |
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 |
Principal Investigator: | Christen L Ebens, MD, MPH | University of Minnesota - Pediatrics Blood and Marrow Transplant |
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 |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
SCD cALD |
Adrenoleukodystrophy Anemia, Sickle Cell Thalassemia Hematologic Diseases Metabolic Diseases Disease Pathologic Processes Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hemoglobinopathies 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 Leukoencephalopathies 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 |