Brief Title
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
Official Title
Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant Following Reducing Intensity Conditioning for Patients With Selected High Risk Non-Malignant Disease
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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Neutrophil Recovery
Secondary Outcome
Overall Survival (OS)
Condition
Sickle Cell Disease
Intervention
Blood and Marrow Transplant
Study Arms / Comparison Groups
rATG, FLU/CY/TBI, & Thiotepa
Description: Anti-Thymocyte Globulin - Rabbit (rATG), Fludarabine (Fludara), Cyclophosphamide (Cytoxan, Neosar), Total Body Irradiation (TBI), & Thiotepa
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Procedure
Estimated Enrollment
20
Start Date
July 2, 2018
Completion Date
November 2025
Primary Completion Date
November 2025
Eligibility Criteria
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
Gender
All
Ages
0 Years - 25 Years
Accepts Healthy Volunteers
No
Contacts
Christen L Ebens, MD, MPH, 612-273-8482, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03367546
Organization ID
2017LS101
Secondary IDs
MT2017-30
Responsible Party
Sponsor
Study Sponsor
Masonic Cancer Center, University of Minnesota
Study Sponsor
Christen L Ebens, MD, MPH, Principal Investigator, University of Minnesota - Pediatrics Blood and Marrow Transplant
Verification Date
May 2022