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

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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


Primary Outcome

Neutrophil Recovery

Secondary Outcome

 Overall Survival (OS)


Sickle Cell Disease


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


* 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


Estimated Enrollment


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

               -  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

               -  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




0 Years - 25 Years

Accepts Healthy Volunteers



Christen L Ebens, MD, MPH, 612-273-8482, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations



Organization ID


Secondary IDs


Responsible Party


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