Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant Following Reducing Intensity Conditioning for Patients With Selected High Risk Non-Malignant Disease

About the study

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 point of contact

Lisa Burke, RN


1 United States site


0 Years - 25 Years


Phase 2

Study type






participation requirements

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


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%.

participation restrictions

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


  • Minneapolis, Minnesota, United States, Masonic Caner Center at University of Minnesota, 55455 [Recruiting]
Last updated 2022-05-23