MT2017-30 :Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant following Reducing Intensity Conditioning for Patients with Selected High Risk Non-Malignant Disease

I'm interested

up to 25 Years old
This study is NOT accepting healthy volunteers
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

Procedure: Blood and Marrow Transplant

Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders

SCD, cALD, Clinics and Surgery Center (CSC)

Christen Ebens -
Christen Ebens
Phase 2
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