Sickle Cell Disease Clinical Trial
Official title:
Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant Following Reducing Intensity Conditioning for Patients With Selected High Risk Non-Malignant Disease
Verified date | February 2024 |
Source | Masonic Cancer Center, University of Minnesota |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Terminated |
Enrollment | 5 |
Est. completion date | December 19, 2022 |
Est. primary completion date | December 19, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 0 Years to 25 Years |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Masonic Caner Center at University of Minnesota | Minneapolis | Minnesota |
Lead Sponsor | Collaborator |
---|---|
Masonic Cancer Center, University of Minnesota |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Neutrophil Recovery | Incidence of neutrophil recovery by day +42 | Day 42 | |
Secondary | Overall Survival (OS) | Incidence of overall survival at 1 year | 1 year | |
Secondary | Primary Graft Failure (neutropenic and non-neutropenic) | Incidence of primary graft failure (neutropenic and non-neutropenic) by day +42 | Day 42 |
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