Status:
TERMINATED
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Sickle Cell Disease
Thalassemia
Eligibility:
All Genders
Up to 25 years
Phase:
PHASE2
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...
Eligibility Criteria
Inclusion
- 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
- Availability of a suitable HLA-matched related donor
- Uncontrolled infection
- Pregnant or breastfeeding
- HIV positive
Key Trial Info
Start Date :
July 2 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 19 2022
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT03367546
Start Date
July 2 2018
End Date
December 19 2022
Last Update
July 12 2024
Active Locations (1)
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1
Masonic Caner Center at University of Minnesota
Minneapolis, Minnesota, United States, 55455