Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
Stopped: Study no longer needed
United States5 participantsStarted 2018-07-02
Plain-language 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.
Who can participate
Age range0 Years – 25 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 …
What they're measuring
1
Neutrophil Recovery
Timeframe: Day 42
Trial details
NCT IDNCT03367546
SponsorMasonic Cancer Center, University of Minnesota