Status:
NOT_YET_RECRUITING
Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors for Blood Diseases in Children and Young Adults
Lead Sponsor:
University of Wisconsin, Madison
Conditions:
Blood Disease
Eligibility:
All Genders
3-40 years
Phase:
PHASE1
Brief Summary
This single institution, phase I clinical trial will determine the safety and feasibility of employing T-cell receptor (TCR) αβ+ and CD19+ (Cluster of Differentiation ) depleted hematopoietic stem cel...
Eligibility Criteria
Inclusion
- No Human leukocyte antigen (HLA) identical sibling available AND
- NO HLA matched unrelated donor available OR urgent need of HSCT precludes time necessary to search for suitable HLA matched unrelated donor AND
- Haploidentical donor OR closely matched unrelated donor available and willing to undergo mobilization and apheresis
- If subject has genetically confirmed inherited bone marrow failure, related donor must be evaluated for this disorder and testing must be negative.
- If subject has sickle cell disease, donor may have only sickle cell trait
- Patient must be diagnosed with one of the following diseases or disorders:
- Hemoglobinopathies
- Sickle Cell Disease for patients ≤ 21 years of age for whom hydroxyurea has been trialed for at least six months, and failed
- Thalassemia Major for patients ≤ 21 years of age
- Acquired Bone Marrow Failure Syndromes
- Paroxysmal Nocturnal Hemoglobinuria with bone marrow failure
- Myelodysplastic Syndromes (lower risk)
- Inherited Bone Marrow Failure Syndromes
- Fanconi Anemia
- Diamond Blackfan Anemia
- Dyskeratosis Congenita and related telomere disorders
- Congenital Thrombocytopenia Syndromes
- Severe Congenital Neutropenia
- Shwachman-Diamond Syndrome
- Age ≤ 40 years (except patients with hemoglobinopathies)
- Life Expectancy ≥ 3 months
- Karnofsky (patients \> 16 years)/Lansky (patients ≤ 16 years) index ≥ 60
- Organ Function Requirements
- Renal Function
- Creatinine clearance or radioisotope Glomerular Filtration Rate (GFR) greater than or equal to 60 ml/min/1.73m\^2
- Liver Function
- Total bilirubin \< 3 mg/dL
- Alanine aminotransferase (ALT)/ Serum glutamic-pyruvic transaminase; synonymous with ALT (SCPT) ≤ 3 x Upper Limit of Normal(ULN) for age
- Cardiac Function
- Ejection fraction of \> 40% by Multiple gated acquisition scan (MUGA) or echocardiogram
- Pulmonary Function
- No evidence of dyspnea at rest
- No supplemental oxygen requirement
- If measured, carbon monoxide diffusion capacity (DLCO) \> 50%
- Willing to use effective birth control method if patient is of reproductive potential
- Informed consent obtained (patient or legal representative)
Exclusion
- Pregnant
- HIV infection
- Uncontrolled, serious active infection at screening
- Significant serious intercurrent illnesses
- Enrollment in any other treatment study that would interfere with the endpoints of this study according to judgement of Principal Investigator(or PI designee).
Key Trial Info
Start Date :
January 1 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 1 2030
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT04806347
Start Date
January 1 2026
End Date
February 1 2030
Last Update
November 24 2025
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