Alpha/Beta T-cell Depleted Blood-forming Stem Cell Transplant From Related or Unrelated Donors for Blood Diseases in Children and Young Adults

Launched by UNIVERSITY OF WISCONSIN, MADISON · Mar 16, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a new approach to treating certain blood diseases in children and young adults using a special type of stem cell transplant. The goal is to see if it's safe and effective to use stem cells from closely matched donors while reducing the risk of complications that can occur after the transplant. The unique technique being tested aims to keep beneficial immune cells while removing those that could cause problems. Researchers hope this method will lead to better recovery and fewer infections after the transplant.

To be eligible for this trial, participants need to be younger than 40 and have specific blood disorders like sickle cell disease or certain inherited bone marrow failures. They must not have a matched sibling donor or a suitable unrelated donor available. Patients will receive a special treatment before the transplant and will be closely monitored for their recovery and any side effects. If you or someone you know is interested and meets the criteria, this could be an important opportunity to help improve treatments for blood diseases.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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 Criteria:
• • 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).