RECRUITING

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

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Conditions

Hemoglobinopathy (Disorder)Severe Aplastic AnemiaBone Marrow Failure Syndrome

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational.

Official Title

Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Non-Malignant Hematological Disorders in Children

Quick Facts

Study Start:2020-07-22
Study Completion:2025-06-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04356469

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:0 Years to 21 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Severe sickle cell disease (HbSS, HbSC, HbSB0, HbSB+, HbSD, HbSE) with at least one of the following criteria:
  2. 1. Cerebrovascular accident lasting longer than 24 hours
  3. 2. Impaired neuropsychological function with abnormal brain MRI/MRA
  4. 3. Patients with frequent (≥ 3 per year for preceding 2 years) painful vaso-occlusive episodes
  5. 4. Recurrent (≥ 3 in lifetime) acute chest syndrome events which have necessitated erythrocyte transfusion therapy
  6. 5. Any combination of ≥ 3 acute chest syndrome episodes and vaso-occlusive pain episodes yearly for 3 years and have failed treatment with hydroxyurea (HU) (at least 6 months on maximum tolerated dose) or who are intolerant to HU therapy
  7. 2. Thalassemia major with at least one of the following criteria:
  8. 1. Transfusion dependency defined as receiving 8 or more transfusions per year
  9. 2. Thalassemia diagnosis documented by clinical assessment, laboratory evidence with microcytic anemia and absence of HbA (\< 10%) on electrophoresis and or confirmation by DNA analysis of alpha and beta gene loci
  10. 3. Genotypically proven thalassemia major for children \< 2 years of age even in the absence of transfusion dependency
  11. 4. Lucarelli class 1 or 2 risk status (i.e. with only 0-2 of the following factors: hepatomegaly, portal fibrosis, or poor response to chelation therapy)
  12. 3. Bone marrow failure syndromes and autoimmune cytopenias:
  13. 1. Severe Aplastic Anemia refractory to immunosuppressive therapy
  14. 2. Diamond Blackfan Anemia refractory to conventional therapy
  15. 3. Inherited Bone Marrow Failure Syndromes such as Fanconi anemia and Shwachman-Diamond syndrome with progressive marrow failure (without cytogenetic evidence of MDS/AML)
  16. 4. Severe Congenital Neutropenia
  17. 5. Congenital Amegakaryocytic Thrombocytopenia
  18. 6. Glanzmann Thrombasthenia
  19. 7. Autoimmune Cytopenias refractory to conventional treatment (including Pure red cell aplasia, Evan's syndrome, Immune thrombocytopenia, autoimmune hemolytic anemia)
  20. 8. Other marrow failure disorders not otherwise specified
  1. 1. Participants who have an HLA-matched sibling who is able and willing to donate bone marrow. Patients with a HLA-matched unrelated donors are not excluded.
  2. 2. Pregnant or breastfeeding females.
  3. 3. Patient has HIV or uncontrolled fungal, bacterial or viral infections.
  4. 4. Patient has received prior solid organ transplant.
  5. 5. Patient has active GVHD (\> grade II) or chronic extensive GVHD due to a previous allograft at the time of inclusion.
  6. 6. For patients with hemoglobinopathy, liver biopsy is necessary if the patient has received chronic transfusions for over a year and has two ferritin levels of ≥ 1000 ng/ml. Patients with cirrhosis, extensive bridging hepatic fibrosis, or active hepatitis are excluded from enrollment.

Contacts and Locations

Study Contact

Jade Hanson, MSN
CONTACT
7277676468
jade.hanson@jhmi.edu

Principal Investigator

Deepak Chellapandian, MD
PRINCIPAL_INVESTIGATOR
Johns Hopkins All Children's Hospital

Study Locations (Sites)

Johns Hopkins All Children's Hospital
Saint Petersburg, Florida, 33701
United States

Collaborators and Investigators

Sponsor: Johns Hopkins All Children's Hospital

  • Deepak Chellapandian, MD, PRINCIPAL_INVESTIGATOR, Johns Hopkins All Children's Hospital

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2020-07-22
Study Completion Date2025-06-30

Study Record Updates

Study Start Date2020-07-22
Study Completion Date2025-06-30

Terms related to this study

Additional Relevant MeSH Terms

  • Hemoglobinopathy (Disorder)
  • Severe Aplastic Anemia
  • Bone Marrow Failure Syndrome