RECRUITING

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children

Talk to us

Conditions

Primary Immune Deficiency DisordersMetabolic Disease

Quick Navigation

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 Primary Immunodeficiency and Inherited Metabolic 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

NCT04414046

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:Not specified to 21 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Patient with any form of primary immune deficiency/dysregulatory disorders characterized by aberrant immune function, abnormal hematopoiesis, systemic or organ specific autoimmunity and/or non-malignant lymphoproliferation. This includes, but not limited to:
  2. 2. Histiocytic disorders including hemophagocytic lymphohistiocytosis (familial HLH (types 1-5), secondary HLH (refractory to therapy or with recurrent episodes of hyper inflammation) and multisystem refractory Langerhans cell histiocytosis.
  3. 3. Metabolic disorders that could improve or stabilize after stem cell transplantation such as mucopolysaccharidoses, neurodegenerative disorders, osteopetrosis, etc.
  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.

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

  • Primary Immune Deficiency Disorders
  • Metabolic Disease