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Johns Hopkins All Children's Hospital

Cancer and Blood Disorders Institute

Active Clinical Trials and Research Studies

The following are the active clinical trials currently available or anticipated in the Center for Cell and Gene Therapy for Non-malignant Conditions. More information is available at ClinicalTrials.gov.

  • TCRαβ T-cell and CD19 B-cell Depleted Haploidentical Hematopoietic Cell Transplantation in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children (HAP-PID).
  • TCRαβ T-cell and CD19 B-cell Depleted Haploidentical Hematopoietic Cell Transplantation in the Treatment of Non-Malignant Hematological Disorders in Children (HAP-HEM).
  • Cell Processing for Hematopoietic Stem Cell Infusion Using the Miltenyi CliniMACS CD34 System.
  • PBMTC 01-0923: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regimen.
  • EDIT-SCD-301-001: A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects with Severe Sickle Cell Disease.
  • LCH-IV: International Collaborative Treatment Protocol for Children and Adolescents with Langerhans Cell Histiocytosis.
  • PIDTC 6901: A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with Severe Combined Immune Deficiency Disorders
  • PIDTC 6902: A Retrospective and Cross-Sectional Analysis of Patients Treated for Severe Combined Immune Deficiency (1968-2010)
  • PIDTC 6903: Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
  • PIDTC 6904: Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990
  • CSIDE study: A phase II randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCRαβ+/CD19+ depleted transplantation.
  • PIDTC 6906: Primary Immune Regulatory Disorders (PIRD): Longitudinal Study of Clinical Presentation, Treatment and Outcomes.
  • PIDTC 6907: Severe Combined Immune Deficiency: Prospective and Longitudinal Study of Genotypes, Management and Outcomes
  • PIDTC 6908: Analysis of Autoinflammation in Chronic Granulomatous Disease Patients Undergoing Hematopoietic Cell Transplantation or Gene Therapy

For more information about participating in a clinical study, click here.