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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.
  • MB107-XSCID-002: A Phase 2, Single- Arm, Open- Label, Multicenter Trial to Assess the Safety and efficacy of MB-107 in Newly Diagnosed Infants with X-Linked Severe Combined Immunodeficiency (anticipated Spring 2021).
  • MB-207-003: A Phase 2, Single- Arm, Open- Label, Multicenter Trial to Assess the Safety and efficacy of MB-107 in previously Treated Infants with X-Linked Severe Combined Immunodeficiency (anticipated Spring 2021).
  • PBMTC 01-0923: A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease Using a Reduced-Intensity Preparatory Regimen.
  • LCH-IV: International Collaborative Treatment Protocol for Children and Adolescents with Langerhans Cell Histiocytosis.
  • 6901: A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with Severe Combined Immune Deficiency Disorders
  • 6902: A Retrospective and Cross-Sectional Analysis of Patients Treated for Severe Combined Immune Deficiency (1968-2010)
  • 6903: Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995
  • 6904: Analysis of Patients Treated for Wiskott-Aldrich Syndrome Since January 1, 1990
  • 6905: A phase II randomized trial of low versus moderate exposure busulfan for infants with severe combined immunodeficiency (SCID) receiving TCRαβ+/CD19+ depleted transplantation (CSIDE).

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