Center for Cell and Gene Therapy for Non-Malignant Conditions

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.