Clinical Trials

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Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 1
  • Intervention Type: Biological
  • Participants: 8
  • Start Date: August 2021
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
Clinical Trial
  • Status: Recruiting
  • Intervention Type: Drug
  • Participants: 50
  • Start Date: August 20, 2020
A Prospective Outcomes Study of Pediatric and Adult Patients With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation With a Reduced-Intensity Conditioning Regimen (PRO-RIC)
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Clinical Trial
  • Status: Recruiting
  • Intervention Type: Biological
  • Participants: 20
  • Start Date: June 25, 2019
Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA-SCID Patients Requiring Enzyme Replacement Therapy
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1
  • Intervention Type: Drug
  • Participants: 5
  • Start Date: December 21, 2018
Phase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan
Clinical Trial
  • Status: Recruiting
  • Phase: N/A
  • Intervention Type: Genetic
  • Participants: 10
  • Start Date: October 30, 2018
Gene Transfer for Adenosine Deaminase-severe Combined Immunodeficiency (ADA-SCID) Using an Improved Self-inactivating Lentiviral Vector (TYF-ADA)
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Biological
  • Participants: 10
  • Start Date: January 19, 2018
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Drug, Genetic
  • Participants: 10
  • Start Date: January 3, 2018
Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1α Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1
  • Intervention Type: Biological
  • Participants: 42
  • Start Date: March 20, 2017
A Phase I Study To Evaluate The Safety And Tolerability of JSP191 For Hematopoietic Cell Transplantation Conditioning To Achieve Engraftment And Immune Reconstitution In Subjects With SCID
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Device, Genetic, Drug
  • Participants: 28
  • Start Date: August 17, 2016
A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 100
  • Start Date: February 4, 2014
A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults ≤55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 2
  • Intervention Type: Drug
  • Participants: 20
  • Start Date: March 2013
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime
Clinical Trial
  • Status: Recruiting
  • Phase: N/A
  • Intervention Type: Drug, Biological
  • Participants: 30
  • Start Date: September 4, 2012
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
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