AmiJayantShahMD

Clinical Trials

• Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for the Human ADA Gene Start of enrollment: 2013 Aug 02

  Completed

  Phase 1, Phase 2

• Gene Therapy for Pyruvate Kinase Deficiency (PKD) Start of enrollment: 2020 Jul 06

  Active, not recruiting

  Phase 1

• Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease Start of enrollment: 2022 Aug 02

  Recruiting

  Phase 3

• A Long-term Follow-up Study in Participants Who Received CTX001 Start of enrollment: 2021 Jan 20

  Enrolling by invitation

  Phase 3

• Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product Start of enrollment: 2016 Jan 22

  Active, not recruiting

• A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD) Start of enrollment: 2019 Jan 24

  Completed

  Phase 3

• Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990 Start of enrollment: 2014 Feb 02

  Completed

• Patients Treated for SCID (1968-Present) Start of enrollment: 2011 May 15

  Enrolling by invitation

• Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995 Start of enrollment: 2014 Jun 01

  Enrolling by invitation

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