Try the modernized beta website. Learn more about the modernization effort.
Working… Menu

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT04046224
Recruitment Status : Recruiting
First Posted : August 6, 2019
Last Update Posted : April 22, 2021
Information provided by (Responsible Party):
Sangamo Therapeutics

Brief Summary:
This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the cDNA for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single dose of ST-920 and followed for a period of 52 weeks.

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: ST-920 Phase 1 Phase 2

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 48 participants
Allocation: N/A
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease
Actual Study Start Date : July 23, 2019
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : February 2024

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Sequential dose escalation
ST-920 is administered as a single infusion
Biological: ST-920
Single dose of investigational product ST-920

Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: Up to 12 months after the ST-920 infusion ]
    Incidence of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • ≥ 18 years of age
  • Documented diagnosis of Fabry disease
  • One or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma

Exclusion Criteria:

  • Known to be unresponsive to ERT
  • Neutralizing antibodies to AAV2/6
  • Currently receiving migalastat (Galafold™)
  • eGFR ≤ 40 ml/min/1.73m2
  • New York Heart Association Class III or higher
  • Active infection with hepatitis A, B or C, HIV or TB
  • History of liver disease such as secondary steatosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, cholangitis or biliary disease within 6 months of informed consent; except for Gilbert's syndrome
  • Elevated circulating serum AFP
  • Recent or recurrent hypersensitivity response to ERT within previous 6 months
  • Current or history of systemic (IV or oral) immunomodulatory agents, or biologics or steroid use in the past 6 months (topical treatment and inhaled allowed).
  • Contraindication to use of corticosteroids
  • History of malignancy except for non-melanoma skin cancer
  • Recent history of alcohol or substance abuse
  • Participation in prior investigational interventional drug or medical device study within previous 3 months
  • Prior treatment with a gene therapy product
  • Known hypersensitivity to components of ST-920 formulation
  • Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study including but not limited to risk of COVID-19 infection

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04046224

Layout table for location contacts
Contact: Patient Advocacy 510-307-7266

Layout table for location information
United States, Georgia
Emory University School of Medicine Recruiting
Atlanta, Georgia, United States, 30322
Contact: Dawn J Laney    404-778-8518   
Principal Investigator: William Wilcox, M.D.         
United States, Iowa
University of Iowa Hospital and Clinics Recruiting
Iowa City, Iowa, United States, 52242
Contact: Lauren VanOpdorp    319-356-0334   
Principal Investigator: John Bernat, M.D., PhD         
United States, Minnesota
University of Minnesota Medical Center Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Brenda Diethelm-Okita    612-625-1594   
Principal Investigator: Chester B Whitley, M.D., PhD         
United States, New York
NYU Langone Health Neurogenetics Active, not recruiting
New York, New York, United States, 10017
Mt. Sinai School of Medicine Recruiting
New York, New York, United States, 10029
Contact: Karli Hedstrom    212-659-1450 ext 81450   
Principal Investigator: Jaya Ganesh, M.D.         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Laurie Bailey    513-636-4507   
Principal Investigator: Robert Hopkin, M.D.         
United States, Virginia
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) Recruiting
Fairfax, Virginia, United States, 22030
Contact: Jacqueline Fikry    571-308-1900 ext 7   
Principal Investigator: Ozlem Goker-Alpan, M.D.         
United Kingdom
Addenbrooke's Hospital Recruiting
Cambridge, United Kingdom, CB2 0QQ
Contact: Lisa Morris-Bacon    +44 (0) 1223 274634   
Principal Investigator: Patrick Deegan, M.D.         
Royal Free Hospital Recruiting
London, United Kingdom
Contact: Masoud Kazemi    02077940500 ext 22488   
Principal Investigator: Derralynn Hughes, M.D.         
Sponsors and Collaborators
Sangamo Therapeutics
Layout table for investigator information
Study Director: Medical Monitor Sangamo Therapeutics, Inc.
Layout table for additonal information
Responsible Party: Sangamo Therapeutics Identifier: NCT04046224    
Other Study ID Numbers: ST-920-201
First Posted: August 6, 2019    Key Record Dates
Last Update Posted: April 22, 2021
Last Verified: April 2021

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sangamo Therapeutics:
Lysosomal Storage Disease
Gene Therapy
Additional relevant MeSH terms:
Layout table for MeSH terms
Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders