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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 19, 2022
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

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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.

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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

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Contact: Patient Advocacy 510-307-7266

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United States, California
University of California, Irvine Recruiting
Irvine, California, United States, 92697
Contact: Diego Canchola    714-456-8123   
Principal Investigator: Madeleine Pahl, M.D.         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32611
Contact: Samantha Norman, MPH, BS    325-273-8218   
Principal Investigator: Coy Heldermon, M.D., PhD         
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: Teresa Kopel    319-467-8147   
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 Withdrawn
New York, New York, United States, 10017
Mt. Sinai School of Medicine Recruiting
New York, New York, United States, 10029
Contact: Alvin McDonald    212-659-1658   
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, Pennsylvania
University of Pittsburgh Medical Center Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Lee Williams    412-692-8412   
Contact: Nadene Henderson    412-692-3475   
Principal Investigator: Damara Ortiz, M.D.         
United States, Virginia
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) Recruiting
Fairfax, Virginia, United States, 22030
Contact: Lauren Noll    571-732-4655   
Principal Investigator: Ozlem Goker-Alpan, M.D.         
United Kingdom
Queen Elizabeth Hospital Recruiting
Birmingham, United Kingdom, B15 2TH
Contact: Shaun Bolton    +44 (0) 1213 716795   
Contact: Heather Small    +44 (0) 1213 716696   
Principal Investigator: Tarekegn G. Hiwot, M.D., PhD         
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    +44 (0) 2077 940500 ext 22488   
Principal Investigator: Derralynn Hughes, M.D.         
Sponsors and Collaborators
Sangamo Therapeutics
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Study Director: Medical Monitor Sangamo Therapeutics, Inc.
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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 19, 2022
Last Verified: April 2022

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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:
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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
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors