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Dose-Ranging Study of ST-920, a rAAV2/6 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.
 
ClinicalTrials.gov Identifier: NCT04046224
Recruitment Status : Recruiting
First Posted : August 6, 2019
Last Update Posted : November 9, 2020
Sponsor:
Information provided by (Responsible Party):
Sangamo Therapeutics

Study Description
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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

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
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, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease
Actual Study Start Date : July 23, 2019
Estimated Primary Completion Date : September 28, 2021
Estimated Study Completion Date : November 28, 2021

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Arms and Interventions
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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


Outcome Measures
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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)


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male ≥ 18 years of age

  • Documented diagnosis of classical Fabry disease as defined by plasma or leukocytes α-Gal A activity and one or more characteristics of classical Fabry disease:

    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 ≤ 60 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
  • Abnormal circulating AFP
  • Recent or continued hypersensitivity response to ERT within previous 6 months
  • Current or history of systemic (IV or oral) immunomodulatory agent or steroid use in the past 6 months (topical treatment allowed).
  • Contraindication to use of corticosteroids for immunosuppression
  • History of malignancy except for non-melanoma skin cancer
  • 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
Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT04046224


Contacts
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Contact: Patient Advocacy 510-307-7266 clinicaltrials@sangamo.com

Locations
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United States, Georgia
Emory University School of Medicine Recruiting
Atlanta, Georgia, United States, 30322
Contact: Dawn J Laney    404-778-8518    dawn.laney@emory.edu   
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    lauren-vanopdorp@uiowa.edu   
Principal Investigator: John Bernat, MD, PhD         
United States, Minnesota
University of Minnesota Medical Center Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Brenda Diethelm-Okita    612-625-1594    dieth001@umn.edu   
Principal Investigator: Chester B Whitley, M.D., PhD         
United States, New York
NYU Langone Health Neurogenetics Recruiting
New York, New York, United States, 10017
Contact: Sara Rodriguez    929-455-5108    Sara.rodriguez@nyulangone.org   
Principal Investigator: Heather Lau, M.D.         
Mt. Sinai School of Medicine Recruiting
New York, New York, United States, 10029
Contact: Karli Hedstrom    212-659-1450 ext 81450    karli.hedstrom@mssm.edu   
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    laurie.bailey@cchmc.org   
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    jfikry@ldrtc.org   
Principal Investigator: Ozlem Goker-Alpan, M.D.         
Sponsors and Collaborators
Sangamo Therapeutics
Investigators
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Study Director: Medical Monitor Sangamo Therapeutics, Inc.
More Information
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Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT04046224    
Other Study ID Numbers: ST-920-201
First Posted: August 6, 2019    Key Record Dates
Last Update Posted: November 9, 2020
Last Verified: November 2020

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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:
Sangamo
Rare
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
Sphingolipidoses
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors