Dose-Ranging Study of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

• ClinicalTrials.gov Identifier: NCT04046224
• Recruitment Status: Recruiting
• First Posted: August 6, 2019
• Last Update Posted: November 9, 2020
• Sponsor: Sangamo Therapeutics
• Information provided by (Responsible Party): Sangamo Therapeutics

Tracking Information

• First Submitted Date: August 1, 2019
• First Posted Date: August 6, 2019
• Last Update Posted Date: November 9, 2020
• Actual Study Start Date: July 23, 2019
• Estimated Primary Completion Date: September 28, 2021 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 2, 2019)

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)

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Dose-Ranging Study of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease
• 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
• 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.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Fabry Disease

Intervention

Biological: ST-920

Single dose of investigational product ST-920

Study Arms

Experimental: Sequential dose escalation

ST-920 is administered as a single infusion

Intervention: Biological: ST-920

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: April 2, 2020): 30
• Original Estimated Enrollment (submitted: August 2, 2019): 18
• Estimated Study Completion Date: November 28, 2021
• Estimated Primary Completion Date: September 28, 2021 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: Male
• Gender Based Eligibility: Yes

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

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

• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT04046224
• Other Study ID Numbers: ST-920-201
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Responsible Party: Sangamo Therapeutics
• Study Sponsor: Sangamo Therapeutics
• Collaborators: Not Provided

Investigators

• Study Director: Medical Monitor; Sangamo Therapeutics, Inc.

• PRS Account: Sangamo Therapeutics
• Verification Date: November 2020