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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 December 2023
• Main ID: NCT04046224
• Date of registration: 01/08/2019
• Prospective Registration: No
• Primary sponsor: Sangamo Therapeutics
• Public title: Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
• Scientific 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 (STAAR)
• Date of first enrolment: July 23, 2019
• Target sample size: 34
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04046224
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Australia, Canada, Germany, Italy, Taiwan, United Kingdom, United States

Contacts

• Name: Medical Monitor
• Affiliation: Sangamo Therapeutics, Inc.

Key inclusion & exclusion criteria

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

- Subject must be fully vaccinated (as per the Centers for Disease Control and
Prevention (CDC) definition in the US and as per local guidelines in other countries)
for COVID-19 at least one month prior to dosing

Additional Inclusion Criteria:

Renal Cohort:

- Screening eGFR value between 40-90 mL/min/1.73 m²

- Linear negative eGFR slope (estimated from at least 3 serum creatinine values within
18 months, including the value obtained during screening visit) of = 2
mL/min/1.73m²/year

Cardiac Cohort:

• Left ventricular hypertrophy (LVH) in 2D echocardiography or CMR defined as an end
diastolic septum and posterior wall thickness =12 mm with no other explanation for LVH, OR
presentation with cardiac changes indicative of disease progression such as decreased
global longitudinal strain on 2D strain echocardiography or low native T1 mapping on CMR

Exclusion Criteria:

- Neutralizing antibodies to AAV6

- 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 clinically significant steatosis, fibrosis,
non-alcoholic steatohepatitis (NASH) and cirrhosis, 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 within 6 months prior to
consent

- Current or history of systemic (IV or oral) immunomodulatory agents, or biologics or
steroid use in the past 6 months prior to consent (topical treatment and inhaled
allowed).

- Contraindication to use of corticosteroids

- History of malignancy except for non-melanoma skin cancer and localized prostate
cancer treated with curative intent

- Recent history of alcohol or substance abuse

- Participation in investigational interventional drug or medical device study
throughout the duration of this study and within previous 3 months prior to consent

- 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

Additional exclusion criteria for:

Renal cohort:

- History of renal dialysis or transplantation

- History of acute kidney insufficiency in the 6 months prior to screening

- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB)
therapy initiated within 4 weeks prior to screening or changed ACE inhibitor or ARB
dose in the 4 weeks prior to screening

- Urine protein to creatinine ratio (UPCR) > 0.5 g/g who are not being treated with an
ACE inhibitor or ARB

Cardiac cohort:

- Significant cardiac fibrosis defined by late gadolinium enhancement on CMR

- Any contraindications to CMR as per local hospital/institution guidelines

- Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB)
therapy initiated within 4 weeks prior to screening or changed ACE inhibitor or ARB
dose in the 4 weeks prior to screening

- NYHA Class IV



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Biological: ST-920

Primary Outcome(s)

Incidence of treatment-emergent adverse events (TEAEs) [Time Frame: Up to 12 months after the ST-920 infusion]

Secondary ID(s)

ST-920-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors