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

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ClinicalTrials.gov Identifier: NCT04046224
Recruitment Status : Recruiting
First Posted : August 6, 2019
Last Update Posted : July 13, 2023
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 : 48 participants
Allocation: Non-Randomized
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 (STAAR)
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


Arms and Interventions
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Arm Intervention/treatment
Experimental: Sequential dose escalation

ST-920 is administered as a single infusion:

  1. Cohort 1: 0.5e13 vg/kg
  2. Cohort 2: 1.0e13 vg/kg
  3. Cohort 3: 3.0e13 vg/kg
  4. Cohort 4: 5.0e13 vg/kg
 Biological: ST-920
Single dose of investigational product ST-920
Experimental: Expansion Cohorts
  1. Anti Alpha-Gal A Antibody Positive Cohort
  2. Anti Alpha-Gal A Antibody Negative Cohort
  3. Female Cohort
  4. Renal Cohort
  5. Cardiac Cohort
 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:   All
Accepts Healthy Volunteers:   No
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
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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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: July 13, 2023
Last Verified: July 2023

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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
Sphingolipidoses
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
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders