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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04046224




Registration number
NCT04046224
Ethics application status
Date submitted
1/08/2019
Date registered
6/08/2019
Date last updated
9/05/2024

Titles & IDs
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)
Secondary ID [1] 0 0
ST-920-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - ST-920

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

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


Treatment: Other: ST-920
Single dose of investigational product ST-920
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events (TEAEs)
Timepoint [1] 0 0
Up to 12 months after the ST-920 infusion

Eligibility
Key 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

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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/07/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2025
Actual
Sample size
Target
34
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Iowa
Country [6] 0 0
United States of America
State/province [6] 0 0
Minnesota
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Virginia
Country [10] 0 0
Canada
State/province [10] 0 0
Alberta
Country [11] 0 0
Germany
State/province [11] 0 0
Hamburg
Country [12] 0 0
Germany
State/province [12] 0 0
Würzburg
Country [13] 0 0
Italy
State/province [13] 0 0
Tuscany
Country [14] 0 0
Taiwan
State/province [14] 0 0
Taipei
Country [15] 0 0
United Kingdom
State/province [15] 0 0
Birmingham
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Cambridge
Country [17] 0 0
United Kingdom
State/province [17] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Sangamo Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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 a-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of a-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.
Trial website
https://clinicaltrials.gov/study/NCT04046224
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor
Address 0 0
Sangamo Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04046224