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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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. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04046224 |
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Recruitment Status :
Active, not recruiting
First Posted : August 6, 2019
Last Update Posted : December 7, 2023
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Sponsor:
Sangamo Therapeutics
Information provided by (Responsible Party):
Sangamo Therapeutics
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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 Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 34 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 : | April 2025 |
| Estimated Study Completion Date : | September 2025 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Fabry disease
MedlinePlus related topics:
Genes and Gene Therapy
| Arm | Intervention/treatment |
|---|---|
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Experimental: Sequential dose escalation
ST-920 is administered as a single infusion:
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Biological: ST-920
Single dose of investigational product ST-920 |
Experimental: Expansion Cohorts
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Biological: ST-920
Single dose of investigational product ST-920 |
Primary Outcome Measures :
- 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)
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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
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
Locations
Show 21 study locations
Sponsors and Collaborators
Sangamo Therapeutics
Investigators
| Study Director: | Medical Monitor | Sangamo Therapeutics, Inc. |
| 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: | December 7, 2023 |
| Last Verified: | December 2023 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Sangamo Therapeutics:
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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 |