Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Stopped early · Phase 1/Phase 2

Conditions studied: Fabry Disease

In brief

This was a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 15 male subjects, who were 16 years of age or older and postpubertal with a confirmed diagnosis of classic Fabry disease based on deficient alpha galactosidase A (AGA) enzyme activity who were considered treatment naïve, i.e., had not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of Screening.

Key facts

Study ID

NCT03454893

Run by

AVROBIO

People needed

15

Starts

2018-02-21

Expected to finish

2022-03-14

Last updated by the study team

2024-01-05

Who can join

Age: 16 and older, up to 50. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Subject was male, 16 years of age or older (18 years of age or older in the US), and post pubertal,(minimum age by region)
• Subject had a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity (defined as < 1% of normal).

You may not qualify if…

• Subject had a galactosidase alpha (GLA) gene mutation associated with late-onset cardiac variant Fabry disease.
• Subject had previously received ERT and/or chaperone therapy within 3 years for treatment of Fabry disease.
• Subject had tested positive for anti-AGA antibodies at the time of screening.
• Subject had eGFR < 60 mL/min/1.73 m² (ie, chronic kidney disease [CKD] stage ≥ 3) at Screening.
• Subject had a prior history of myocardial infarction (MI).
• Subject had a history of coronary artery disease (CAD) with angina requiring percutaneous transluminal coronary angioplasty (with or without stent placement) and/or coronary artery bypass graft (CABG).
• Subject had a history of moderate to severe valvular heart disease requiring valve replacement.
• Subject had a history of heart failure, moderate to severe diastolic dysfunction, and/or left ventricular ejection fraction (LVEF) ≤ 45% on echocardiogram (ECHO) performed at rest at Screening.
• Subject had a history of clinically significant cardiac arrhythmia (eg, heart block [second or third degree], atrial fibrillation requiring therapy, ventricular fibrillation, ventricular tachycardia, supraventricular tachycardia, or cardiac arrest).
• Note [history of intermittent atrial fibrillation not requiring treatment was allowed].
• Subject had a prior history of stroke and/or transient ischemic attack (TIA).
• Subject had aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≥ 3 times the upper limit of normal (ULN) at Screening.
• Subject had a prior history of (or current) malignancy; the one exception is a prior history of resected basal cell carcinoma.
• Subject had previously received treatment with AVR-RD-01 or any other gene therapy.
• Other inclusion/exclusion criteria apply.

Where it is running

• Hackensack University Medical Center — Hackensack, New Jersey, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• Royal Melbourne Hospital — Melbourne, Parkville VIC, Australia
• Royal Perth Hospital — Perth, Australia
• Hospital de Clinicas de Porto Alegre — Porto Alegre, Rio Grande do Sul, Brazil

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.