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

• Status: Recruiting
• Trial end date: 2021-11-01
• Target enrollment: 0
• Participant gender: Male

Summary

This is a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 8 to 12 male subjects 16 years of age or older (18 years of age or older in the US) and postpubertal with a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity who have not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of Screening.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AvroBio

Criteria

Inclusion Criteria:

1. Subject is male, 16 years of age or older (18 years of age or older in the US), and postpubertal,(minimum age by region)

2. Subject has a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity (defined as < 1% of normal).

Exclusion Criteria:

1. Subject has previously received ERT and/or chaperone therapy within 3 years for treatment of Fabry disease.

2. Subject has a GLA gene mutation associated with late-onset cardiac variant Fabry disease.

3. Subject has tested positive for anti-AGA antibodies at the time of screening.

4. Subject has eGFR < 60 mL/min/1.73 m² (ie, chronic kidney disease [CKD] stage ≥ 3) at Screening.

5. Subject has a prior history of myocardial infarction (MI).

6. Subject has 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).

7. Subject has a history of moderate to severe valvular heart disease requiring valve replacement.

8. Subject has 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.

9. Subject has a history of clinically significant cardiac arrhythmia (eg, heart block [second or third degree], atrial fibrillation requiring therapy (history of intermittent atrial fibrillation not requiring treatment is allowed), ventricular fibrillation, ventricular tachycardia, supraventricular tachycardia, or cardiac arrest).

10. Subject has a prior history of stroke and/or transient ischemic attack (TIA).

11. Subject has aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≥ 3 times the upper limit of normal (ULN) at Screening.

12. Subject has a prior history of (or current) malignancy; the one exception is a prior history of resected basal cell carcinoma.

13. Subject has previously received treatment with AVR-RD-01 or any other gene therapy.