Study of DCR-A1AT in Healthy Adult Volunteers

• Status: Recruiting
• Trial end date: 2022-06-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a research study to test an experimental study drug (belcesiran, also known as DCR-A1AT). This drug is being tested to see if it helps people with a rare condition known as Alpha-1 Antitrypsin Deficiency, or A1ATD. Prior to initiation of this study belcesiran had not yet been tested in humans. All study participants will be randomly assigned to either receive the study drug or a placebo. This will allow for the sponsor to compare the effects of the study drug with that of the placebo. A placebo looks like the study drug but does not contain any of the study drug.

The main purpose of the first part of the study is to evaluate the safety profile of the study drug in people who do not have A1ATD. This part of the study will also help find the dose of the study drug that has an acceptable safety profile for testing.

• Phase: Phase 1
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Dicerna Pharmaceuticals, Inc.
• Treatments: Alpha 1-Antitrypsin

Criteria

Inclusion Criteria:

• Male or Female aged 18 to 55 years, inclusive. Female participants must be either surgically sterile or postmenopausal. No women of childbearing potential are eligible for enrollment.

• Overtly Healthy, as determined by the investigator.

• Serum A1AT protein concentration >100 mg/dL

• Adequate forced expiratory volume in one second (FEV1) and adequate FEV1/forced vital capacity (FVC) ratio

• Non-smokers with a <2 pack-year history and smoking cessation for at least 6 months with a negative urinary cotinine test a screening

Exclusion Criteria:

• Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially affect participant safety

• Clinically significant abnormal laboratory tests

• Received an experimental drug within past 4 months

• Prior to use of RNAi drug or oligonucleotide-based therapy

• Known human immunodeficiency virus (HIV), hepatitis C virus (HCV), or Hepatitis B (HBV)

• Serum creatinine or estimated glomerular filtration rate (eGFR) outside normal reference ranges.