belcesiran for alpha 1-Antitrypsin Deficiency

Phase-Based Progress Estimates
alpha 1-Antitrypsin Deficiency
belcesiran - Drug
All Sexes
What conditions do you have?

Study Summary

This trial will test the safety and effectiveness of a new drug, belcesiran, in adults with a specific liver disease. The trial will have two groups of patients, who will receive different doses of the drug or a placebo. The trial will last 24 or 48 weeks, depending on the group, and patients will have a liver biopsy at the end to check for improvement.

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for alpha 1-Antitrypsin Deficiency

Study Objectives

12 Primary · 4 Secondary · Reporting Duration: Cohort 1 and Cohort 2: from baseline to Day 1, Day 2, Day 3, Day 15, Day 29, Day 30, Day 57, Day 85, Day 113, Day 141, Day 169]

Day 169
Hour 24
Plasma concentration of Belcesiran on Day 1
Plasma concentration of Belcesiran on Day 29
Day 169
Predose plasma concentration of Belcesiran
Day 169
Changes from baseline in pulmonary function tests
Day 337
Change from baseline in coagulation parameters
Change from baseline in hematologic parameters
Changes from baseline (defined as predose Day 1) in 12-lead electrocardiograms (ECG)
Changes from baseline (defined as predose Day 1) in vital sign measurements
Changes from baseline in Serum A1AT protein concentrations
Day 337
Week 48
The incidence of adverse events (AE), serious adverse events (SAE), and AE leading to study drug discontinuation.
The incidence of clinically significant physical examination (PE) findings.
Day 3379
Changes from baseline (defined as predose Day 29) in vital sign measurements

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Other trials for alpha 1-Antitrypsin Deficiency

Trial Design

2 Treatment Groups

Experimental: belcesiran
1 of 2
1 of 2
Experimental Treatment
Non-Treatment Group

54 Total Participants · 2 Treatment Groups

Primary Treatment: belcesiran · Has Placebo Group · Phase 2

Experimental: belcesiran
Experimental Group · 1 Intervention: belcesiran · Intervention Types: Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Other

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: cohort 1 and cohort 2: from baseline to day 1, day 2, day 3, day 15, day 29, day 30, day 57, day 85, day 113, day 141, day 169]

Who is running the clinical trial?

Dicerna Pharmaceuticals, Inc.Lead Sponsor
14 Previous Clinical Trials
486 Total Patients Enrolled
Hardean Achneck, MDStudy DirectorDicerna Pharmaceuticals
2 Previous Clinical Trials
30 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 6 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 4th, 2021

Last Reviewed: October 18th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

Who else is applying?

What state do they live in?
How old are they?
18 - 65100.0%
What site did they apply to?
Centre Hospitalier de l'Universite de Montreal (CHUM)100.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%