TerminatedPhase 2

A Study of Belcesiran in Patients With AATLD

Stopped: Development project was discontinued

United States, Australia, Austria16 participantsStarted 2021-02-12

Plain-language summary

This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ AATD-associated liver disease (AATLD). The study will be conducted in 3 separate cohorts. A total of up to 16 participants may be enrolled in Cohort 1 and 2. A total number of 30 subjects will be enrolled in cohort 3. The 3 cohorts are differentiated by the duration of the treatment period, the number of doses administered, and the timing of the second liver biopsy.

Who can participate

Age range

18 Years – 75 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 18 to 75 years, inclusive, at the time of consent. * Documented diagnosis of PiZZ-type alpha-1 antitrypsin deficiency, confirmed by genotyping. Historical genotyping data may be used, if available. * AATD-associated liver disease documented by liver biopsy at Screening. * Consent to undergo paired liver biopsies. * Lung, renal and liver function within acceptable limits * Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in this protocol. Exclusion Criteria: * History of chronic liver disease other than non-alcoholic fatty liver disease from any cause other than PiZZ-type alpha-1 antitrypsin deficiency. * Child-Pugh Score B or C. * History of one single severe exacerbation of underlying lung disease in the year prior to randomization. * History of clinically significant respiratory infections (including pneumonia and lower respiratory tract infections), as determined by the Investigator, in the 3 months prior to screening * Use of an RNAi drug at any time.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Timeframe: Up to 2.6 years

Number of Participants With TEAEs and SAEs

Timeframe: Up to 2.6 years

Change From Baseline in Pulmonary Function Tests (PFTs): Forced Vital Capacity (FVC)

Timeframe: Baseline (Day 1), week 96

Change From Baseline in PFT: Forced Expiratory Volume in One Second (FEV1)

Timeframe: Baseline (Day 1), week 96

Change From Baseline in PFT: FEV1/FVC Ratio

Timeframe: Baseline (Day 1), week 96

Change From Baseline in PFT: Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)

Timeframe: Baseline (Day 1), week 96

Change From Baseline in 12 -Lead Electrochardiograms (ECGs): Mean Heart Rate

Timeframe: Baseline (Day 1), week 96

Trial details

NCT IDNCT04764448

SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-12-08

Results submitted2024-12-06

View on ClinicalTrials.gov More Alpha 1-Antitrypsin Deficiency trials

Plain-language summary

Who can participate

Age range

18 Years – 75 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Number of Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Timeframe: Up to 2.6 years

Number of Participants With TEAEs and SAEs

Timeframe: Up to 2.6 years

Change From Baseline in Pulmonary Function Tests (PFTs): Forced Vital Capacity (FVC)

Timeframe: Baseline (Day 1), week 96

Change From Baseline in PFT: Forced Expiratory Volume in One Second (FEV1)

Timeframe: Baseline (Day 1), week 96

Change From Baseline in PFT: FEV1/FVC Ratio

Timeframe: Baseline (Day 1), week 96

Change From Baseline in PFT: Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)

Timeframe: Baseline (Day 1), week 96

Change From Baseline in 12 -Lead Electrochardiograms (ECGs): Mean Heart Rate

Timeframe: Baseline (Day 1), week 96