← Back to Search

Anti-sense oligonucleotide

Fazirsiran for Alpha-1 Antitrypsin Deficiency

Phase 3

Recruiting

Research Sponsored by Takeda

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

The participant must have a diagnosis of the protease inhibitor Z mutation (PiZZ) genotype AATD. A diagnosis of PiZZ from source-verifiable medical records is permitted. Otherwise, participants must undergo PiZZ confirmatory testing (genotyping for PiS and PiZ alleles) at screening. PiMZ or PiSZ genotypes are not permitted.

The participant has evidence of METAVIR stage F1 liver fibrosis, evaluated by a centrally read baseline liver biopsy during the screening period; or confirmed as meeting all the entry criteria by central reading from a previous biopsy conducted within 1 year before the screening period using an adequate liver biopsy and slides as defined in the study laboratory manual.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from start of study drug administration up to eos (week 124)

Awards & highlights

Pivotal Trial

Summary

This trial is studying a medication called fazirsiran in people with a liver disease caused by an abnormal protein called Z-AAT. The medication aims to reduce the build-up of this abnormal protein in the

Who is the study for?

This trial is for adults aged 18-75 with Alpha-1 Antitrypsin Deficiency (AATD) and mild liver scarring. Participants must have a specific genetic form of AATD (PiZZ), no liver cancer, and be able to follow the study plan. They need a negative COVID-19 test and must consent to two liver biopsies.

What is being tested?

The trial tests Fazirsiran, which aims to reduce abnormal alpha-1 antitrypsin protein in the liver that causes scarring. Over about 2 years, participants will receive either Fazirsiran or a placebo injection to assess long-term safety and treatment tolerance.

What are the potential side effects?

While not explicitly listed here, potential side effects may include reactions at the injection site, changes in liver function tests, fatigue, abdominal pain or discomfort related to the condition or treatment process.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with the PiZZ genotype for AATD.

Select...

My liver has mild fibrosis, confirmed by a recent biopsy.

Select...

I do not have liver cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from start of study drug administration up to eos (week 124)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from start of study drug administration up to eos (week 124)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from start of study drug administration up to eos (week 124) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change From Baseline in Whole Lung 15th Percentile Density as Measured by Computed Tomography (CT) Lung Densitometry

Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs)

Number of Participants With Clinically Significant Change From Baseline in Pulmonary Function Parameters

+3 more

Secondary study objectives

Change From Baseline in Intrahepatic Portal Inflammation Score in Liver Biopsy at Week 106

Change From Baseline in Intrahepatic Z-AAT Protein Polymer Burden Assessed by Periodic Acid Schiff Plus Diastase (PAS+D) Staining in Liver Biopsy at Week 106

Change From Baseline in Markers of Liver Injury

+6 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Fazirsiran 200 mgExperimental Treatment1 Intervention

Participants will receive fazirsiran 200 milligrams (mg), injection, subcutaneously on Day 1, at Week 4 and then every 12 weeks (Q12W) for up to Week 100.

Group II: PlaceboPlacebo Group1 Intervention

Participants will receive fazirsiran matching placebo injection, subcutaneously on Day 1, at Week 4 and Q12W for up to Week 100.

0 / 3Eligibility criteria met