Fazirsiran for Alpha-1 Antitrypsin Deficiency

Recruiting in Palo Alto (17 mi)

+71 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Takeda

Disqualifiers: Liver decompensation, Chronic liver diseases, others

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing fazirsiran, a drug that may reduce liver scarring and improve liver health, in patients with liver fibrosis. The drug works by decreasing harmful proteins and reducing inflammation in the liver.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Eligibility Criteria

Adults aged 18-75 with Alpha-1 Antitrypsin Deficiency (AATD) causing liver scarring, who are non-smokers for at least a year and have a BMI of 18.0-39.0 kg/m2 can join this trial. They must not have liver cancer, drug abuse history within the last year, certain blood or liver issues, other chronic liver diseases, recent malignancies except some skin cancers or in situ cervical cancer.

Inclusion Criteria

My BMI is between 18.0 and 39.0.

I have been diagnosed with the PiZZ genotype for AATD.

My liver fibrosis is at stage F2, F3, or F4 based on a recent biopsy.

+5 more

Exclusion Criteria

I have had medium or large varices, or ones with red signs, but small ones without bleeding are okay.

I had cancer in the last 5 years, but it was either skin cancer, superficial bladder tumors, or in situ cervical cancer, or I've been disease-free for over a year.

I have been treated with RNAi therapy for AATD-LD before.

+10 more

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive fazirsiran or placebo via subcutaneous injection on Day 1, at Week 4, and then every 12 weeks thereafter up to Week 196

196 weeks

Injections on Day 1, Week 4, and every 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including liver biopsies and assessments of liver function and fibrosis

34 weeks

Liver biopsies and assessments at Week 106, Week 202, and up to Week 230

Participant Groups

The study is testing if Fazirsiran injections can reduce liver scarring compared to placebo in people with AATD-related fibrosis. It will also assess how the body processes Fazirsiran and its impact on disease progression using biopsies and biomarkers.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: FazirsiranExperimental Treatment1 Intervention

Participants will receive fazirsiran 200 milligram per milliliter (mg/ml) subcutaneous (SC) injection on Day 1, at Week 4, and then every 12 weeks (Q12 W) thereafter up to Week 196.

Group II: PlaceboPlacebo Group1 Intervention

Participants will receive placebo on Day 1, at Week 4, and Q12 W thereafter up to Week 196.