Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)

Conditions

• Alpha1-Antitrypsin Deficiency

Eligibility Criteria

Sex

ALL

Age

18 Years - 75 Years

Healthy Volunteers

Not accepted

Interventions

• Fazirsiran Injection — DRUG
  Fazirsiran will be injected subcutaneously.
• Placebo — DRUG
  Fazirsiran matching placebo.

Study Details

The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT. Making an abnormal version of the AAT protein can result in liver disease as Z-AAT builds up in liver cells, which leads to liver problems such as liver scarring (fibrosis), continuing liver damage (cirrhosis), and eventually end stage liver disease. Fazirsiran is a medicine that reduces the creation of the Z-AAT protein and thus the build-up of this abnormal protein in the liver. People with this type of liver disease who already have mild liver scarring will take part in the study. They will be treated with fazirsiran or a placebo for about 2 years. This study will check the long-term safety of fazirsiran, whether participants tolerate the treatment and if there are any effects on liver scarring. A liver biopsy, a way of collecting a small tissue sample from the liver, will be taken twice during the study.

Key Dates

Start date

Mar 1, 2024

Status verified

Mar 2026

Primary completion

Aug 26, 2028

Completion

Aug 26, 2028

Study Design

Enrollment

50 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Fazirsiran 200 mg
  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.
• Placebo Comparator: Placebo
  Participants will receive fazirsiran matching placebo injection, subcutaneously on Day 1, at Week 4 and Q12W for up to Week 100.

Primary Outcome Measure

Number of Participants With Adverse Events (AEs) and Serious AEs (SAEs) [ Time Frame: From start of study drug administration up to End of study (EOS) (Week 124) ]

Central Contacts

• Takeda Contact
  +1-877-825-3327
  [email protected]

Locations (20)

Find similar trials in Phoenix, AZ

Related Studies

• Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
  PHASE3 · Recruiting · Takeda · Birmingham, Alabama
• A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
  Recruiting · Takeda · Gainesville, Florida