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)
Sponsored by Takeda
About this trial
Last updated 18 days ago
Study ID
TAK-999-3002
Status
Recruiting
Type
Interventional
Phase
Phase 3
Placebo
Yes
Accepting
18 to 75 Years
All
Not accepting
Healthy Volunteers
Trial Timing
Started a year ago
What is this trial about?
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 endstage 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.
What are the participation requirements?
Inclusion Criteria
Men and women from 18 to 75 years old can take part in this study.
Must have the specific gene for abnormal Z-AAT protein (confirmed by a genetic test), and already have Stage F1 liver scarring (fibrosis) shown in the liver biopsy.
Must have acceptable lung function.
Exclusion Criteria
Cannot have liver scarring at stage F2, F3, or F4 as shown in the liver biopsy.
Cannot have had symptoms or signs of liver failure or other types of chronic liver disease.
Cannot have had recent, ongoing, or frequent lung infections, or breathing difficulties, or need long term treatment with oxygen.
Cannot be exposed to chemicals, mineral dust, or metals that are toxic to the lungs.
Cannot be smokers.
Additional entry criteria will be discussed with the study doctor.
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For more information, view the full study details:
NCT06165341