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Anti-sense oligonucleotide

Fazirsiran 200 mg 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

Study 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.Check my eligibility

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.See study design

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

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I have been diagnosed with the PiZZ genotype for AATD.

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

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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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

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

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

Find a Location

Who is running the clinical trial?

TakedaLead Sponsor

1,203 Previous Clinical Trials

4,177,894 Total Patients Enrolled

Study DirectorStudy DirectorTakeda

1,211 Previous Clinical Trials

489,222 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there any available openings for patients to participate in this medical study?

"Based on the information provided by clinicaltrials.gov, this particular clinical trial is not actively seeking participants at present. The initial posting of this study was on January 18th, 2024, and it was most recently updated on January 11th, 2024. However, there are currently a total of 272 other ongoing clinical trials that are actively recruiting patients."

Answered by AI

Is the age criterion for participants in this medical study limited to individuals below 55 years of age?

"As outlined in the study's inclusion criteria, individuals aged 18 and above but not exceeding 75 are eligible for participation."

Answered by AI

What are the specific criteria for eligibility to partake in this study?

"To be eligible for this research study, potential participants must have a medical condition known as alpha-1 antitrypsin deficiency and fall within the age range of 18 to 75 years. A total of 50 individuals will be selected to participate in this clinical trial."

Answered by AI

Has the Food and Drug Administration (FDA) officially sanctioned the use of Fazirsiran at a dosage of 200 mg?

"Considering that this trial is in Phase 3 and has accumulated multiple rounds of safety data, our team at Power rates the safety of Fazirsiran 200 mg as a 3 on a scale from 1 to 3."

Answered by AI

What are the main goals that this investigation aims to achieve?

"The primary objective of this study, spanning from baseline to week 100, is to determine the number of participants who experience adverse events (AEs) and serious AEs (SAEs). Secondary outcomes will include assessing changes in Vibration-Controlled Transient Elastography (VCTE)-Derived Liver Stiffness compared to baseline. Additionally, it aims to evaluate the number of participants with no change or a decrease in Intrahepatic Portal Inflammation Score at Week 106 based on pathology slide reads. The inflammation score ranges from 0-3, where higher scores indicate more severe inflammation. Lastly, the study will"

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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)

2024. "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)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06165341.

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