Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)
NCT ID: NCT03945292
Last Updated: 2025-11-04
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
40 participants
INTERVENTIONAL
2019-08-07
2023-09-18
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Fazirsiran 25 mg DB/200 mg OL
Double-blind (DB) Period: Participants with no fibrosis: Administered on Day 1 and Week 4. Participants with fibrosis: Administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses.
Open-label (OL) Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 25 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study.
Fazisiran Injection
solution for subcutaneous (sc) injection
Fazirsiran 100 mg DB/200 mg OL
DB Period: Participants with no fibrosis: Fazirsiran 100 mg administered on Day 1 and Week 4. Participants with fibrosis: Fazirsiran 100 mg administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses.
OL Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 100 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study.
Fazisiran Injection
solution for subcutaneous (sc) injection
Fazirsiran 200 mg DB/200 mg OL
DB Period: Participants with no fibrosis: Fazirsiran 200 mg administered on Day 1 and Week 4.
Participants with fibrosis: Fazirsiran 200 mg administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses.
OL Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 200 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study
Fazisiran Injection
solution for subcutaneous (sc) injection
Placebo DB / Fazirsiran 200 mg OL
DB Period: Participants with no fibrosis: Placebo administered on Day 1 and Week 4. Participants with fibrosis: Placebo administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses.
OL Period: Participants with fibrosis at Screening who received double-blind (DB) placebo and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study.
Fazisiran Injection
solution for subcutaneous (sc) injection
Placebo
sterile normal saline (0.9% NaCl), calculated to match active comparator, for sc injection
Interventions
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Fazisiran Injection
solution for subcutaneous (sc) injection
Placebo
sterile normal saline (0.9% NaCl), calculated to match active comparator, for sc injection
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Liver biopsy at Screening indicating liver fibrosis (score less than F4); a patient with no fibrosis may participate based on a previous biopsy conducted within one year
* Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
* Willing to provide written informed consent and to comply with study requirements
* Non-smoker for at least 1 year
* No abnormal finding of clinical relevance at Screening
Exclusion Criteria
* Previous diagnosis or diagnosis at Screening of definitive liver cirrhosis
* Previous lung or liver transplant due to AATD
* Regular use of alcohol within one month prior to Screening
* Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study involving therapeutic intervention
* Use of illicit drugs within 1 year prior to Screening
18 Years
75 Years
ALL
No
Sponsors
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Arrowhead Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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Research Site 5
Birmingham, Alabama, United States
Research Site 9
Phoenix, Arizona, United States
Research Site 14
La Jolla, California, United States
Research Site 17
Los Angeles, California, United States
Research Site 11
Sacramento, California, United States
Research Site 15
San Francisco, California, United States
Research Site 13
Stanford, California, United States
Research Site 2
Gainesville, Florida, United States
Research Site 7
Indianapolis, Indiana, United States
Research Site 3
Iowa City, Iowa, United States
Research Site 10
St Louis, Missouri, United States
Research Site 12
New York, New York, United States
Research Site 1
Charleston, South Carolina, United States
Research Site 4
Nashville, Tennessee, United States
Research Site 19
Houston, Texas, United States
Research Site 16
Salt Lake City, Utah, United States
Research Site 27
Aachen, , Germany
Research Site 22
Pavia, , Italy
Research Site 23
Leiden, , Netherlands
Research Site 24
Funchal, , Portugal
Research Site 20
Barcelona, , Spain
Countries
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References
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Clark VC, Strange C, Strnad P, Sanchez AJ, Kwo P, Pereira VM, van Hoek B, Barjaktarevic I, Corsico AG, Pons M, Goldklang M, Gray M, Kuhn B, Vargas HE, Vierling JM, Vuppalanchi R, Brantly M, Kappe N, Chang T, Schluep T, Zhou R, Hamilton J, San Martin J, Loomba R. Fazirsiran for Adults With Alpha-1 Antitrypsin Deficiency Liver Disease: A Phase 2 Placebo Controlled Trial (SEQUOIA). Gastroenterology. 2024 Oct;167(5):1008-1018.e5. doi: 10.1053/j.gastro.2024.06.028. Epub 2024 Jul 2.
Remih K, Amzou S, Strnad P. Alpha1-antitrypsin deficiency: New therapies on the horizon. Curr Opin Pharmacol. 2021 Aug;59:149-156. doi: 10.1016/j.coph.2021.06.001. Epub 2021 Jul 10.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2018-003385-14
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AROAAT2001
Identifier Type: -
Identifier Source: org_study_id
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