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Also known as Fazirsiran Injection, TAK-999
Fazirsiran · 4 trials · 2 indications
An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of the study intervention, whether or not it is considered related to the study intervention. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of the study intervention. An SAE is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, congenital anomaly/birth defect, suspected transmission of any infectious agent, an important medical event. AEs and SAEs including any pulmonary AEs or SAEs indicative of worsening pulmonary condition (example, pulmonary exacerbation, respiratory infection, significant pulmonary function test decline) will be reported.
Standard pulmonary function parameters will be used to study lung function. Clinical significance of pulmonary function parameters will be determined at the investigator's discretion.
Change from baseline in whole lung 15th percentile density as measured by CT lung densitometry will be assessed.
Vital signs include body temperature, respiratory rate, blood pressure (systolic and diastolic), pulse (beats per minute) and pulse oximetry. Clinical significance of vital signs will be determined at the investigator's discretion.
12-lead ECG will be evaluated. Any clinically significant change in ECG assessments will be determined at the investigator's discretion.
Laboratory parameters assessments include hematology, biochemistry including liver tests, coagulation, and urinalysis. Clinical significance of laboratory parameters will be determined at the investigator's discretion.
Standard pulmonary function parameters measured will be used to study lung function. Clinical significance of pulmonary function parameters will be determined at the investigator's discretion.
Laboratory parameters include hematology, biochemistry including liver tests, coagulation, and urinalysis. Clinical significance of laboratory parameters will be determined at the investigator's discretion.
Reduction from baseline of at least 1 stage of histologic fibrosis METAVIR staging in the centrally read liver biopsy at Week 106 in AATD-LD with METAVIR stage F2 and F3 fibrosis will be assessed.
| Arm | Type | Description |
|---|---|---|
| Fazirsiran 200 mg | EXPERIMENTAL | 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 | PLACEBO_COMPARATOR | Participants will receive fazirsiran matching placebo injection, subcutaneously on Day 1, at Week 4 and Q12W for up to Week 100. |
| Fazirsiran | EXPERIMENTAL | 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. |
| Arm 1, Mild HI: Fazirsiran 200 mg | EXPERIMENTAL | Participants with mild hepatic impairment (HI) will receive fazirsiran 200 milligrams (mg) subcutaneous (SC) injection on Day 1. |
| Arm 2, Moderate HI: Fazirsiran 200 mg | EXPERIMENTAL | Participants with moderate HI will receive fazirsiran 200 mg SC injection on Day 1. The dose may be modified after review of available safety and PK data by the sponsor study team in consultation with the investigator. |
| Arm 3, Severe HI: Fazirsiran 200 mg | EXPERIMENTAL | Participants with severe HI will receive fazirsiran 200 mg SC injection on Day 1. The dose may be modified after review of available safety and PK data by the sponsor study team in consultation with the investigator. |
| Arm 4, Normal Hepatic Function: Fazirsiran 200 mg | EXPERIMENTAL | Participants with normal hepatic function will receive fazirsiran 200 mg SC injection on Day 1. The dose may be modified after review of available safety and PK data by the sponsor study team in consultation with the investigator. |
| Name | Type | Description |
|---|---|---|
| Fazirsiran Injection | DRUG | Fazirsiran will be injected subcutaneously. |
| Placebo | DRUG | Fazirsiran matching placebo. |
| Fazirsiran | DRUG | Fazirsiran SC injection |
Inclusion Criteria: * In the opinion of the investigator, the participant is capable of understanding and fully complying with the protocol requirements and adhering to the protocol schedule. * The participant is able to read, understand, and complete the study questionnaires electronically per the...
Fazirsiran is an investigational RNA therapy being developed for alpha-1 antitrypsin deficiency with associated liver disease, including hepatic impairment and liver fibrosis. It is designed to reduce production of the abnormal alpha-1 antitrypsin protein that accumulates in the liver. It is not yet approved for any indication.
Fazirsiran is a small interfering RNA (siRNA) therapy, belonging to the -siran drug class. It targets the production of alpha-1 antitrypsin protein in the liver, aiming to reduce the accumulation of abnormal protein that causes liver damage in alpha-1 antitrypsin deficiency.
Fazirsiran is being developed by Takeda Pharmaceutical Company Limited, which trades on the New York Stock Exchange under the ticker TAK. Takeda is responsible for the clinical development program evaluating fazirsiran in alpha-1 antitrypsin deficiency liver disease.
Fazirsiran is in Phase 3 clinical development. The program includes three Phase 3 trials and one completed Phase 1 trial. It is an investigational therapy and has not been approved by the FDA or any regulatory agency for commercial use.
Fazirsiran is being studied in several clinical trials. Phase 3 trials include NCT06165341, NCT05899673, and NCT05677971, evaluating safety and efficacy in alpha-1 antitrypsin liver disease. A completed Phase 1 trial, NCT05891158, assessed the drug in people with and without liver problems.
Yes, Fazirsiran Injection is an alternative name for fazirsiran. Both terms refer to the same investigational siRNA therapy developed by Takeda for alpha-1 antitrypsin deficiency liver disease.