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Also known as Fazirsiran Injection
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 small molecule being developed for Alpha1-Antitrypsin Deficiency with hepatic impairment and liver disease. It is a siRNA therapeutic targeting the abnormal alpha-1 antitrypsin protein that causes liver damage and fibrosis. Fazirsiran is currently in Phase 3 clinical trials for these rare disease indications.
Fazirsiran is a small interfering RNA (siRNA) therapeutic. It works by targeting the messenger RNA that encodes the abnormal alpha-1 antitrypsin protein, reducing its production. This helps prevent the accumulation of the toxic protein in the liver, which is responsible for liver disease and fibrosis in patients with Alpha1-Antitrypsin Deficiency.
Fazirsiran is being developed by Takeda Pharmaceutical Company Limited, which trades under the ticker TAK. The company is conducting Phase 3 clinical trials to evaluate the safety and efficacy of Fazirsiran in patients with Alpha1-Antitrypsin Deficiency and liver disease.
Fazirsiran is currently in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. The Phase 3 program includes trials evaluating its safety and efficacy in patients with Alpha1-Antitrypsin Deficiency and liver fibrosis, as well as a long-term extension study.
Fazirsiran is being studied in several clinical trials. NCT05677971 is a Phase 3 trial in Alpha1-Antitrypsin Deficiency with liver fibrosis, enrolling 160 participants. NCT05899673 is a Phase 3 extension study for long-term safety. NCT06165341 is a Phase 3 trial in patients with mild liver fibrosis. A Phase 1 trial, NCT05891158, studied Fazirsiran in people with and without liver problems.
Yes, Fazirsiran is also known as Fazirsiran Injection. The drug is administered as an injection and is being developed by Takeda Pharmaceutical Company Limited for the treatment of Alpha1-Antitrypsin Deficiency-related liver disease.