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Label indication AMVUTTRA is a transthyretin-directed small interfering RNA indicated for the treatment of: the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults ( 1.1 ) the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits ( 1.2 ) 1.1 Polyneuropathy of Hereditary… FDA label
Also known as ALN-TTRSC (revusiran), ALN-TTRSC, ALN-TTRSC04, Vutrisiran, AMVUTTRA
ALN-TTRSC02 · 5 trials · 4 indications
An AE is any untoward medical occurrence in a patient or clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment.
All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) were compared between treatment groups using a modified Andersen-Gill model with a robust variance. All-cause mortality included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits. The number of participants with at least one CV event or all-cause mortality event have been reported here. No imputation was done for participants who dropped out early for the primary analysis of this composite outcome endpoint.
All-cause mortality and recurrent CV events (CV hospitalizations and urgent HF visits) were compared between treatment groups using a modified Andersen-Gill model with a robust variance. All-cause mortality included heart transplantation and LVAD placement events along with deaths; recurrent CV events included CV hospitalizations and urgent HF visits. The number of participants with at least one CV event or all-cause mortality event have been reported here. No imputation was done for participants who dropped out early for the primary analysis of this composite outcome endpoint.
| Arm | Type | Description |
|---|---|---|
| Vutrisiran 25 mg | EXPERIMENTAL | Participants will receive vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M). |
| Placebo | PLACEBO_COMPARATOR | Participants received vutrisiran matching placebo administered SC q3M during the 36-month DB period. After the DB period, participants receive vutrisiran, 25 mg, SC injection, q3M for up to 24 months in the OLE period. |
| ALN-TTRSC04 | EXPERIMENTAL | Participants will be administered a single dose of ALN-TTRSC04. |
| ALN-TTRSC02 | ACTIVE_COMPARATOR | - |
| Sterile normal saline 0.9% for SC administration | PLACEBO_COMPARATOR | - |
| ALN-TTRSC (revusiran) | ACTIVE_COMPARATOR | - |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Vutrisiran | DRUG | Vutrisiran administered SC q3M |
| Sterile Normal Saline (0.9% NaCl) | DRUG | Sterile normal saline (0.9% NaCl) will be administered by SC injection. |
| ALN-TTRSC04 | DRUG | ALN-TTRSC04 will be administered by subcutaneous (SC) injection. |
| Placebo | DRUG | Placebo will be administered by SC injection. |
| ALN-TTRSC02 | DRUG | Ascending doses of ALN-TTRSC02 by subcutaneous (SC) injection |
| ALN-TTRSC (revusiran) | DRUG | Ascending doses of ALN-TTRSC (revusiran) by subcutaneous (SC) injection |
Inclusion Criteria: * Patients who are participating in the patisiran parent studies ALN-TTR02-011 or ALN-TTR02-014 or who have completed the 24-month OLE Period in the vutrisiran parent study ALN-TTRSC02-003 Exclusion Criteria: * Has permanently discontinued study drug administration while parti...
ALN-TTRSC02 is an investigational RNA therapy developed by Alnylam Pharmaceuticals for transthyretin-mediated amyloidosis, also called ATTR amyloidosis. It is designed to reduce production of the transthyretin protein. It has completed Phase 1 clinical testing in healthy volunteers and remains in clinical development.
ALN-TTRSC02 targets transthyretin, or TTR, a protein produced mainly in the liver. By targeting TTR, the RNA therapy aims to lower transthyretin levels, which is the protein that misfolds and deposits in tissues in TTR-mediated amyloidosis. TTR is the molecular target of this investigational therapy.
ALN-TTRSC02 is being developed by Alnylam Pharmaceuticals, Inc., which trades on the Nasdaq under the ticker ALNY. Alnylam is the sponsor of the Phase 1 studies of ALN-TTRSC02 conducted in the United Kingdom.
ALN-TTRSC02 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA. Two Phase 1 studies of ALN-TTRSC02 have been completed, and no active trials of this asset are currently listed.
ALN-TTRSC02 has been studied in two completed Phase 1 trials: NCT02797847, a safety and tolerability study in 80 healthy subjects, and NCT05661916, a study of safety, tolerability, pharmacokinetics and pharmacodynamics in 96 healthy subjects. Both were conducted in the United Kingdom.
ALN-TTRSC02 is also known as ALN-TTRSC, ALN-TTRSC04, and revusiran. Revusiran is the earlier name associated with the ALN-TTRSC program, which was studied in the completed Phase 1 trial NCT01814839 in 85 healthy volunteers. These names refer to the same Alnylam RNA therapy program for TTR-mediated amyloidosis.