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Also known as ALN-TTR02
ALN-TTR01 · 2 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| ALN-TTR02 | ACTIVE_COMPARATOR | - |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | - |
| ALN-TTR01 | ACTIVE_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| ALN-TTR02 | DRUG | Dose levels between 0.01 and 0.5 mg/kg by intravenous (IV) infusion |
| Sterile Normal Saline (0.9% NaCl) | DRUG | Calculated volume to match active comparator |
| ALN-TTR01 | DRUG | Dose levels between 0.01 and 1.0 mg/kg by intravenous (IV) infusion |
Inclusion Criteria: * Body mass index must be between 18.0 kg/m2 and ≤ 31.5 kg/m2; * Non-smokers for at least 3 months preceding screening; * Females subjects must be of non-childbearing potential e.g., post-menopausal or pre-menopausal with surgical sterilization; * Males agree to use appropriate ...
ALN-TTR01 is an investigational drug developed by Alnylam Pharmaceuticals for transthyretin-mediated amyloidosis, a rare disease in which abnormal transthyretin protein deposits damage tissues and organs. It is a small molecule therapy that has been studied in early-stage clinical trials.
ALN-TTR01 is being developed for the treatment of transthyretin-mediated amyloidosis, also known as ATTR amyloidosis. This condition involves the buildup of misfolded transthyretin protein in organs such as the heart and nerves. ALN-TTR01 is intended to address the underlying cause of this rare disease.
ALN-TTR01 is made by Alnylam Pharmaceuticals, Inc., a biotechnology company focused on RNA interference therapeutics. Alnylam trades on the Nasdaq under the ticker symbol ALNY. The company is developing ALN-TTR01 for transthyretin-mediated amyloidosis.
ALN-TTR01 is in Phase 1 clinical development. It has completed a Phase 1 trial in patients with transthyretin-mediated amyloidosis. As an investigational drug, it has not been approved by the FDA for any indication and remains in early-stage testing.
ALN-TTR01 has been evaluated in a completed Phase 1 trial, NCT01148953, which studied its safety and tolerability in patients with transthyretin-mediated amyloidosis across France, Portugal, Sweden, and the United Kingdom. A related Phase 1 trial, NCT01559077, tested ALN-TTR02 in healthy volunteers in the United Kingdom.
ALN-TTR01 and ALN-TTR02 are related names, with ALN-TTR02 sometimes used to refer to a similar investigational compound from Alnylam. Both are associated with the same development program for transthyretin-mediated amyloidosis, but they are distinct drug candidates studied in separate Phase 1 trials.