Hereditary transthyretin-mediated amyloidosis(hATTR amyloidosis)
Conditions
Interventions
investigational material(s)
Generic name etc : Patisiran (ALN-TTR02)
INN of investigational material : Patisiran
Therapeutic category code : 129 Other agents affecting peripheral nervous system
Dosage
Sponsors
Alnylam Pharmaceuticals, Inc. / Medpace Japan KK
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Have completed a patisiran study (i.e., completed the last efficacy visit in the parent study) and, in the opinion of the investigator, tolerated study drug Be willing and able to comply with the protocol-required visit schedule and visit requirements and provide written informed consent
Exclusion criteria
Exclusion criteria: Any new or uncontrolled condition that could make the patient unsuitable for participation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| safety Safety and tolerability of long-term dosing of patisiran as measured by the proportion of subjects with adverse events leading to discontinuation of study drug 5 years | — |
Secondary
| Measure | Time frame |
|---|---|
| safety efficacy Changes from baseline in the following items Neurologic impairment score: NIS, mNIS+7, NIS+7 QOL: QOL-DN, EuroQOL(EQ-5D) Others: R-ODS, COMPASS 31, serum TTR level, mBMI, NIS-Weakness(NIS-W), 10-Meter Walk Test and grip strength test | — |
Countries
Asia except Japan, Europe, Japan, North America, Oceania, South America
Outcome results
None listed