ALN-TTR02-006
Not Applicable
Completed
- Conditions
- Hereditary transthyretin-mediated amyloidosis(hATTR amyloidosis)
- Registration Number
- JPRN-jRCT2080224174
- Lead Sponsor
- Alnylam Pharmaceuticals, Inc. / Medpace Japan KK
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 211
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
Any new or uncontrolled condition that could make the patient unsuitable for participation
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method safety<br>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<br>5 years
- Secondary Outcome Measures
Name Time Method safety<br>efficacy<br>Changes from baseline in the following items<br>Neurologic impairment score: NIS, mNIS+7, NIS+7<br>QOL: QOL-DN, EuroQOL(EQ-5D)<br>Others: R-ODS, COMPASS 31, serum TTR level, mBMI, NIS-Weakness(NIS-W), 10-Meter Walk Test and grip strength test