Phase 2
Completed N=27
The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
TTR-mediated Amyloidosis
Source: ClinicalTrials.gov NCT01961921 ↗
Enrolled (actual)
27
Serious AEs
25.9%
Results posted
Oct 2018
Primary outcomePrimary: The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation — 26; 7; 2 participants
Summary
The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation |
26; 7; 2 | — |
| SECONDARY Percentage Change From Baseline in Serum TTR Levels |
-82.06; -79.73; -92.54 | — |
| SECONDARY Change From Baseline in the Modified Neuropathy Impairment Score +7 (mNIS+7) |
50.5; -7.00 | — |
| SECONDARY Change From Baseline in Quality of Life and Disability as Assessed by the EuroQoL (Quality of Life)-5 Dimensions (EQ-5D), EuroQoL Visual Analog Scale (EQ-VAS) Questionnaires and Rasch-built Overall Disability Scale (R-ODS) |
0.76; 0.00; 70.0; 0.0; 38.5; -1.0 | — |
| SECONDARY Change in Gait Speed With 10-meter Walk Test |
1.13; 0.09 | — |
| SECONDARY Mean Change From Baseline in Hand Grip Strength |
23.88; 1.54 | — |
| SECONDARY Change From Baseline in Nutritional Status (Modified Body Mass Index, mBMI) |
1049.09; -39.85 | — |
Eligibility Criteria
Inclusion Criteria
- Previously received and tolerated ALN-TTR02 (patisiran) in Study ALN-TTR02-002.
- Adequate Karnofsky performance status, liver function, and renal function.
Exclusion Criteria
- Pregnant or nursing.
- Has had a liver transplant.
- Has a New York Heart Association heart failure classification >2.
- Has unstable angina.
- Has uncontrolled clinically significant cardiac arrhythmia.
Data sourced from ClinicalTrials.gov (NCT01961921). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.