Phase 2 N=27 Treatment

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

Bottom Line

View on ClinicalTrials.gov: NCT01961921 ↗

Enrolled (actual)

Serious AEs

25.9%

Results posted

Oct 2018

Primary outcome: Primary: The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation — 26; 7; 2 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: ALN-TTR02 (patisiran) administered by intravenous (IV) infusion (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Alnylam Pharmaceuticals
Primary completion: Jul 2016

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	—

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).

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.

View full record on ClinicalTrials.gov →

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.