Phase 3 N=211 Treatment

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Amyloidosis

Bottom Line

View on ClinicalTrials.gov: NCT02510261 ↗

Enrolled (actual)

211

Serious AEs

63.0%

Results posted

Dec 2023

Primary outcome: Primary: Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation — 49.0; 16.8; 0.0 percentage of participants

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Patisiran (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Alnylam Pharmaceuticals
Primary completion: Nov 2022

Outcome Measures

Outcome	Result	p-value
PRIMARY Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation	49.0; 16.8; 0.0	—
SECONDARY Change From Baseline in the Total Neuropathy Impairment Score (NIS) at Year 5	81.47; 62.26; 35.48; 11.45; 10.72; 11.18	—
SECONDARY Change From Baseline in the Total Modified NIS (mNIS +7) Composite Score At Year 3	101.07; 74.72; 45.66; -6.69; 8.07; 5.46	—
SECONDARY Change From Baseline in the NIS+7 Total Score at Week 52	98.42; 78.74; 49.66; 1.44; 1.49; 3.23	—
SECONDARY Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Total Score at Year 5	72.7; 54.5; 34.0; 3.3; 4.5; -18.0	—
SECONDARY Change From Baseline in the EuroQOL-5 Dimensions-5 Levels (EQ-5D-5L) Index Score at Year 5	0.4614; 0.6444; 0.7663; 0.0361; -0.0548; -0.0166	—
SECONDARY Change From Baseline in the EuroQoL Visual Analogue Scale (EQ-VAS) Score at Year 5	46.0; 57.8; 69.1; 9.3; -1.5; 1.5	—
SECONDARY Change From Baseline in the Composite Autonomic Symptom Score (COMPASS 31) Total Score at Week 52	33.92; 25.37; 15.93; -3.70; 0.37; -1.24	—
SECONDARY Change From Baseline in the Modified Body Mass Index (mBMI) at Year 5	881.8; 970.7; 1002.3; 74.0; 31.6; 77.8	—
SECONDARY Change From Baseline in the Rasch-built Overall Disability Scale (R-ODS) at Year 5	20.3; 29.7; 36.7; -2.9; -4.1; -2.7	—
SECONDARY Change From Baseline in the NIS+7 Component: NIS-Weakness (NIS-W) Score at Year 5	47.01; 33.26; 15.02; 9.10; 7.37; 6.97	—
SECONDARY Change From Baseline in the 10-meter Walk Test (10-MWT) Speed at Year 5	0.538; 0.851; 1.262; 0.011; -0.050; -0.121	—
SECONDARY Change From Baseline in the Hand Grip Strength at Week 52	10.23; 18.03; 27.86; 0.14; -0.34; -0.28	—
SECONDARY Number of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Stage	1; 9; 3; 8; 35; 5	—
SECONDARY Number of Participants With Change From Baseline in the Familial Amyloidotic Polyneuropathy (FAP) Stage	0; 3; 1; 5; 13; 2	—
SECONDARY Number of Participants With Change From Baseline in the New York Heart Association (NYHA) Classification	2; 13; 1; 4; 18; 4	—
SECONDARY Change From Baseline in the Intraepidermal Nerve Fiber Density (IENFD) at Year 5	4.71; 5.41; 8.99; 0.28; -2.75; -5.53	—
SECONDARY Change From Baseline in the Sweat Gland Nerve Fiber Density (SGNFD) at Year 5	10.82; 9.67; 9.14; -0.56; -2.49; -3.29	—
SECONDARY Change From Baseline in the Dermal Amyloid Burden at Year 5	8.163; 8.205; 5.908; -3.775; -1.103; -3.100	—
SECONDARY Change From Baseline in the Cardiac Biomarker: Serum Troponin I at Year 5	0.109; 0.112; 0.088; 0.546; -0.005; 0.014	—
SECONDARY Change From Baseline in the Cardiac Biomarker: N-terminal Prohormone of B-type Natriuretic Peptide (NT-proBNP) at Year 5	1957.649; 1017.217; 281.899; -18.490; 209.126; 78.146	—
SECONDARY Change From Baseline in the Echocardiogram Parameter: Average Peak Longitudinal Strain at Year 5	-15.63; -15.96; -17.72; 3.43; 2.30; 1.62	—
SECONDARY Change From Baseline in the Echocardiogram Parameter: Left Ventricular (LV) Mass at Year 5	238.166; 236.604; 198.570; 2.723; -4.222; -17.152	—
SECONDARY Change From Baseline in the Echocardiogram Parameter: LV End-diastolic Volume at Year 5	80.591; 83.616; 107.818; 7.173; -1.279; -11.710	—
SECONDARY Change From Baseline in the Echocardiogram Parameter: LV Relative Wall Thickness at Year 5	0.780; 0.717; 0.593; -0.066; -0.061; -0.037	—
SECONDARY Change From Baseline in the Echocardiogram Parameter: Mean LV Wall Thickness at Year 5	1.538; 1.463; 1.249; -0.035; -0.041; -0.042	—
SECONDARY Change From Baseline in the Echocardiogram Parameter: Cardiac Output at Year 5	3.547; 3.840; 4.873; -0.010; -0.283; -0.594	—
SECONDARY Percent Change From Baseline in Serum TTR Levels at Year 5	-90.010; -37.660; -39.965	—

Summary

The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).

Eligibility 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

Any new or uncontrolled condition that could make the participant unsuitable for participation

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02510261). 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.