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

Sponsor

Alnylam Pharmaceuticals (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT02510261

Collaborator

(none)

211

Enrollment

Locations

Arm

Duration (Months)

3.8

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Amyloidosis	Drug: Patisiran (ALN-TTR02)	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

211 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients With Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Patisiran Clinical Study

Study Start Date :

Jul 1, 2015

Anticipated Primary Completion Date :

Aug 1, 2022

Anticipated Study Completion Date :

Aug 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Patisiran (ALN-TTR02)	Drug: Patisiran (ALN-TTR02) administered by intravenous (IV) infusion

Arm

Intervention/Treatment

Experimental: Patisiran (ALN-TTR02)

Drug: Patisiran (ALN-TTR02)

administered by intravenous (IV) infusion

Outcome Measures

Primary Outcome Measures

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 [Up to 5 years]

Secondary Outcome Measures

Assessment of changes from baseline in neurologic impairment assessed using the Neuropathy Impairment Score (NIS) associated with long-term dosing of patisiran [Baseline up to 5 years]
Assessment of changes from baseline in neurologic impairment assessed using the Modified NIS (mNIS +7) composite score associated with long-term dosing of patisiran [Baseline up to 3 years]
Assessment of changes from baseline in neurologic impairment assessed using NIS+7 associated with long-term dosing of patisiran [Baseline up to 3 years]
Assessment of changes from baseline in quality of life using the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire associated with long-term dosing of patisiran [Baseline up to 5 years]
Assessment of changes from baseline in quality of life using the EuroQOL (EQ-5D) questionnaire associated with long-term dosing of patisiran [Baseline up to 5 years]
Assessment of changes from baseline in autonomic function assessed using the Composite Autonomic Symptom Score (COMPASS 31) with long-term dosing of patisiran [Baseline up to 52 weeks]
Assessment of changes from baseline in serum TTR lowering associated with long-term dosing of patisiran [Baseline up to 5 years]
Assessment of changes from baseline in nutritional status using modified body mass index (mBMI) associated with long-term dosing of patisiran [Baseline up to 5 years]
Assessment of changes from baseline in disability reported by patients using the Rasch-built Overall Disability Scale (R-ODS) associated with long-term dosing of patisiran [Baseline up to 5 years]
Assessment of changes from baseline in motor function assessed by NIS-Weakness (NIS-W) with long-term dosing of patisiran [Baseline up to 5 years]
Assessment of changes from baseline in motor function assessed by timed 10-meter walk test with long-term dosing of patisiran [Baseline up to 5 years]
Assessment of changes from baseline in motor function assessed by grip strength test with long-term dosing of patisiran [Baseline up to 52 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 85 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Trial Site	La Mesa	California	United States
2	Clinical Trial Site	Aurora	Colorado	United States
3	Clinical Trial Site	Jacksonville	Florida	United States	32224
4	Clinical Trial Site	Chicago	Illinois	United States
5	Clinical Trial Site	Baltimore	Maryland	United States
6	Clinical Trial Site	Boston	Massachusetts	United States
7	Clinical Trial Site	Detroit	Michigan	United States
8	Clinical Trial Site	Rochester	Minnesota	United States
9	Clinical Trial Site	Joplin	Missouri	United States	64804
10	Clinical Trial Site	Saint Louis	Missouri	United States
11	Clinical Trial Site	Cooperstown	New York	United States
12	Clinical Trial Site	New York	New York	United States	10029
13	Clinical Trial Site	New York	New York	United States	10032
14	Clinical Trial Site	Columbus	Ohio	United States	43210
15	Clinical Trial Site	Buenos Aires		Argentina
16	Clinical Trial Site	Westmead		Australia
17	Clinical Trial Site	Rio de Janeiro		Brazil
18	Clinical Trial Site	São Paulo		Brazil
19	Clinical Trial Site	Sofia		Bulgaria
20	Clinical Trial Site	Vancouver	British Columbia	Canada
21	Clinical Trial Site	Nicosia		Cyprus
22	Clinical Trial Site	Fort De France	Martinique	France	97261
23	Clinical Trial Site	Saint-Pierre	Reunion Island	France	97448
24	Clinical Trial Site	Creteil		France
25	Clinical Trial Site	Le Kremlin-bicetre		France
26	Clinical Trial Site	Lille		France
27	Clinical Trial Site	Marseille Cedex		France
28	Clinical Trial Site	Cologne		Germany	50937
29	Clinical Trial Site	Heidelberg		Germany
30	Clinical Trial Site	Muenster		Germany
31	Clinical Trial Site	Pavia		Italy
32	Clinical Trial Site	Rome		Italy
33	Clinical Trial Site	Sicily		Italy
34	Clinical Trial Site	Ehime		Japan
35	Clinical Trial Site	Fukuoka		Japan
36	Clinical Trial Site	Hiroshima		Japan
37	Clinical Trial Site	Kumamoto		Japan
38	Clinical Trial Site	Nagano		Japan
39	Clinical Trial Site	Okawasuji		Japan
40	Clinical Trial Site	Ono		Japan
41	Clinical Trial Site	Ōita		Japan
42	Clinical Trial Site	Seoul		Korea, Republic of
43	Clinical Trial Site	Kuala Lumpur		Malaysia
44	Clinical Trial Site	Tlalpan		Mexico
45	Clinical Trial Site	Groningen		Netherlands
46	Clinical Trial Site	Lisbon		Portugal
47	Clinical Trial Site	Porto		Portugal
48	Clinical Trial Site	Barcelona		Spain
49	Clinical Trial Site	Huelva		Spain
50	Clinical Trial Site	Madrid		Spain
51	Clinical Trial Site	Palma De Mallorca		Spain
52	Clinical Trial Site	Umeå		Sweden
53	Clinical Trial Site	Taipei		Taiwan	11217
54	Clinical Trial Site	Taipei		Taiwan
55	Clinical Trial Site	Istanbul		Turkey
56	Clinical Trial Site	London		United Kingdom