A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Sponsor

Alnylam Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT04201418

Collaborator

(none)

Enrollment

Locations

29.2

Actual Duration (Months)

2.5

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.

Condition or Disease	Intervention/Treatment	Phase
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis Polyneuropathy	Drug: Patisiran

Study Design

Study Type:

Observational

Actual Enrollment :

67 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

A Phase 4 Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of Hereditary Transthyretin-Mediated (ATTRv) Amyloidosis With a V122I or T60A Mutation

Actual Study Start Date :

Dec 18, 2019

Actual Primary Completion Date :

May 24, 2022

Actual Study Completion Date :

May 24, 2022

Arms and Interventions

Arm	Intervention/Treatment
Patisiran Prospective Cohort Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy.	Drug: Patisiran Patisiran-lipid complex injection, for intravenous use Other Names: ONPATTRO ALN-TTR02
Patisiran Mixed Cohort Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment.	Drug: Patisiran Patisiran-lipid complex injection, for intravenous use Other Names: ONPATTRO ALN-TTR02
Patisiran Retrospective Cohort Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.	Drug: Patisiran Patisiran-lipid complex injection, for intravenous use Other Names: ONPATTRO ALN-TTR02

Arm

Intervention/Treatment

Patisiran Prospective Cohort

Patients who are naive to patisiran at study enrollment with the intention to initiate commercial patisiran therapy.

Drug: Patisiran

Patisiran-lipid complex injection, for intravenous use

Other Names:

ONPATTRO

ALN-TTR02

Patisiran Mixed Cohort

Patients who are currently on commercial patisiran therapy for less than 12 months at study enrollment.

Drug: Patisiran

Patisiran-lipid complex injection, for intravenous use

Other Names:

ONPATTRO

ALN-TTR02

Patisiran Retrospective Cohort

Patients who have been on commercial patisiran therapy for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.

Drug: Patisiran

Patisiran-lipid complex injection, for intravenous use

Other Names:

ONPATTRO

ALN-TTR02

Outcome Measures

Primary Outcome Measures

Percentage of Participants with Stable or Improved Polyneuropathy Disability (PND) Score at 12 Months Relative to Baseline [Baseline, Month 12]
PND Scores: Stage 0=No symptoms, Stage 1=Sensory disturbances but preserved walking capability, Stage 2=Impaired walking capacity, but ability to walk without a stick or crutches, Stage 3A/B=Walking with the help of 1 or 2 sticks or crutches, Stage 4=confined to wheel chair or bedridden.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Diagnosed with ATTRv amyloidosis with polyneuropathy, with a documented V122I or T60A mutation
PND score of I-IIIB at baseline.
Exposure to commercial patisiran in one of the 3 cohorts:
Prospective Cohort: Naive to patisiran treatment at the time of enrollment with intention to initiate treatment with patisiran.
Mixed cohort: Currently on commercial patisiran therapy for less than 12 months at study enrollment.
Retrospective cohort: Exposed to commercial patisiran treatment for at least 12 months prior to study enrollment, regardless of current treatment status at enrollment.

Exclusion Criteria:

New York Heart Association (NYHA) heart failure classification ≥3
Karnofsky Performance Status (KPS) <60%
Unstable congestive heart failure (CHF)
Known primary amyloidosis (AL) or leptomeningeal amyloidosis
Prior major organ transplant
Previously received patisiran
Previous treatment with a TTR silencing therapy

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Clinical Trial Site	Los Angeles	California	United States	90095
2	Clinical Trial Site	San Diego	California	United States	92103
3	Clinical Trial Site	Aurora	Colorado	United States	80045
4	Clinical Trial Site	Farmington	Connecticut	United States	06030
5	Clinical Trial Site	Jacksonville	Florida	United States	32224
6	Clinical Trial Site	Tampa	Florida	United States	33609
7	Clinical Trial Site	Braselton	Georgia	United States	30517
8	Clinical Trial Site	Macon	Georgia	United States	31201
9	Clinical Trial Site	Chicago	Illinois	United States	60611
10	Clinical Trial Site	Chicago	Illinois	United States	60612
11	Clinical Trial Site	Boston	Massachusetts	United States	02111
12	Clinical Trial Site	Detroit	Michigan	United States	48202
13	Clinical Trial Site	Jackson	Mississippi	United States	39216
14	Clinical Trial Site	Kansas City	Missouri	United States	64111
15	Clinical Trial Site	Omaha	Nebraska	United States	68198
16	Clinical Trial Site	Chapel Hill	North Carolina	United States	27599
17	Clinical Trial Site	Charlotte	North Carolina	United States	28207
18	Clinical Trial Site	Columbus	Ohio	United States	43210
19	Clinical Trial Site	Allentown	Pennsylvania	United States	18103
20	Clinical Trial Site	Bethlehem	Pennsylvania	United States	18015
21	Clinical Trial Site	Lancaster	Pennsylvania	United States	17602
22	Clinical Trial Site	Pittsburgh	Pennsylvania	United States	15212
23	Clinical Trial Site	Pittsburgh	Pennsylvania	United States	15232
24	Clinical Trial Site	Germantown	Tennessee	United States	38138
25	Clinical Trial Site	Austin	Texas	United States	78756
26	Clinical Trial Site	San Antonio	Texas	United States	78229
27	Clinical Trial Site	Milwaukee	Wisconsin	United States	53226