NCT02595983

Brief Summary

The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation. Dosing has been discontinued; patients are being followed-up for safety.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2015

Shorter than P25 for phase_2

Geographic Reach
6 countries

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2015

Completed
29 days until next milestone

First Submitted

Initial submission to the registry

October 30, 2015

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 4, 2015

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 6, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 6, 2017

Completed
2.1 years until next milestone

Results Posted

Study results publicly available

March 28, 2019

Completed
Last Updated

March 28, 2019

Status Verified

March 1, 2019

Enrollment Period

1.4 years

First QC Date

October 30, 2015

Results QC Date

February 23, 2018

Last Update Submit

March 26, 2019

Conditions

Transthyretin (TTR)-Mediated AmyloidosisFamilial Amyloidotic Polyneuropathy (FAP)ATTR AmyloidosisFamilial Amyloid Neuropathies

Keywords

FAPFamilial Amyloidotic PolyneuropathyATTR AmyloidosisOrthotopic Liver TransplantRNAi therapeutic

Outcome Measures

Primary Outcomes (1)

  • Percentage Change From Baseline in Serum TTR at Month 6

    A negative percentage change from baseline at Month 6 indicates a reduction in serum TTR level.

    Month 6

Secondary Outcomes (4)

  • Percentage Change From Baseline in Serum TTR Over 18 Months

    Weeks 3, 7, 12, 18, 24, 26 (Month 6), 39 (Month 9), 52 (Month 12), 57, 78 (Month 18)

  • Change From Baseline in Modified Neurological Impairment Score (mNIS +7) Composite Score Over 18 Months

    Baseline, Months 6, 12, 18

  • Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) Questionnaire Score

    Baseline, Months 6, 12, 18

  • Number of Participants in Each Polyneuropathy Disability (PND) Stage Based on Worst Post-Baseline Score

    Baseline, Months 6, 12, 18

Study Arms (1)

All Patients

EXPERIMENTAL

All patients who received at least 1 dose of revusiran (ALN-TTRSC)

Drug: Revusiran

Interventions

RevusiranDRUG

500mg Revusiran by subcutaneous (sc) injection

Also known as: ALN-TTRSC
All Patients

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of FAP (familial amyloidotic polyneuropathy) with documented TTR mutation
  • Received an orthotopic liver transplant ≥12 months before the date of informed consent
  • An increase in polyneuropathy disability (PND) score post-transplant
  • Polyneuropathy Disability score of ≤3b

You may not qualify if:

  • New York Heart Association (NYHA) classification of \>2
  • Other known causes of sensorimotor or autonomic neuropathy (eg, autoimmune disease)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Clinical Trial SIte

Paris, France

Location

Clinical Trial SIte

Münster, Germany

Location

Clinical Trial SIte

Porto, Portugal

Location

Clinical Trial SIte

Majorca, Spain

Location

Clinical Trial Site

Umeå, Sweden

Location

Clinical Trial SIte

London, United Kingdom

Location

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Interventions

revusiran

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesAmyloid NeuropathiesPeripheral Nervous System DiseasesNeuromuscular DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesAmyloidosis, FamilialMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesAmyloidosisProteostasis Deficiencies

Limitations and Caveats

Dosing of participants was discontinued early in this study and a protocol amendment allowed additional safety follow-up visits. This study completed based on the amendment, but only a limited amount of data was collected in this study.

Results Point of Contact

Title
Chief Medical Office
Organization
Alnylam Pharmaceuticals Inc
Clinicaltrials@alnylam.com
866.330.0326

Study Officials

  • John Vest, MD

    Alnylam Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 30, 2015

First Posted

November 4, 2015

Study Start

October 1, 2015

Primary Completion

February 6, 2017

Study Completion

February 6, 2017

Last Updated

March 28, 2019

Results First Posted

March 28, 2019

Record last verified: 2019-03

Locations

ES(1)SE(1)FR(1)PT(1)GB(1)DE(1)