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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 Clinical Study with Patisiran

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To assess the safety and efficacy of long-term dosing with patisiran in transthyretin-mediated amyloidosis (ATTR) patients with familial amyloidotic polyneuropathy (FAP)

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Ethical review
Approved WMO
Status
Completed
Health condition type
Neurological disorders congenital
Study type
Interventional

ID

NL-OMON50597

Source

ToetsingOnline

Brief title

TTR206

Condition

  • Neurological disorders congenital
  • Neurological disorders NEC

Synonym

Familial ATTR, FAP (familial amyloidotic polyneuropathy)

Research involving

Human

Sponsors and support

Primary sponsor :
Alnylam Pharmaceuticals
Source(s) of monetary or material Support :
Alnylam Pharmaceuticals

Intervention

Keyword :
ALN-TTR02, Familial amyloidotic polyneuropathy, patisiran, RNAi therapeutic

Outcome measures

Primary outcome

The objective of this study is to assess the safety and efficacy of long-term

dosing with patisiran in ATTR patients with FAP.

Secondary outcome

None

Background summary

This is a multicenter, open-label extension study designed to evaluate the
long-term safety and efficacy of patisiran in patients with FAP who have
completed a prior study with patisiran.

Study objective

To assess the safety and efficacy of long-term dosing with patisiran in
transthyretin-mediated amyloidosis (ATTR) patients with familial amyloidotic
polyneuropathy (FAP)

Study design

This is a multicenter, multinational, open-label extension study to evaluate
the long-term safety and efficacy of patisiran in patients with FAP who have
previously completed a patisiran study.

Intervention

Patients will receive 0.3 mg/kg patisiran once every 21 days administered as an
IV infusion over 80 minutes (approximately 1ml/min for the first 15 minutes
followed by approximately 3 ml/min for the remaining part of the infusion) by a
controlled infusion device.

Study burden and risks

Patients are required to visit the hospital more often than during standard
treatment. The patient's participation in this study will last for
approximately 5 years. Efficacy and safety testing will be performed. Visits
involve the administration of study medication and standard safety tests

Public
Alnylam Pharmaceuticals

300 Third Street
Cambridge MA 02142
US
Scientific
Alnylam Pharmaceuticals

300 Third Street
Cambridge MA 02142
US

Listed location countries

Netherlands

Age

Adults (18-64 years)
Elderly (65 years and older)

Inclusion criteria

1. Male or female of 18 to 85 years of age (inclusive)
2. Have a diagnosis of FAP with documented TTR mutation
3. Have completed a patisiran study
4. Adequate liver function and renal function

Exclusion criteria

1. Pregnant or nursing
2. Have an active infection requiring systemic antiviral or antimicrobial
therapy
3. Have poorly controlled diabetes mellitus
4. Have uncontrolled clinically significant cardiac arrhythmia or unstable
angina

Design

Study phase :
3
Study type :
Interventional
Masking :
Open (masking not used)
Control :
Uncontrolled
Primary purpose :
Treatment

Recruitment

NL
Recruitment status
:
Completed
Start date (anticipated) :
Enrollment :
3
Type :
Actual

Approved WMO
Date :
Application type :
First submission
Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
Date :
Application type :
First submission
Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
Date :
Application type :
Amendment
Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
Date :
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Amendment
Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
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Approved WMO
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Approved WMO
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
Date :
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
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Approved WMO
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Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
Date :
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Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
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Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
Date :
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Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
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CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
Date :
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Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)
Approved WMO
Date :
Application type :
Amendment
Review commission :
CCMO: Centrale Commissie Mensgebonden Onderzoek (Den Haag)

Followed up by the following (possibly more current) registration

No registrations found.

Other (possibly less up-to-date) registrations in this register

No registrations found.

In other registers

Register ID
EudraCT EUCTR2014-003877-40-NL
ClinicalTrials.gov NCT01960348
CCMO NL56671.000.16

Date completed :
Results posted :
Actual enrolment :
2

First publication