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Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Primary Purpose

FAP, Familial Amyloid Polyneuropathy, TTR

Status

Completed

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Inotersen

About this trial

This is an interventional treatment trial for FAP focused on measuring FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Satisfactory completion of dosing & efficacy assessments in ISIS 420915-CS2

Exclusion Criteria:

Any new condition or worsening of existing condition that could make the patient unsuitable for participation, or interfere with the patient participating in and/or completing the study

Sites / Locations

University of California, Irvine
Indiana University School of Medicine
Johns Hopkins University Bayview Medical Center
Boston University School of Medicine - Amyloid Treatment & Research Program
Mayo Clinic
Mount Sinai Medical Center
Columbia University Medical Center - The Neurological Institute
Oregon Health & Science University
Penn Presbyterian Medical Center
FLENI
Federal University of Rio de Janeiro - University Hospital
AACD
CHU Henri Mondor - Department of Neurology
CHU Bicetre Aphp French Referral Center for FAP/Cornamyl Network
UKM; Universitätsklinikum Münster, Klinik für Transplantationsmedizin
Universita Degli Studi Di Messina - Azienda Ospedaliera Universitaria Policlinico "Gaetano Martino"
Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico San Matteo
CHLN - Hospital de Santa Maria
CHP-HGSA, Unidade Clinica de Paramiloidose
Hospital Universitari Vall D' Hebron
Hospital Clinic
University College London - National Amyloidosis Centre

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Previous Placebo-Inotersen 300 mg

Previous Inotersen-Inotersen 300 mg

Arm Description

Participants received subcutaneous (SC) doses of 300 milligrams (mg) inotersen once weekly for up to 260 weeks. Participants who received inotersen-matching placebo in the previous study- ISIS 420915-CS2 (NCT01737398) were included in this group.

Participants received SC doses of 300 mg inotersen once weekly for up to 260 weeks. Participants who received inotersen in the previous study- ISIS 420915-CS2 were included in this group.

Outcomes

Primary Outcome Measures

Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug

An adverse event (AE) is any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. An SAE is any untoward medical occurrence that at any dose that results in death, is life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, leads to a congenital anomaly/birth defect, or is an important medical event. TEAEs considered related to the study drug as assessed by the Investigator are reported.

Percentage of Participants With Change From Baseline in Vital Signs

Vital signs included blood pressure, heart rate, respiratory rate, and temperature. Only categories with at least one participant with event are reported.

Percentage of Participants With Change From Baseline in Weight

As prespecified in the protocol, percentage of participants with change from baseline in weight is reported in 2 categories, decrease of ≥7% from Baseline and increase of ≥7% from Baseline.

Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Test Values

Clinical laboratory tests included the analysis of chemistry, haematology, and urinalysis. Any value outside the normal range will be flagged for the attention of the investigator who will assess whether or not a flagged value is of clinical significance. Only those categories with at least one participant with event are reported. Normal range of creatinine clearance is 110 to 150 mL/min in males and 100 to 130 mL/min in females. Normal urine protein to creatinine (P/C) ratio= <0.2. Normal range for Alanine Aminotransferase (ALT) is 4 to 36 units per liter (U/L). Platelets normal range=140×10^9/L to 400×10^9/L.

Percentage of Participants With Change From Baseline in QT Interval Corrected Using Fridericia's Formula (QTcF) as Determined by Electrocardiogram (ECG)

Normal QTcF at Baseline is defined as ≤450 milliseconds (ms) for males or ≤470 ms for females. Percentage of participants with QT interval outside of normal range are reported.

Percentage of Participants Using Concomitant Medication for Nervous and Cardiovascular System Disorders

A concomitant therapy was any non-protocol-specified drug or substance (including over-the counter medications, herbal medications, and vitamin supplements) administered between signing of informed consent and the final post-treatment visit for treating nervous and cardiovascular system disorders.

Percentage of Participants With Change From Baseline in Ophthalmic Examination as Assessed by Visual Acuity Changes

Percentage of Participants With Change From Baseline in Light Detection Ability Measured by Electroretinography

Secondary Outcome Measures

Change From Baseline in the Modified Neuropathy Impairment Score (mNIS)+7 Composite Score at Weeks 78 and 156

The mNIS+7 composite score is a measure of neurologic impairment that evaluates muscle weakness, sensation, reflexes, nerve conduction, and autonomic function. The mNIS+7 Composite Score has a range of -22.32 to 346.32 and a higher mNIS+7 composite score indicates worsening disease. A positive change from Baseline indicates worsening of polyneuropathy impairments. Mixed Effects Model with Repeated Measures (MMRM) was used for the analysis.

Change From Baseline in the mNIS +7 Component: Heart Rate to Deep Breathing Score at Weeks 78 and 156

Heart rate to deep breathing is a quantitative autonomic test using the CASE IV instrument that measures a participant's change in heart rate after deep breathing. The score of this component ranges from 0 to 3.72 points. Higher scores indicate impairment. MMRM was used for the analysis.

Change From Baseline in the mNIS +7 Component: Nerve Conduction Score at Weeks 78 and 156

The nerve conduction tests are quantitative tests that measure the conduction attributes of preselected nerves. The score range of this component is 0 to 18.6 points. Higher scores indicate impairment. MMRM was used for the analysis.

Change From Baseline in the mNIS +7 Component: Heat-Pain Sensory Score at Weeks 78 and 156

The Heat-Pain Sensory test uses the CASE IV instrument to perform standardized psychophysical measurement to determine pain sensory thresholds in response to heat. The maximum score of this component is 0 to 40 points. Higher scores indicate impairment. MMRM was used for the analysis.

Change From Baseline in the mNIS +7 Component: Touch-Pressure Sensory Score at Weeks 78 and 156

The Touch-Pressure Sensory test uses the CASE IV instrument to perform standardized psychophysical measurement to determine pressure sensory thresholds in response to touch. The score range of this component is 0 to 40 points. Higher scores indicate impairment. MMRM was used for the analysis.

Change From Baseline in the Neuropathy Impairment (NIS) Composite Score at Week 52 of Years 4 and 5

The NIS score is a measure of neurologic impairment. The NIS Score has a range of 0 to 244 and a higher NIS score indicates lower function. A positive change from Baseline indicates worsening. MMRM was used for the analysis.

Change From Baseline in the NIS Component: Cranial Nerves Score at Week 52 of Years 4 and 5

Cranial Nerve assessment involves testing 3rd and 6th nerves and facial, palate, and tongue weakness. The score range for this component is 0 to 40 points. Higher scores indicate worsening. MMRM was used for the analysis.

Change From Baseline in the NIS Component: Muscle Weakness Score at Week 52 of Years 4 and 5

Muscle weakness involves testing 19 movements of muscles. The score range of this component is 0 to 152 points. Higher scores indicate worsening. MMRM was used for the analysis.

Change From Baseline in the NIS Component: Reflexes Score at Week 52 of Years 4 and 5

The Reflexes Score involves testing 5 reflexes to stimuli. The score range of this component is 0 to 20 points. Higher scores indicate worsening. MMRM was used for the analysis.

Change From Baseline in the NIS Component: Sensory Score at Week 52 of Years 4 and 5

The Sensory Score is based on testing an index finger and a big toe each to 4 stimuli. The score of this component ranges from 0 to 32 points. Higher scores indicate impairment. MMRM was used for the analysis.

Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) Questionnaire Total Score at Weeks 78 and 156

The Norfolk QoL-DN score is a measure of physical function/large fiber neuropathy, symptoms, activities of daily living, small fiber neuropathy, and autonomic neuropathy. The Norfolk QoL-DN total score has a range of -4 to 136, and a higher Norfolk QoL-DN score indicates poorer QoL. A positive change from Baseline indicates worsening in the QoL. MMRM was used for the analysis.

Change From Baseline in the Norfolk QoL-DN Physical Functioning/Large Fiber Neuropathy Domain Score

The Norfolk QoL-DN physical functioning/large fiber neuropathy domain score is a sub-score of the total Norfolk QoL-DN Questionnaire. The Norfolk QoL-DN physical function/large fiber neuropathy domain score has a range of -4 to 56, and a higher Norfolk QoL-DN domain score indicates poorer quality of life (QoL). A positive change from Baseline indicates worsening in the QoL. MMRM was used for the analysis.

Change From Baseline in the Modified Body Mass Index (mBMI) at Weeks 78 and 156

BMI=weight (kg)/[height (m)^2]. The mBMI is the BMI multiplied by the serum albumin (g/L).

Change From Baseline in the Body Mass Index (BMI) at Weeks 78 and 156

BMI=weight (kg)/[height (m)^2].

Percentage of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Score

PND score is defined as I = sensory disturbances in limbs without motor impairment; II = difficulty walking without the need of a walking aid; III = one stick or one crutch required for walking; IV = two sticks or two crutches needed. V = wheelchair required or patient confined to bed. The change from Baseline values have been categorized as: improved, not changed, worsened, and unknown. Percentage of participants with changes from Baseline are presented category-wise in this outcome measure. Only categories with at least one participant with event are reported.

Percent Change From Baseline in Global Longitudinal Strain (GLS) by Echocardiogram (ECHO) in the Cardiomyopathy-ECHO (CM-ECHO) Set

GLS by ECHO is a measure of cardiac systolic function.

Percent Change From Baseline in GLS by ECHO in the CS3 ECHO Subgroup

GLS by ECHO is a measure of cardiac systolic function.

Change From Baseline in Transthyretin (TTR) Level

Transthyretin protein concentration in serum was measured.

Change From Baseline in Retinol Binding Protein 4 (RBP4) Level

RBP4 protein concentration in serum was measured.

Ctrough: Trough Plasma Concentration of ISIS 420915

Full Information

NCT ID

NCT02175004

First Posted

June 12, 2014

Last Updated

January 13, 2023

Sponsor

Ionis Pharmaceuticals, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT02175004

Brief Title

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

Official Title

An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (FAP)

Study Type

Interventional

2. Study Status

Record Verification Date

January 2023

Overall Recruitment Status

Completed

Study Start Date

June 26, 2014 (Actual)

Primary Completion Date

September 11, 2020 (Actual)

Study Completion Date

January 7, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Ionis Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

Detailed Description

Familial Amyloid Polyneuropathy (FAP) is a rare, hereditary disease caused by mutations in the transthyretin (TTR) protein. TTR is made by the liver and secreted into the blood. TTR mutations cause it to misfold and deposit in multiple organs causing FAP. IONIS-TTR Rx is an antisense drug that is designed to decrease the amount of mutant and normal TTR made by the liver. It is predicted that decreasing the amount of TTR protein will result in a decrease in the formation of TTR deposits, and thus slow or stop disease progression. This study evaluates the safety and tolerability of extended dosing with IONIS-TTR Rx in patients with Familial Amyloid Polyneuropathy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis

Keywords

FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Non-Randomized

Enrollment

135 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Previous Placebo-Inotersen 300 mg

Arm Type

Experimental

Arm Description

Arm Title

Previous Inotersen-Inotersen 300 mg

Arm Type

Experimental

Arm Description

Participants received SC doses of 300 mg inotersen once weekly for up to 260 weeks. Participants who received inotersen in the previous study- ISIS 420915-CS2 were included in this group.

Intervention Type

Drug

Intervention Name(s)

Inotersen

Other Intervention Name(s)

TEGSEDI, IONIS-TTR Rx, ISIS 420915

Intervention Description

Inotersen SC

Primary Outcome Measure Information:

Title

Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Related to Study Drug

Description

Time Frame

From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)

Title

Percentage of Participants With Change From Baseline in Vital Signs

Description

Vital signs included blood pressure, heart rate, respiratory rate, and temperature. Only categories with at least one participant with event are reported.

Time Frame

From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)

Title

Percentage of Participants With Change From Baseline in Weight

Description

As prespecified in the protocol, percentage of participants with change from baseline in weight is reported in 2 categories, decrease of ≥7% from Baseline and increase of ≥7% from Baseline.

Time Frame

From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)

Title

Percentage of Participants With Clinically Significant Change From Baseline in Laboratory Test Values

Description

Time Frame

From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)

Title

Percentage of Participants With Change From Baseline in QT Interval Corrected Using Fridericia's Formula (QTcF) as Determined by Electrocardiogram (ECG)

Description

Normal QTcF at Baseline is defined as ≤450 milliseconds (ms) for males or ≤470 ms for females. Percentage of participants with QT interval outside of normal range are reported.

Time Frame

From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)

Title

Percentage of Participants Using Concomitant Medication for Nervous and Cardiovascular System Disorders

Description

Time Frame

From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)

Title

Percentage of Participants With Change From Baseline in Ophthalmic Examination as Assessed by Visual Acuity Changes

Time Frame

From first dose of study drug up to 3 months post treatment period of 260 weeks (Up to approximately 272 weeks)

Title

Percentage of Participants With Change From Baseline in Light Detection Ability Measured by Electroretinography

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

Secondary Outcome Measure Information:

Title

Change From Baseline in the Modified Neuropathy Impairment Score (mNIS)+7 Composite Score at Weeks 78 and 156

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

Title

Change From Baseline in the mNIS +7 Component: Heart Rate to Deep Breathing Score at Weeks 78 and 156

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

Title

Change From Baseline in the mNIS +7 Component: Nerve Conduction Score at Weeks 78 and 156

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

Title

Change From Baseline in the mNIS +7 Component: Heat-Pain Sensory Score at Weeks 78 and 156

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

Title

Change From Baseline in the mNIS +7 Component: Touch-Pressure Sensory Score at Weeks 78 and 156

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

Title

Change From Baseline in the Neuropathy Impairment (NIS) Composite Score at Week 52 of Years 4 and 5

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Week 52 of Years 4 and 5

Title

Change From Baseline in the NIS Component: Cranial Nerves Score at Week 52 of Years 4 and 5

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Week 52 of Years 4 and 5

Title

Change From Baseline in the NIS Component: Muscle Weakness Score at Week 52 of Years 4 and 5

Description

Muscle weakness involves testing 19 movements of muscles. The score range of this component is 0 to 152 points. Higher scores indicate worsening. MMRM was used for the analysis.

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Week 52 of Years 4 and 5

Title

Change From Baseline in the NIS Component: Reflexes Score at Week 52 of Years 4 and 5

Description

The Reflexes Score involves testing 5 reflexes to stimuli. The score range of this component is 0 to 20 points. Higher scores indicate worsening. MMRM was used for the analysis.

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Week 52 of Years 4 and 5

Title

Change From Baseline in the NIS Component: Sensory Score at Week 52 of Years 4 and 5

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Week 52 of Years 4 and 5

Title

Change From Baseline in the Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) Questionnaire Total Score at Weeks 78 and 156

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156 and at the end of each subsequent treatment year (Week 52 of Years 4 and 5)

Title

Change From Baseline in the Norfolk QoL-DN Physical Functioning/Large Fiber Neuropathy Domain Score

Description

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156 and at the Week 52 of Year 4

Title

Change From Baseline in the Modified Body Mass Index (mBMI) at Weeks 78 and 156

Description

BMI=weight (kg)/[height (m)^2]. The mBMI is the BMI multiplied by the serum albumin (g/L).

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

Title

Change From Baseline in the Body Mass Index (BMI) at Weeks 78 and 156

Description

BMI=weight (kg)/[height (m)^2].

Time Frame

Baseline, Weeks 78 and 156

Title

Percentage of Participants With Change From Baseline in the Polyneuropathy Disability (PND) Score

Description

Time Frame

Baseline, Weeks 78 and 156 and at the end of each subsequent treatment year (Week 52 of each year)

Title

Percent Change From Baseline in Global Longitudinal Strain (GLS) by Echocardiogram (ECHO) in the Cardiomyopathy-ECHO (CM-ECHO) Set

Description

GLS by ECHO is a measure of cardiac systolic function.

Time Frame

Baseline, Weeks 78 and 156

Title

Percent Change From Baseline in GLS by ECHO in the CS3 ECHO Subgroup

Description

GLS by ECHO is a measure of cardiac systolic function.

Time Frame

Weeks 78 and 156

Title

Change From Baseline in Transthyretin (TTR) Level

Description

Transthyretin protein concentration in serum was measured.

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156

Title

Change From Baseline in Retinol Binding Protein 4 (RBP4) Level

Description

RBP4 protein concentration in serum was measured.

Time Frame

Baseline (Baseline is the Baseline of the Previous Study- Study CS2), Weeks 78 and 156, and at the end of each subsequent treatment year (Week 52 of Years 4 and 5)

Title

Ctrough: Trough Plasma Concentration of ISIS 420915

Time Frame

Pre-dose on Days 1, 43, 85, 120, 176, 267, 358, 449, 540, 631, 722, 813, 904, 995, 1086, 1268; Days 1359 and 1450 of Year 4; Days 1632, 1723 and 1814 of Year 5

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Satisfactory completion of dosing & efficacy assessments in ISIS 420915-CS2 Exclusion Criteria: Any new condition or worsening of existing condition that could make the patient unsuitable for participation, or interfere with the patient participating in and/or completing the study

Facility Information:

Facility Name

University of California, Irvine

City

Orange

State/Province

California

ZIP/Postal Code

92868

Country

United States

Facility Name

Indiana University School of Medicine

City

Indianapolis

State/Province

Indiana

ZIP/Postal Code

46202

Country

United States

Facility Name

Johns Hopkins University Bayview Medical Center

City

Baltimore

State/Province

Maryland

ZIP/Postal Code

21205

Country

United States

Facility Name

Boston University School of Medicine - Amyloid Treatment & Research Program

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02118

Country

United States

Facility Name

Mayo Clinic

City

Rochester

State/Province

Minnesota

ZIP/Postal Code

55905

Country

United States

Facility Name

Mount Sinai Medical Center

City

New York

State/Province

New York

ZIP/Postal Code

10029

Country

United States

Facility Name

Columbia University Medical Center - The Neurological Institute

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

Facility Name

Oregon Health & Science University

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

Penn Presbyterian Medical Center

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

FLENI

City

Buenos Aires

Country

Argentina

Facility Name

Federal University of Rio de Janeiro - University Hospital

City

Rio de Janeiro

ZIP/Postal Code

CEP 21941913

Country

Brazil

Facility Name

AACD

City

Sao Paulo

Country

Brazil

Facility Name

CHU Henri Mondor - Department of Neurology

City

Creteil

ZIP/Postal Code

94000

Country

France

Facility Name

CHU Bicetre Aphp French Referral Center for FAP/Cornamyl Network

City

Le Kremlin Bicetre

ZIP/Postal Code

94275

Country

France

Facility Name

UKM; Universitätsklinikum Münster, Klinik für Transplantationsmedizin

City

Munster

ZIP/Postal Code

48149

Country

Germany

Facility Name

Universita Degli Studi Di Messina - Azienda Ospedaliera Universitaria Policlinico "Gaetano Martino"

City

Messina

State/Province

Sicily

ZIP/Postal Code

98124

Country

Italy

Facility Name

Centro per lo Studio e la Cura delle Amiloidosi Sistemiche - Fondazione IRCCS Policlinico San Matteo

City

Pavia

ZIP/Postal Code

27100

Country

Italy

Facility Name

CHLN - Hospital de Santa Maria

City

Lisbon

ZIP/Postal Code

1649-035

Country

Portugal

Facility Name

CHP-HGSA, Unidade Clinica de Paramiloidose

City

Porto

ZIP/Postal Code

4099-001

Country

Portugal

Facility Name

Hospital Universitari Vall D' Hebron

City

Barcelona

ZIP/Postal Code

08035

Country

Spain

Facility Name

Hospital Clinic

City

Barcelona

ZIP/Postal Code

08036

Country

Spain

Facility Name

University College London - National Amyloidosis Centre

City

London

ZIP/Postal Code

NW3 2PF

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

35908242

Citation

Brannagan TH, Coelho T, Wang AK, Polydefkis MJ, Dyck PJ, Berk JL, Drachman B, Gorevic P, Whelan C, Conceicao I, Plante-Bordeneuve V, Merlini G, Obici L, Plana JMC, Gamez J, Kristen AV, Mazzeo A, Gentile L, Narayana A, Olugemo K, Aquino P, Benson MD, Gertz M; NEURO-T. T. R. Open-Label Extension Investigators. Long-term efficacy and safety of inotersen for hereditary transthyretin amyloidosis: NEURO-TTR open-label extension 3-year update. J Neurol. 2022 Dec;269(12):6416-6427. doi: 10.1007/s00415-022-11276-8. Epub 2022 Jul 31.

Results Reference

derived

PubMed Identifier

35799473

Citation

Karam C, Brown D, Yang M, Done N, Zhu JJ, Greatsinger A, Bozas A, Vera-Llonch M, Signorovitch J. Long-term treatment effects of inotersen on health-related quality of life in patients with hATTR amyloidosis with polyneuropathy: Analysis of the open-label extension of the NEURO-TTR trial. Muscle Nerve. 2022 Oct;66(4):438-446. doi: 10.1002/mus.27675. Epub 2022 Aug 4.

Results Reference

derived

PubMed Identifier

34355354

Citation

Yarlas A, Lovley A, McCausland K, Brown D, Vera-Llonch M, Conceicao I, Karam C, Khella S, Obici L, Waddington-Cruz M. Early Data on Long-term Impact of Inotersen on Quality-of-Life in Patients with Hereditary Transthyretin Amyloidosis Polyneuropathy: Open-Label Extension of NEURO-TTR. Neurol Ther. 2021 Dec;10(2):865-886. doi: 10.1007/s40120-021-00268-x. Epub 2021 Aug 5.

Results Reference

derived

PubMed Identifier

32343462

Citation

Brannagan TH, Wang AK, Coelho T, Waddington Cruz M, Polydefkis MJ, Dyck PJ, Plante-Bordeneuve V, Berk JL, Barroso F, Merlini G, Conceicao I, Hughes SG, Kwoh J, Jung SW, Guthrie S, Pollock M, Benson MD, Gertz M; NEURO-TTR open-label extension investigators. Early data on long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2-year update from the open-label extension of the NEURO-TTR trial. Eur J Neurol. 2020 Aug;27(8):1374-1381. doi: 10.1111/ene.14285. Epub 2020 May 29.

Results Reference

derived

Learn more about this trial

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

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