Back to resultsA Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Primary Purpose
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Eplontersen
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy focused on measuring ION-682884, hATTR-PN, TTR, EQ-5D-5L, COMPASS-31
Eligibility Criteria
Inclusion Criteria:
- Satisfactory completion of ION-682884-CS3 (NCT04136184) (Index Study) as judged by the Investigator and Sponsor, or diagnosis of hATTR-PN and satisfactory completion of either study ISIS 420915-CS101 or study 2018-P001436 (NCT03702829) (both are Investigator-Sponsored studies with inotersen - the unconjugated version of Eplontersen) as judged by the Investigator and Sponsor.
- Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
Satisfy the following:
Females: must be non-pregnant and non-lactating and either:
- Surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy);
- Post-menopausal (defined as 12 months of spontaneous amenorrhea in females > 55 years of age or, in females ≤ 55 years, 12 months of spontaneous amenorrhea without an alternative medical cause and follicle-stimulating hormone (FSH) levels in the postmenopausal range for the laboratory involved;
- Abstinent*;
- If engaged in sexual relations of child-bearing potential, agree to use highly effective contraceptive methods from the time of signing the informed consent form until at least 24 weeks after the last dose of Eplontersen and agree to receive pregnancy tests per protocol.
- Males: Surgically sterile (i.e., bilateral orchidectomy) or abstinent*, if engaged in sexual relations with a woman of child-bearing potential (WOCBP), the participant or the participant's non-pregnant female partner must use a highly effective contraceptive method from the time of signing the informed consent form until at least 24 weeks after the last dose of Eplontersen. *Abstinence (i.e., refraining from heterosexual intercourse throughout the duration of study participation) is only acceptable as true abstinence, i.e., when this is in line with the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial and withdrawal are not acceptable methods of contraception.
- Willingness to adhere to vitamin A supplementation per protocol.
Exclusion Criteria:
1. Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the participant unsuitable for enrollment or could interfere with the participant taking part in or completing the study.
Sites / Locations
- Mayo ClinicRecruiting
- Indiana University Health University HospitalRecruiting
- Johns Hopkins University Neurology Research OfficeRecruiting
- Boston University School of MedicineRecruiting
- The Neurological Institute of New YorkRecruiting
- University of North Carolina Hospitals - Neurology ClinicRecruiting
- Oregon Health and Science UniversityRecruiting
- University of Washington Medical CenterRecruiting
- Hospital Italiano de Buenos AiresRecruiting
- Instituto FleniRecruiting
- Hospital El CruceRecruiting
- Perron Institute for Neurological and Translational ScienceRecruiting
- Hospital Universitario Clementino Fraga FilhoRecruiting
- Universidade Estadual de CampinasRecruiting
- Instituto de Neurologia de CuritibaRecruiting
- Hospital das Clínicas da Faculdade de Medicina de Ribeirão PretoRecruiting
- Vancouver General HospitalRecruiting
- Toronto General HospitalRecruiting
- The Cyprus Institute of Neurology and GeneticsRecruiting
- Centre Hospitalier Universitaire de ToulouseRecruiting
- Hôpital de la TimoneRecruiting
- Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Fondazione Istituto Neurologico Carlo BestaRecruiting
- Fondazione IRCCS Policlinico San MatteoRecruiting
- Auckland City HospitalRecruiting
- Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa MariaRecruiting
- Centro Hospitalar Universitário do Porto - Hospital Geral de Santo AntonioRecruiting
- Hospital Clínico San CarlosRecruiting
- Hospital Son LlàtzerRecruiting
- Norrlands UniversitetssjukhusRecruiting
- China Medical University HospitalRecruiting
- National Taiwan University HospitalRecruiting
- Taipei Veterans General HospitalRecruiting
- Chang Gung Memorial HospitalRecruiting
- Istanbul Üniversitesi - Istanbul Tip FakültesiRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Eplontersen
Arm Description
Eplontersen will be administered by subcutaneous (SC) injection once every 4 weeks for up to 3 years (157 weeks).
Outcomes
Primary Outcome Measures
Change From Baseline in Platelet Count
Number of Participants With Clinically Significant Changes From Baseline in Renal Function
Change From Baseline in Adverse Events
Change From Baseline in Number of Concomitant Medications Used
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Change From Baseline in Body Weight
Number of Participants With Clinically Significant Changes From Baseline in Physical Examination Findings
Number of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Tests
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
Number of Participants With Clinically Significant Changes From Baseline in Thyroid Panel Tests
Number of Participants With Clinically Significant Changes From Baseline in Coagulation Tests
Number of Participants With Clinically Significant Changes From Baseline in Inflammatory Panel Tests
Number of Participants With Clinically Significant Changes From Baseline in Complement and Immunogenicity Tests
Secondary Outcome Measures
Change From Baseline in Neuropathy Impairment Score (NIS)
NIS is a composite, quantitative measure of both large-and small-fiber dysfunction used to evaluate the participant's muscle strength, sensation, and reflexes. Total NIS is graded on a scale of 0-244, with a higher score indicating greater impairment.
Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Questionnaire
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 score indicates poorer quality of life.
Change From Baseline in Neuropathy Symptom and Change Score (NSC)
NSC score is a questionnaire composed of 38 questions that assess the presence and severity of neuropathy symptoms (including weakness, loss of temperature and pain sensation, and manifestations associated with autonomic nervous system dysfunction).
Change From Baseline in Serum Transthyretin (TTR) Concentration
Change From Baseline in Physical Component Summary Score (PCS) of 36-Item Short Form Survey (SF-36)
The SF-36 is composed of 8 multi-item scales (35 items) assessing physical function (10 items), role limitations due to physical health problems (4 items), bodily pain (2 items), general health (5 items), vitality (4 items), social functioning (2 items), role limitations due to emotional problems (3 items) and emotional well-being (5 items). Each of the 8 scales is scored from 0 to 100 with higher scores indicating better health. The 8 scales can be aggregated into a PCS score, which is also scaled from 0 to 100 with higher scores indicating better health.
Change From Baseline in Polyneuropathy Disability Score (PND)
PND score assesses disease severity using a 5-stage scoring system. It includes Stage 0: no impairment; Stage 1: sensory disturbances but preserved walking capabilities; Stage 2: impaired walking capacity, but ability to walk without a stick or crutches; Stage 3A/B: walking with help of 1 or 2 sticks or crutches; Stage 4: confined to wheel chair or bedridden.
Change From Baseline in Modified Body Mass Index (mBMI)
mBMI is defined as body mass index in kilograms per square meter (kg/m^2) multiplied by serum albumin in grams per liter (g/L).
Change From Baseline in Composite Autonomic Symptom Score-31 (COMPASS-31)
COMPASS-31 is a 31-question participant-reported assessment that measures autonomic symptoms across 6 weighted domains on a 100-point scale: orthostatic intolerance (40 points), vasomotor (5 points), secretomotor (15 points), gastrointestinal (25 points), bladder (10 points), and pupillomotor (15 points). A higher score indicates worse autonomic dysfunction.
Change From Baseline in 5 Level EQ-5D (EQ-5D-5L)
The EQ-5D-5L is a standard measure of health-related quality of life. EQ-5D-5L consists of two components: a health state profile and a visual analog scale (VAS). EQ-5D health state profile comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: 1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems. The 5D-5L systems are converted into a single index utility score between 0 to 1, where a higher score indicates a better health state. The VAS records the participant's health on a 0-100 mm VAS scale, with 0 indicating "the worst health you can imagine" and 100 indicating "the best health you can imagine". Higher scores of EQ VAS indicate better health.
Full Information
NCT ID
NCT05071300
First Posted
September 23, 2021
Last Updated
October 4, 2023
Sponsor
Ionis Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT05071300
Brief Title
A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Official Title
An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 4, 2022 (Actual)
Primary Completion Date
July 2024 (Anticipated)
Study Completion Date
July 2024 (Anticipated)
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
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to evaluate the safety and tolerability of extended dosing with Eplontersen in participants with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN).
Detailed Description
This is a multicenter, open-label, Phase 3 study in up to approximately 140 participants. Eligible participants will receive Eplontersen once every 4 weeks for up to 157 weeks. Participants will also receive daily supplemental doses of the recommended daily allowance (RDA) of vitamin A. This study will consist of the following periods: less than or equal to (≤) 8-week screening period, a 157 weeks treatment period, and a 24-week post-treatment evaluation period.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Keywords
ION-682884, hATTR-PN, TTR, EQ-5D-5L, COMPASS-31
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
140 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Eplontersen
Arm Type
Experimental
Arm Description
Eplontersen will be administered by subcutaneous (SC) injection once every 4 weeks for up to 3 years (157 weeks).
Intervention Type
Drug
Intervention Name(s)
Eplontersen
Other Intervention Name(s)
AKCEA-TTR-LRx, ION-682884, IONIS-TTR-LRx
Intervention Description
Eplontersen will be administered by SC injection.
Primary Outcome Measure Information:
Title
Change From Baseline in Platelet Count
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Renal Function
Time Frame
Baseline to Week 181
Title
Change From Baseline in Adverse Events
Time Frame
Baseline to Week 181
Title
Change From Baseline in Number of Concomitant Medications Used
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Time Frame
Baseline to Week 181
Title
Change From Baseline in Body Weight
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Physical Examination Findings
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Tests
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Thyroid Panel Tests
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Coagulation Tests
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Inflammatory Panel Tests
Time Frame
Baseline to Week 181
Title
Number of Participants With Clinically Significant Changes From Baseline in Complement and Immunogenicity Tests
Time Frame
Baseline to Week 181
Secondary Outcome Measure Information:
Title
Change From Baseline in Neuropathy Impairment Score (NIS)
Description
NIS is a composite, quantitative measure of both large-and small-fiber dysfunction used to evaluate the participant's muscle strength, sensation, and reflexes. Total NIS is graded on a scale of 0-244, with a higher score indicating greater impairment.
Time Frame
Baseline to Week 181
Title
Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Questionnaire
Description
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 score indicates poorer quality of life.
Time Frame
Baseline to Week 181
Title
Change From Baseline in Neuropathy Symptom and Change Score (NSC)
Description
NSC score is a questionnaire composed of 38 questions that assess the presence and severity of neuropathy symptoms (including weakness, loss of temperature and pain sensation, and manifestations associated with autonomic nervous system dysfunction).
Time Frame
Baseline to Week 181
Title
Change From Baseline in Serum Transthyretin (TTR) Concentration
Time Frame
Baseline to Week 181
Title
Change From Baseline in Physical Component Summary Score (PCS) of 36-Item Short Form Survey (SF-36)
Description
The SF-36 is composed of 8 multi-item scales (35 items) assessing physical function (10 items), role limitations due to physical health problems (4 items), bodily pain (2 items), general health (5 items), vitality (4 items), social functioning (2 items), role limitations due to emotional problems (3 items) and emotional well-being (5 items). Each of the 8 scales is scored from 0 to 100 with higher scores indicating better health. The 8 scales can be aggregated into a PCS score, which is also scaled from 0 to 100 with higher scores indicating better health.
Time Frame
Baseline to Week 181
Title
Change From Baseline in Polyneuropathy Disability Score (PND)
Description
PND score assesses disease severity using a 5-stage scoring system. It includes Stage 0: no impairment; Stage 1: sensory disturbances but preserved walking capabilities; Stage 2: impaired walking capacity, but ability to walk without a stick or crutches; Stage 3A/B: walking with help of 1 or 2 sticks or crutches; Stage 4: confined to wheel chair or bedridden.
Time Frame
Baseline to Week 181
Title
Change From Baseline in Modified Body Mass Index (mBMI)
Description
mBMI is defined as body mass index in kilograms per square meter (kg/m^2) multiplied by serum albumin in grams per liter (g/L).
Time Frame
Baseline to Week 181
Title
Change From Baseline in Composite Autonomic Symptom Score-31 (COMPASS-31)
Description
COMPASS-31 is a 31-question participant-reported assessment that measures autonomic symptoms across 6 weighted domains on a 100-point scale: orthostatic intolerance (40 points), vasomotor (5 points), secretomotor (15 points), gastrointestinal (25 points), bladder (10 points), and pupillomotor (15 points). A higher score indicates worse autonomic dysfunction.
Time Frame
Baseline to Week 181
Title
Change From Baseline in 5 Level EQ-5D (EQ-5D-5L)
Description
The EQ-5D-5L is a standard measure of health-related quality of life. EQ-5D-5L consists of two components: a health state profile and a visual analog scale (VAS). EQ-5D health state profile comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: 1=no problems, 2=slight problems, 3=moderate problems, 4=severe problems, and 5=extreme problems. The 5D-5L systems are converted into a single index utility score between 0 to 1, where a higher score indicates a better health state. The VAS records the participant's health on a 0-100 mm VAS scale, with 0 indicating "the worst health you can imagine" and 100 indicating "the best health you can imagine". Higher scores of EQ VAS indicate better health.
Time Frame
Baseline to Week 181
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Satisfactory completion of ION-682884-CS3 (NCT04136184) (Index Study) as judged by the Investigator and Sponsor, or diagnosis of hATTR-PN and satisfactory completion of either study ISIS 420915-CS101 or study 2018-P001436 (NCT03702829) (both are Investigator-Sponsored studies with inotersen - the unconjugated version of Eplontersen) as judged by the Investigator and Sponsor.
Must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
Satisfy the following:
Females: must be non-pregnant and non-lactating and either:
Surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy);
Post-menopausal (defined as 12 months of spontaneous amenorrhea in females > 55 years of age or, in females ≤ 55 years, 12 months of spontaneous amenorrhea without an alternative medical cause and follicle-stimulating hormone (FSH) levels in the postmenopausal range for the laboratory involved;
Abstinent*;
If engaged in sexual relations of child-bearing potential, agree to use highly effective contraceptive methods from the time of signing the informed consent form until at least 24 weeks after the last dose of Eplontersen and agree to receive pregnancy tests per protocol.
Males: Surgically sterile (i.e., bilateral orchidectomy) or abstinent*, if engaged in sexual relations with a woman of child-bearing potential (WOCBP), the participant or the participant's non-pregnant female partner must use a highly effective contraceptive method from the time of signing the informed consent form until at least 24 weeks after the last dose of Eplontersen. *Abstinence (i.e., refraining from heterosexual intercourse throughout the duration of study participation) is only acceptable as true abstinence, i.e., when this is in line with the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial and withdrawal are not acceptable methods of contraception.
Willingness to adhere to vitamin A supplementation per protocol.
Exclusion Criteria:
1. Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the participant unsuitable for enrollment or could interfere with the participant taking part in or completing the study.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ionis Pharmaceuticals
Phone
(844) 483-0646
Email
IonisHATTRPNstudy@clinicaltrialmedia.com
Facility Information:
Facility Name
Mayo Clinic
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Individual Site Status
Recruiting
Facility Name
Indiana University Health University Hospital
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Individual Site Status
Recruiting
Facility Name
Johns Hopkins University Neurology Research Office
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Individual Site Status
Recruiting
Facility Name
Boston University School of Medicine
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Individual Site Status
Recruiting
Facility Name
The Neurological Institute of New York
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Name
University of North Carolina Hospitals - Neurology Clinic
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Individual Site Status
Recruiting
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Washington Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Individual Site Status
Recruiting
Facility Name
Hospital Italiano de Buenos Aires
City
Buenos Aires
ZIP/Postal Code
C1199ABB
Country
Argentina
Individual Site Status
Recruiting
Facility Name
Instituto Fleni
City
Buenos Aires
ZIP/Postal Code
C1428 AQK
Country
Argentina
Individual Site Status
Recruiting
Facility Name
Hospital El Cruce
City
Florencio Varela
ZIP/Postal Code
1888
Country
Argentina
Individual Site Status
Recruiting
Facility Name
Perron Institute for Neurological and Translational Science
City
Murdoch
State/Province
Western Australia
ZIP/Postal Code
6150
Country
Australia
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Clementino Fraga Filho
City
Botafogo
State/Province
Rio De Janeiro
ZIP/Postal Code
22281-100
Country
Brazil
Individual Site Status
Recruiting
Facility Name
Universidade Estadual de Campinas
City
Campinas
ZIP/Postal Code
13083-888
Country
Brazil
Individual Site Status
Recruiting
Facility Name
Instituto de Neurologia de Curitiba
City
Curitiba
ZIP/Postal Code
81210-310
Country
Brazil
Individual Site Status
Recruiting
Facility Name
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
City
São Paulo
ZIP/Postal Code
14051-140
Country
Brazil
Individual Site Status
Recruiting
Facility Name
Vancouver General Hospital
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V5Z 1M9
Country
Canada
Individual Site Status
Recruiting
Facility Name
Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G2C4
Country
Canada
Individual Site Status
Recruiting
Facility Name
The Cyprus Institute of Neurology and Genetics
City
Égkomi
ZIP/Postal Code
2371
Country
Cyprus
Individual Site Status
Recruiting
Facility Name
Centre Hospitalier Universitaire de Toulouse
City
Toulouse
State/Province
Haute-Garonne
ZIP/Postal Code
31059
Country
France
Individual Site Status
Recruiting
Facility Name
Hôpital de la Timone
City
Marseille
ZIP/Postal Code
13385
Country
France
Individual Site Status
Recruiting
Facility Name
Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Fondazione Istituto Neurologico Carlo Besta
City
Milano
ZIP/Postal Code
20133
Country
Italy
Individual Site Status
Recruiting
Facility Name
Fondazione IRCCS Policlinico San Matteo
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Individual Site Status
Recruiting
Facility Name
Auckland City Hospital
City
Grafton
ZIP/Postal Code
1023
Country
New Zealand
Individual Site Status
Recruiting
Facility Name
Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria
City
Lisboa
ZIP/Postal Code
1649-035
Country
Portugal
Individual Site Status
Recruiting
Facility Name
Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio
City
Porto
ZIP/Postal Code
4099-001
Country
Portugal
Individual Site Status
Recruiting
Facility Name
Hospital Clínico San Carlos
City
Madrid
ZIP/Postal Code
28040
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Son Llàtzer
City
Palma De Mallorca
ZIP/Postal Code
07198
Country
Spain
Individual Site Status
Recruiting
Facility Name
Norrlands Universitetssjukhus
City
Umeå
ZIP/Postal Code
901 85
Country
Sweden
Individual Site Status
Recruiting
Facility Name
China Medical University Hospital
City
Taichung
ZIP/Postal Code
40447
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
National Taiwan University Hospital
City
Taipei City
ZIP/Postal Code
100
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Taipei Veterans General Hospital
City
Taipei
ZIP/Postal Code
11217
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Chang Gung Memorial Hospital
City
Taoyuan
ZIP/Postal Code
333
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Istanbul Üniversitesi - Istanbul Tip Fakültesi
City
Istanbul
ZIP/Postal Code
34093
Country
Turkey
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
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