Back to resultsNEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Primary Purpose
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Eplontersen
Inotersen
Sponsored by
About this trial
This is an interventional treatment trial for Hereditary Transthyretin-Mediated Amyloid Polyneuropathy focused on measuring Polyneuropathy, Amyloidosis, ATTR, TTR
Eligibility Criteria
Inclusion Criteria:
- Aged 18 to 82 years at the time of informed consent
- Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent
- Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non-pregnant female partner must be using a highly effective contraceptive method
Diagnosis of hereditary transthyretin-mediated polyneuropathy as defined by meeting all 3 of the following:
- Stage 1 or Stage 2 Familial Amyloid Polyneuropathy (FAP) or Coutinho Stage
- Documented genetic mutation in the TTR gene
- Symptoms and signs consistent with neuropathy associated with transthyretin amyloidosis, including NIS ≥ 10 and ≤ 130
Exclusion Criteria:
- Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a subject unsuitable for inclusion, including but not limited to abnormal safety labs
- Karnofsky performance status ≤ 50
- Other causes of sensorimotor or autonomic neuropathy (e.g., autoimmune disease), including uncontrolled diabetes
- Prior liver transplant or anticipated liver transplant within 1-yr of Screening
- New York Heart Association (NYHA) functional classification of ≥ 3
- Acute coronary syndrome within 6 months of screening or major surgery within 3 months of Screening
- Other types of amyloidosis
- Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the Study
- Current treatment with any approved drug for hereditary TTR amyloidosis such as Vyndaqel® / Vyndamax™ (tafamidis), Tegsedi™ (inotersen), Onpattro™ (patisiran), off-label use of diflunisal or doxycycline, and tauroursodeoxycholic acid (TUDCA). If previously treated with Vyndaqel® / Vyndamax™, diflunisal or doxycycline, and TUDCA, must have discontinued treatment for at least 2 weeks prior to Study Day 1
- Previous treatment with Tegsedi™ (Inotersen) or Onpattro™ (patisiran), or other oligonucleotide or RNA therapeutic (including siRNA)
Sites / Locations
- Mayo Clinic - Arizona
- Mayo Clinic - Jacksonville
- Indiana University School of Medicine - Indianapolis
- University of Kansas Medical Center
- Johns Hopkins University Neurology Research Office
- Boston University School of Medicine
- Mayo Clinic - Rochester
- The Neurological Institute of New York
- University of North Carolina Hospitals - Neurology Clinic
- Oregon Health and Science University
- Penn Presbyterian Medical Center
- University of Washington Medical Center
- Hospital Italiano de Buenos Aires
- Hospital El Cruce
- STAT Research
- Instituto Fleni
- Perron Institute for Neurological and Translational Science
- Instituto de Neurologia de Curitiba
- Instituto de Neurologia de Curitiba
- Universidade Estadual de Campinas
- Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
- Hospital Universitário Clementino Fraga Filho
- Associação de Assistência à Criança Deficiente - Unidade Lar Escola
- Toronto General Hospital
- The Cyprus Institute of Neurology and Genetics
- Centre Hospitalier Universitaire de Toulouse
- Hôpital Bicêtre
- Hôpital de la Timone
- Universitätsklinikum Würzburg
- Universitätsklinikum Heidelberg
- University General Hospital of Heraklion (PAGNI)
- Azienda Ospedaliera Universitaria Policlinico Gaetano Martino
- Fondazione IRCCS Istituto Neurologico Carlo Besta
- Fondazione IRCCS Policlinico San Matteo
- Fondazione Policlinico Universitario Agostino Gemelli
- Auckland City Hospital
- Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria
- Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio
- Hospital Son Llàtzer
- Hospital Son Llàtzer
- Hospital Clínico San Carlos
- Norrlands Universitetssjukhus
- Chang Gung Memorial Hospital - Linkou Branch
- China Medical University Hospital
- Taipei Veterans General Hospital
- National Taiwan University Hospital
- İstanbul Üniversitesi - Istanbul Tıp Fakültesi
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
Eplontersen
Inotersen
Arm Description
Eplontersen by subcutaneous injection once every 4 weeks.
Inotersen by subcutaneous injection once weekly through week 34. Participants will then convert to Eplontersen administered subcutaneously once every 4 weeks until the end of study.
Outcomes
Primary Outcome Measures
Change from baseline in mNIS+7 at Week 66
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 score indicates lower function.
Change from baseline in the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66
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.
Percent change from baseline in serum TTR concentration at Week 66
Percent change from baseline in serum transthyretin (TTR) concentration at Week 35
Change from baseline in modified neuropathy impairment score plus 7 (mNIS+7) at Week 35
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 score indicates lower function.
Secondary Outcome Measures
Change from baseline in Norfolk QOL-DN at Week 35
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 (NSC) score at Weeks 35 and 66
NSC score is a questionnaire composed of 38 questions that assess the presence and severity of these neuropathy symptoms (including weakness, loss of temperature and pain sensation, and manifestations associated with autonomic nervous system dysfunction).
Change from baseline in the Physical Component Summary (PCS) score of the 36-Item Short Form Survey (SF-36) at Week 65
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 (PND) score at Week 65
The PND is a 6-stage scoring system: 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) at Week 65
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).
Full Information
NCT ID
NCT04136184
First Posted
October 21, 2019
Last Updated
August 21, 2023
Sponsor
Ionis Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT04136184
Brief Title
NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Official Title
A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
January 15, 2020 (Actual)
Primary Completion Date
April 11, 2023 (Actual)
Study Completion Date
July 12, 2023 (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
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No
5. Study Description
Brief Summary
To evaluate the efficacy and safety of eplontersen after administration for 65 weeks to patients with hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN), as compared to the NEURO-TTR trial (NCT01737398). For more information, please visit http://www.neuro-ttransform.com/.
Detailed Description
This is a multicenter, open-label study in up to 140 participants, who will be randomized to receive subcutaneous (SC) injections of either eplontersen once every 4 weeks or inotersen once a week. Participants will also receive daily supplemental doses of the recommended daily allowance of vitamin A. Participants included in the inotersen reference arm will be crossed over to eplontersen at Week 37 after completing the Week 35 assessments.
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
Polyneuropathy, Amyloidosis, ATTR, TTR
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
168 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Eplontersen
Arm Type
Experimental
Arm Description
Eplontersen by subcutaneous injection once every 4 weeks.
Arm Title
Inotersen
Arm Type
Active Comparator
Arm Description
Inotersen by subcutaneous injection once weekly through week 34. Participants will then convert to Eplontersen administered subcutaneously once every 4 weeks until the end of study.
Intervention Type
Drug
Intervention Name(s)
Eplontersen
Other Intervention Name(s)
ION-682884, AKCEA-TTR-LRx, IONIS-TTR-LRx
Intervention Description
Eplontersen by subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
Inotersen
Other Intervention Name(s)
TEGSEDI, ISIS 420915
Intervention Description
Inotersen by subcutaneous injection
Primary Outcome Measure Information:
Title
Change from baseline in mNIS+7 at Week 66
Description
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 score indicates lower function.
Time Frame
Baseline, Week 66
Title
Change from baseline in the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire at Week 66
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, Week 66
Title
Percent change from baseline in serum TTR concentration at Week 66
Time Frame
Baseline, Week 66
Title
Percent change from baseline in serum transthyretin (TTR) concentration at Week 35
Time Frame
Baseline, Week 35
Title
Change from baseline in modified neuropathy impairment score plus 7 (mNIS+7) at Week 35
Description
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 score indicates lower function.
Time Frame
Baseline, Week 35
Secondary Outcome Measure Information:
Title
Change from baseline in Norfolk QOL-DN at Week 35
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, Week 35
Title
Change from baseline in Neuropathy Symptom and Change (NSC) score at Weeks 35 and 66
Description
NSC score is a questionnaire composed of 38 questions that assess the presence and severity of these neuropathy symptoms (including weakness, loss of temperature and pain sensation, and manifestations associated with autonomic nervous system dysfunction).
Time Frame
Baseline, Week 35, Week 66
Title
Change from baseline in the Physical Component Summary (PCS) score of the 36-Item Short Form Survey (SF-36) at Week 65
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, Week 65
Title
Change from baseline in Polyneuropathy Disability (PND) score at Week 65
Description
The PND is a 6-stage scoring system: 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, Week 65
Title
Change from baseline in modified body mass index (mBMI) at Week 65
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, Week 65
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
82 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Aged 18 to 82 years at the time of informed consent
Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal or abstinent
Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non-pregnant female partner must be using a highly effective contraceptive method
Diagnosis of hereditary transthyretin-mediated polyneuropathy as defined by meeting all 3 of the following:
Stage 1 or Stage 2 Familial Amyloid Polyneuropathy (FAP) or Coutinho Stage
Documented genetic mutation in the TTR gene
Symptoms and signs consistent with neuropathy associated with transthyretin amyloidosis, including NIS ≥ 10 and ≤ 130
Exclusion Criteria:
Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a subject unsuitable for inclusion, including but not limited to abnormal safety labs
Karnofsky performance status ≤ 50
Other causes of sensorimotor or autonomic neuropathy (e.g., autoimmune disease), including uncontrolled diabetes
Prior liver transplant or anticipated liver transplant within 1-yr of Screening
New York Heart Association (NYHA) functional classification of ≥ 3
Acute coronary syndrome within 6 months of screening or major surgery within 3 months of Screening
Other types of amyloidosis
Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the Study
Current treatment with any approved drug for hereditary TTR amyloidosis such as Vyndaqel® / Vyndamax™ (tafamidis), Tegsedi™ (inotersen), Onpattro™ (patisiran), off-label use of diflunisal or doxycycline, and tauroursodeoxycholic acid (TUDCA). If previously treated with Vyndaqel® / Vyndamax™, diflunisal or doxycycline, and TUDCA, must have discontinued treatment for at least 2 weeks prior to Study Day 1
Previous treatment with Tegsedi™ (Inotersen) or Onpattro™ (patisiran), or other oligonucleotide or RNA therapeutic (including siRNA)
Facility Information:
Facility Name
Mayo Clinic - Arizona
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
Mayo Clinic - Jacksonville
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
Indiana University School of Medicine - Indianapolis
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Johns Hopkins University Neurology Research Office
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21224
Country
United States
Facility Name
Boston University School of Medicine
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Mayo Clinic - Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
The Neurological Institute of New York
City
New York
State/Province
New York
ZIP/Postal Code
10032-3784
Country
United States
Facility Name
University of North Carolina Hospitals - Neurology Clinic
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Facility Name
Oregon Health and 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
University of Washington Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Hospital Italiano de Buenos Aires
City
Ciudad Autónoma De Buenos Aires
State/Province
Buenos Aires
ZIP/Postal Code
C1199ABB
Country
Argentina
Facility Name
Hospital El Cruce
City
Florencio Varela
State/Province
Buenos Aires
ZIP/Postal Code
1888
Country
Argentina
Facility Name
STAT Research
City
Buenos Aires
ZIP/Postal Code
C1023AAB
Country
Argentina
Facility Name
Instituto Fleni
City
Buenos Aires
ZIP/Postal Code
C1428 AQK
Country
Argentina
Facility Name
Perron Institute for Neurological and Translational Science
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
Instituto de Neurologia de Curitiba
City
Curitiba
State/Province
Parana
ZIP/Postal Code
81210-310
Country
Brazil
Facility Name
Instituto de Neurologia de Curitiba
City
Curitiba
State/Province
Paraná
ZIP/Postal Code
81210-310
Country
Brazil
Facility Name
Universidade Estadual de Campinas
City
Campinas
ZIP/Postal Code
13083-970
Country
Brazil
Facility Name
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
City
Ribeirão Preto
ZIP/Postal Code
14049-900
Country
Brazil
Facility Name
Hospital Universitário Clementino Fraga Filho
City
Rio De Janeiro
ZIP/Postal Code
21941-617
Country
Brazil
Facility Name
Associação de Assistência à Criança Deficiente - Unidade Lar Escola
City
São Paulo
ZIP/Postal Code
04032-060
Country
Brazil
Facility Name
Toronto General Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M4C 3E7
Country
Canada
Facility Name
The Cyprus Institute of Neurology and Genetics
City
Egkomi
ZIP/Postal Code
2371
Country
Cyprus
Facility Name
Centre Hospitalier Universitaire de Toulouse
City
Toulouse
State/Province
Haute-Garonne
ZIP/Postal Code
31059
Country
France
Facility Name
Hôpital Bicêtre
City
Le Kremlin-Bicêtre
State/Province
Ile-De-France
ZIP/Postal Code
94270
Country
France
Facility Name
Hôpital de la Timone
City
Marseille
ZIP/Postal Code
13005
Country
France
Facility Name
Universitätsklinikum Würzburg
City
Würzburg
State/Province
Bayern
ZIP/Postal Code
97080
Country
Germany
Facility Name
Universitätsklinikum Heidelberg
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
University General Hospital of Heraklion (PAGNI)
City
Heraklion
State/Province
Crete
ZIP/Postal Code
711 10
Country
Greece
Facility Name
Azienda Ospedaliera Universitaria Policlinico Gaetano Martino
City
Messina
ZIP/Postal Code
98124
Country
Italy
Facility Name
Fondazione IRCCS Istituto Neurologico Carlo Besta
City
Milano
ZIP/Postal Code
20133
Country
Italy
Facility Name
Fondazione IRCCS Policlinico San Matteo
City
Pavia
ZIP/Postal Code
27100
Country
Italy
Facility Name
Fondazione Policlinico Universitario Agostino Gemelli
City
Roma
ZIP/Postal Code
00168
Country
Italy
Facility Name
Auckland City Hospital
City
Grafton
State/Province
Auckland
ZIP/Postal Code
1023
Country
New Zealand
Facility Name
Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria
City
Lisbon
ZIP/Postal Code
1649-028
Country
Portugal
Facility Name
Centro Hospitalar Universitário do Porto - Hospital Geral de Santo Antonio
City
Porto
ZIP/Postal Code
4099-001
Country
Portugal
Facility Name
Hospital Son Llàtzer
City
Palma De Mallorca
State/Province
Illes Balears
ZIP/Postal Code
07198
Country
Spain
Facility Name
Hospital Son Llàtzer
City
Palma
State/Province
Illes Balears
ZIP/Postal Code
07198
Country
Spain
Facility Name
Hospital Clínico San Carlos
City
Madrid
ZIP/Postal Code
28040
Country
Spain
Facility Name
Norrlands Universitetssjukhus
City
Umeå
ZIP/Postal Code
907 37
Country
Sweden
Facility Name
Chang Gung Memorial Hospital - Linkou Branch
City
Taoyuan City
State/Province
Guishan District
ZIP/Postal Code
333
Country
Taiwan
Facility Name
China Medical University Hospital
City
Taichung City
State/Province
Taichung
ZIP/Postal Code
40447
Country
Taiwan
Facility Name
Taipei Veterans General Hospital
City
Taipei City
State/Province
Taipei
ZIP/Postal Code
11217
Country
Taiwan
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
İstanbul Üniversitesi - Istanbul Tıp Fakültesi
City
İstanbul
ZIP/Postal Code
34093
Country
Turkey
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
33638113
Citation
Coelho T, Ando Y, Benson MD, Berk JL, Waddington-Cruz M, Dyck PJ, Gillmore JD, Khella SL, Litchy WJ, Obici L, Monteiro C, Tai LJ, Viney NJ, Buchele G, Brambatti M, Jung SW, St L O'Dea L, Tsimikas S, Schneider E, Geary RS, Monia BP, Gertz M. Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. Neurol Ther. 2021 Jun;10(1):375-389. doi: 10.1007/s40120-021-00235-6. Epub 2021 Feb 26.
Results Reference
derived
Links:
URL
https://ionistrials.com/neuro-ttransform/
Description
The NEURO-TTRansform Study Website
Learn more about this trial
NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
We'll reach out to this number within 24 hrs