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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05071300




Registration number
NCT05071300
Ethics application status
Date submitted
23/09/2021
Date registered
8/10/2021

Titles & IDs
Public 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
Scientific 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
Secondary ID [1] 0 0
2024-511201-32-00
Secondary ID [2] 0 0
ION-682884-CS13
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Transthyretin-Mediated Amyloid Polyneuropathy 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Eplontersen

Experimental: Eplontersen - Eplontersen will be administered by subcutaneous (SC) injection once every 4 weeks for up to 3 years (157 weeks).


Treatment: Drugs: Eplontersen
Eplontersen will be administered by SC injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Platelet Count
Timepoint [1] 0 0
Baseline to Week 181
Primary outcome [2] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Renal Function
Timepoint [2] 0 0
Baseline to Week 181
Primary outcome [3] 0 0
Number of Participants with Clinically Significant Changes from Baseline in Transaminases
Timepoint [3] 0 0
Baseline to Week 181
Primary outcome [4] 0 0
Change From Baseline in Adverse Events
Timepoint [4] 0 0
Baseline to Week 181
Primary outcome [5] 0 0
Change From Baseline in Number of Concomitant Medications Used
Timepoint [5] 0 0
Baseline to Week 181
Primary outcome [6] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Timepoint [6] 0 0
Baseline to Week 181
Primary outcome [7] 0 0
Change From Baseline in Body Weight
Timepoint [7] 0 0
Baseline to Week 181
Primary outcome [8] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Physical Examination Findings
Timepoint [8] 0 0
Baseline to Week 181
Primary outcome [9] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Tests
Timepoint [9] 0 0
Baseline to Week 181
Primary outcome [10] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
Timepoint [10] 0 0
Baseline to Week 181
Primary outcome [11] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Thyroid Panel Tests
Timepoint [11] 0 0
Baseline to Week 181
Primary outcome [12] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Coagulation Tests
Timepoint [12] 0 0
Baseline to Week 181
Primary outcome [13] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Inflammatory Panel Tests
Timepoint [13] 0 0
Baseline to Week 181
Primary outcome [14] 0 0
Number of Participants With Clinically Significant Changes From Baseline in Complement and Immunogenicity Tests
Timepoint [14] 0 0
Baseline to Week 181
Secondary outcome [1] 0 0
Change From Baseline in Neuropathy Impairment Score (NIS)
Timepoint [1] 0 0
Baseline to Week 181
Secondary outcome [2] 0 0
Change From Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) Questionnaire
Timepoint [2] 0 0
Baseline to Week 181
Secondary outcome [3] 0 0
Change From Baseline in Neuropathy Symptom and Change Score (NSC)
Timepoint [3] 0 0
Baseline to Week 181
Secondary outcome [4] 0 0
Change From Baseline in Serum Transthyretin (TTR) Concentration
Timepoint [4] 0 0
Baseline to Week 181
Secondary outcome [5] 0 0
Change From Baseline in Physical Component Summary Score (PCS) of 36-Item Short Form Survey (SF-36)
Timepoint [5] 0 0
Baseline to Week 181
Secondary outcome [6] 0 0
Change From Baseline in Polyneuropathy Disability Score (PND)
Timepoint [6] 0 0
Baseline to Week 181
Secondary outcome [7] 0 0
Change From Baseline in Modified Body Mass Index (mBMI)
Timepoint [7] 0 0
Baseline to Week 181
Secondary outcome [8] 0 0
Change From Baseline in Composite Autonomic Symptom Score-31 (COMPASS-31)
Timepoint [8] 0 0
Baseline to Week 181
Secondary outcome [9] 0 0
Change From Baseline in 5 Level EQ-5D (EQ-5D-5L)
Timepoint [9] 0 0
Baseline to Week 181

Eligibility
Key inclusion criteria
1. 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.
2. 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.
3. Satisfy the following:

1. 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.
2. 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.
4. Willingness to adhere to vitamin A supplementation per protocol.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
4/01/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2029
Actual
Sample size
Target
Accrual to date
Final
151
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Perron Institute for Neurological and Translational Science - Murdoch
Recruitment postcode(s) [1] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Indiana
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Oregon
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Argentina
State/province [9] 0 0
Buenos Aires
Country [10] 0 0
Argentina
State/province [10] 0 0
Florencio Varela
Country [11] 0 0
Brazil
State/province [11] 0 0
Rio De Janeiro
Country [12] 0 0
Brazil
State/province [12] 0 0
Campinas
Country [13] 0 0
Brazil
State/province [13] 0 0
Curitiba
Country [14] 0 0
Brazil
State/province [14] 0 0
São Paulo
Country [15] 0 0
Canada
State/province [15] 0 0
British Columbia
Country [16] 0 0
Canada
State/province [16] 0 0
Ontario
Country [17] 0 0
Cyprus
State/province [17] 0 0
Égkomi
Country [18] 0 0
France
State/province [18] 0 0
Haute-Garonne
Country [19] 0 0
France
State/province [19] 0 0
Marseille
Country [20] 0 0
Italy
State/province [20] 0 0
Milano
Country [21] 0 0
Italy
State/province [21] 0 0
Pavia
Country [22] 0 0
Portugal
State/province [22] 0 0
Lisboa
Country [23] 0 0
Portugal
State/province [23] 0 0
Porto
Country [24] 0 0
Spain
State/province [24] 0 0
Madrid
Country [25] 0 0
Spain
State/province [25] 0 0
Palma De Mallorca
Country [26] 0 0
Sweden
State/province [26] 0 0
Umeå
Country [27] 0 0
Taiwan
State/province [27] 0 0
Taichung
Country [28] 0 0
Taiwan
State/province [28] 0 0
Taipei City
Country [29] 0 0
Taiwan
State/province [29] 0 0
Taipei
Country [30] 0 0
Taiwan
State/province [30] 0 0
Taoyuan
Country [31] 0 0
Turkey
State/province [31] 0 0
Istanbul

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ionis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Ionis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
(844) 483-0646
Fax 0 0
Email 0 0
IonisHATTRPNstudy@clinicaltrialmedia.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/ionis/


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05071300