A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884,… (NCT05071300) | Clinical Trial Compass
Active — Not RecruitingPhase 3
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
United States151 participantsStarted 2022-01-04
Plain-language 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).
Who can participate
Age range18 Years
SexALL
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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:
✓. 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.
What they're measuring
1
Change From Baseline in Platelet Count
Timeframe: Baseline to Week 181
2
Number of Participants With Clinically Significant Changes From Baseline in Renal Function
Timeframe: Baseline to Week 181
3
Number of Participants with Clinically Significant Changes from Baseline in Transaminases
Timeframe: Baseline to Week 181
4
Change From Baseline in Adverse Events
Timeframe: Baseline to Week 181
5
Change From Baseline in Number of Concomitant Medications Used
Timeframe: Baseline to Week 181
6
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Timeframe: Baseline to Week 181
7
Change From Baseline in Body Weight
Timeframe: Baseline to Week 181
8
Number of Participants With Clinically Significant Changes From Baseline in Physical Examination Findings