Clinical trial
An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
Name
ION-682884-CS13
Description
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).
Trial arms
Trial start
2022-01-04
Estimated PCD
2024-07-01
Trial end
2024-07-01
Status
Recruiting
Phase
Early phase I
Treatment
Eplontersen
Eplontersen will be administered by SC injection.
Arms:
Eplontersen
Other names:
AKCEA-TTR-LRx, ION-682884, IONIS-TTR-LRx
Size
140
Primary endpoint
Change From Baseline in Platelet Count
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Renal Function
Baseline to Week 181
Change From Baseline in Adverse Events
Baseline to Week 181
Change From Baseline in Number of Concomitant Medications Used
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Vital Signs
Baseline to Week 181
Change From Baseline in Body Weight
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Physical Examination Findings
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Tests
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Electrocardiogram (ECG) Parameters
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Thyroid Panel Tests
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Coagulation Tests
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Inflammatory Panel Tests
Baseline to Week 181
Number of Participants With Clinically Significant Changes From Baseline in Complement and Immunogenicity Tests
Baseline to Week 181
Eligibility criteria
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.
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 140, 'type': 'ESTIMATED'}}
Updated at
2023-10-06
1 organization
1 product
1 indication
Organization
Ionis PharmaceuticalsProduct
Eplontersen