Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients with Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 165

Inclusion Criteria

1. Satisfactory completion of ION-682884-CS3 as judged by the Investigator and Sponsor, OR diagnosis of hATTR-PN and satisfactory completion of study ISIS 420915-CS101 (an Investigator-Sponsored study with inotersen - the unconjugated version of ION-682884) 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:
a. 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 FSH levels in the postmenopausal range for the laboratory involved)
- Abstinent*or
- If engaged in sexual relations of child-bearing potential, agree to use highly effective contraceptive methods (Section 6.4.1) from the time of signing the informed consent form until at least 24 weeks after the last dose of ION-682884 and agree to receive pregnancy tests per protocol
b. Males: Surgically sterile (i.e., bilateral orchidectomy) or abstinent*, if engaged in sexual relations with a woman of child-bearing potential (WOCBP), the patient’s non-pregnant female partner must use a highly effective contraceptive method (Section 6.4.1) from the time of signing the informed consent form until at least 24 weeks after the last dose of ION-682884 *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 patient. 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
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 116
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 52

Exclusion Criteria

1. Have any new condition or worsening of existing condition that in the opinion of the Investigator or Sponsor would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

Related Trials