A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants

Ionis Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Healthy Participants

Treatments: Drug: Olezarsen

Study type: Interventional

Funder types: Industry

Identifiers

NCT05579860

ISIS 678354-CS20

Details and patient eligibility

About

The purpose of the study is to assess the bioequivalence of olezarsen between 2 subcutaneous (SC) formulations [(autoinjector (AI) and vial] at 2 dose levels in healthy adult participants.

Full description

This is a Phase 1, single-dose, randomized, open-label, two-period, crossover study in approximately 100 healthy participants. The study will consist of an up to 28-day Screening Period, two treatment periods of 15 days each - with the second treatment period starting 28-42 days after the first begins - and a Post-Treatment Follow-Up Period lasting 91 days after the second treatment period begins. Eligible participants will be randomized to two dose groups (50 mg or 80 mg olezarsen). Participants will be dosed once at the beginning of each treatment period, one time using a single-use AI and one time using a single-use vial. Within each dose group, approximately half of the participants will be dosed with the AI first, with the other participants being dosed with the vial first.

Enrollment: 104 patients

Sex: All

Ages: 18 to 64 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

1. Must have given written informed consent (signed and dated) and any authorizations required by local law, be able to comply with all study requirements prior to receiving any study drug, and be able to communicate effectively with clinic staff
2. Must be a healthy, adult male or female, 18 to 64 years of age (inclusive), and non-smoking (for at least 6 months prior to first study drug administration)
3. Females must be of non-childbearing potential

Exclusion criteria

1. Have a known history or presence of any clinically significant hepatic (e.g., hepatic impairment), renal/genitourinary (e.g., renal impairment), gastrointestinal, cardiovascular, cerebrovascular, pulmonary, endocrine, immunological, musculoskeletal, neurological, psychiatric, dermatological, or hematological disease or condition unless determined as not clinically significant by the PI/sub-investigator

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: None (Open label)

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104 participants in 2 patient groups

Olezarsen Dose Level 1

Experimental group

Description:

Participants will receive two doses of Dose Level 1 each, using one of the following two sequences: (i) AI on Day 1 of Treatment Period 1, followed by vial on Day 1 of Treatment Period 2; or (ii) vial on Day 1 of Treatment Period 1, followed by AI on Day 1 of Treatment Period 2. A washout period of 28-42 days will be maintained between the 2 treatment periods.

Treatment:

Drug: Olezarsen

Olezarsen Dose Level 2

Experimental group

Description:

Participants will receive two doses of Dose Level 2 each, using one of the following two sequences: (i) AI on Day 1 of Treatment Period 1, followed by vial on Day 1 of Treatment Period 2; or (ii) vial on Day 1 of Treatment Period 1, followed by AI on Day 1 of Treatment Period 2 A washout period of at least 28 days will be maintained between the 2 treatment periods.

Treatment:

Drug: Olezarsen