A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants

Conditions

• Familial Chylomicronemia Syndrome

Eligibility Criteria

Sex

ALL

Age

2 Years - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Olezarsen — DRUG
  Olezarsen will be administered by subcutaneous injection.

Study Details

The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.

Key Dates

First listed

Jul 27, 2026

Start date

Jul 31, 2026

Status verified

Jul 2026

Primary completion

Aug 31, 2030

Completion

Aug 31, 2031

Study Design

Enrollment

12 participants (estimated)

Allocation

NA

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1
  Participants aged 12 to \<18 years will receive multiple doses of olezarsen, at a dose level that depends on body weight, once every month by subcutaneous injection for up to 1 year. All participants may continue to the optional 1-year long-term extension period with the last dose received being after 2 years of treatment.
• Experimental: Cohort 2
  Participants aged 2 to \<12 years will receive multiple doses of olezarsen, at a dose level based on information obtained from Cohort 1, once every month by subcutaneous injection for up to 1 year. All participants may continue to the optional 1-year long-term extension period with the last dose received being after 2 years of treatment.

Primary Outcome Measure

Percent Change from Baseline in Fasting Triglycerides (TG) [ Time Frame: At 6 Months ]

Central Contacts

• Ionis Pharmaceuticals, Inc.
  (844) 200-6410
  [email protected]

Locations (3)

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