A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
NCT07727538 · Status: RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 12
Last updated 2026-08-26
Summary
The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.
Conditions
- Familial Chylomicronemia Syndrome
Interventions
- DRUG
-
Olezarsen
Olezarsen will be administered by subcutaneous injection.
Sponsors & Collaborators
-
Ionis Pharmaceuticals, Inc.
lead INDUSTRY
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- PARALLEL
Eligibility
- Min Age
- 2 Years
- Max Age
- 17 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-07-31
- Primary Completion
- 2030-08-31
- Completion
- 2031-08-31
- FDA Drug
- Yes
Countries
- United States
Study Locations
More Related Trials
-
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy
NCT00607386 ·Status: COMPLETED ·Phase: PHASE4
-
A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy
NCT02251600 ·Status: COMPLETED ·Phase: PHASE1
-
A Study to Assess the Effectiveness and Safety of Canakinumab in Clinical Use in Patients With Schnitzler's Syndrome
NCT07598422 ·Status: RECRUITING
-
A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1
NCT02312011 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Absorption of Corticosteroids in Children With Juvenile Dermatomyositis
NCT00004357 ·Status: COMPLETED ·Phase: PHASE2
-
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
NCT00258011 ·Status: COMPLETED ·Phase: PHASE3
-
An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy
NCT07573631 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE2
-
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
NCT05422482 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1
-
A Study to Assess the Safety and Pharmacokinetics of Multiple Ascending Subcutaneous Doses of DS-2325a in Healthy Subjects
NCT05583669 ·Status: COMPLETED ·Phase: PHASE1
-
A Study of Galcanezumab in Healthy Participants
NCT02576951 ·Status: COMPLETED ·Phase: PHASE1
-
A 90 Day, Phase 3,Open Labeled Exploratory Study of RELiZORB
NCT03530852 ·Status: RECRUITING ·Phase: NA
-
Treatment of the Cholesterol Defect in Smith-Lemli-Opitz Syndrome
NCT00272844 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
NCT07593391 ·Status: RECRUITING ·Phase: PHASE3
-
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
NCT05025241 ·Status: COMPLETED ·Phase: PHASE2
-
The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients
NCT02044692 ·Status: UNKNOWN
-
Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
NCT03936894 ·Status: UNKNOWN ·Phase: PHASE1/PHASE2
-
An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
NCT06435312 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3
-
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
NCT06181136 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2
-
Study of Idursulfase-beta (GC1111) in Hunter Syndrome
NCT02663024 ·Status: UNKNOWN ·Phase: PHASE2
-
An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
NCT02907619 ·Status: TERMINATED ·Phase: PHASE2
-
Extension Study of NS-089/NCNP-02 in DMD
NCT05135663 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2
-
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
NCT01301898 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Phase I/II Study of SRP-4053 in DMD Patients
NCT02310906 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Rasburicase in Tumor Lysis Syndrome
NCT00302653 ·Status: COMPLETED ·Phase: PHASE4
-
Burosumab for CSHS
NCT03993821 ·Status: COMPLETED ·Phase: EARLY_PHASE1