Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age12-17
SponsorArrowhead Pharmaceuticals
About this trial
This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \<18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) ≥116 milligrams per deciliter (mg/dL) on maximally tolerated lipid-lowering therapy.
Eligibility criteria
Qualifiers
Adolescents 12 to <18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study
Body weight ≥35 kilograms (kg) at screening
HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening genotype) or clinical diagnosis
Screening LDL-C ≥116 mg/dL (3 mmol/L)
Disqualifiers
Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)
Use of an antisense oligonucleotide molecule within 3 months before Day 1
Use of evinacumab within 3 months before Day 1
Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses
Trial design
Treatments tested in this trial
- Zodasiran
Treatment groups
12 Participants
are divided into 1 treatment groupLocations
Sponsors and collaborators
Source ClinicalTrials.gov