Homozygous familial hypercholesterolemia
Conditions
Interventions
5 doses of zodasiran or placebo by subcutaneous (sc) injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period).
Sponsors
Medical Monitor
Eligibility
Inclusion criteria
Inclusion criteria: Age=>12 years, non pregnant, non lactating, do not plan to become pregnant during the study Body weight =>35 kg at Screening as patients could theoretically be 500 mg/dL[13 mmol/L] OR treated LDL-C concentration of =>300 mg/dL [=>8 mmol/L] either accompanied by TGs 250 mg/dL [6.5 mmol/L] OR cutaneous or tendinous xanthoma before 10 years of age) LDL-C =>70 mg/dL (1.8 mmol/L) Hemoglobin A1c (HbA1c) <=9.5% Total bilirubin <2xULN, unless in previously confirmed cases of Gilbert's syndrome Alanine aminotransferase or aspartate aminotransferase <3xULN On standard of care, maximally tolerated lipid-lowering therapy
Exclusion criteria
Exclusion criteria: 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 Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1 Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen) Estimated glomerular filtration rate <30 mL/min
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period) [Time Frame: Baseline, Month 12] | — |
Countries
Australia, Austria, Belgium, Brazil, Canada, Czech Republic, France, Georgia, Germany, Greece, Israel, Italy, Japan, Netherlands, New Zealand, Saudi Arabia, South Africa, Spain, Sweden, Turkey, United States
Contacts
Public ContactTomoko Kubota
Medpace Japan K.K.
Outcome results
None listed