RECRUITING

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia

Description

This multicenter, randomized, placebo-controlled study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH). After completion of the double blind (DB) treatment period subjects will be eligible to continue in the optional open-label extension (OLE) period of the study. All placebo subjects who opt to continue will transition to active drug during the OLE Period.

Study Overview

Study Details

Study overview

This multicenter, randomized, placebo-controlled study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH). After completion of the double blind (DB) treatment period subjects will be eligible to continue in the optional open-label extension (OLE) period of the study. All placebo subjects who opt to continue will transition to active drug during the OLE Period.

Phase 3 Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia

Condition
Homozygous Familial Hypercholesterolemia
Intervention / Treatment

-

Contacts and Locations

Cincinnati

Metabolic and Atherosclerosis Research Center, Cincinnati, Ohio, United States, 45227

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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 \<35 kg as the study continues.
  • * HoFH based on a supportive genetic test or a clinical diagnosis (total cholesterol \>500 mg/dL\[13 mmol/L\] OR treated LDL-C concentration of ≥300 mg/dL \[≥8 mmol/L\] either accompanied by TGs \<300 mg/dL \[3.4 mmol/L\] AND both parents with documented total cholesterol \>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 \<3×ULN
  • * On standard of care, maximally tolerated lipid-lowering therapy
  • * 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

Ages Eligible for Study

12 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Arrowhead Pharmaceuticals,

Study Record Dates

2027-08-20