RECRUITING

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

Conditions

Homozygous Familial Hypercholesterolemia

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2025-06-17

Study Completion:2027-08-20

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07037771

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.

Ages Eligible for Study:12 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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

Inclusion Criteria

Exclusion 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

Contacts and Locations

Study Contact

Medical Monitor

CONTACT

626-304-3400

YOSEMITE@arrowheadpharma.com

Study Locations (Sites)

Metabolic and Atherosclerosis Research Center

Cincinnati, Ohio, 45227

United States

Collaborators and Investigators

Sponsor: Arrowhead Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-06-17

Study Completion Date2027-08-20

Study Record Updates

Study Start Date2025-06-17

Study Completion Date2027-08-20

Terms related to this study

Additional Relevant MeSH Terms

Homozygous Familial Hypercholesterolemia