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

ConditionHomozygous Familial Hypercholesterolemia
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age12+
SponsorArrowhead Pharmaceuticals

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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). For adolescents 12 to <18 years of age, 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 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)

Use of evinacumab within 3 months before Day 1. Evinacumab use is prohibited during the study.

Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses

Trial design

Design model

Parallel

Treatments tested in this trial

  • zodasiran Injection

    Drug

    ARO-ANG3 Injection

  • Placebo

    Drug

    sterile normal saline (0.9% NaCl)

Treatment groups

60 Participants
are divided into 2 treatment groups
Group A: zodasiranExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 2 interventions

Trial outcomes

Primary outcomes

1

Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)

Time frame
Baseline, Month 12

Secondary outcomes

1

Change and Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB) (Randomized Period)

Time frame
Baseline, Month 12
2

Change and Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C) (Randomized Period)

Time frame
Baseline, Month 12
3

Change from Baseline to Month 12 in Fasting LDL-C (Randomized Period)

Time frame
Baseline, Month 12
4

Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C (Randomized Period)

Time frame
Baseline, Month 12

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Arrowhead Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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