Health TrialsbyHealthPanel

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

ConditionHomozygous Familial Hypercholesterolemia
Trial statusRecruiting
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

This trial does not accept healthy volunteers

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

Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)

Trial design

Design model

Single group

Treatments tested in this trial

  • Zodasiran

    Drug

    By sc injection

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: ZodasiranExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Percent Change from Baseline to Month 12 in Fasting LDL-C

Time frame
Baseline, Month 12

Secondary outcomes

1

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

Time frame
Baseline, Month 12
2

Change from Baseline to Month 12 in Fasting ApoB

Time frame
Baseline, Month 12
3

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

Time frame
Baseline, Month 12
4

Change from Baseline to Month 12 in Fasting Non-HDL-C

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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