Health TrialsbyHealthPanel

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

ConditionHomozygous Familial Hypercholesterolemia
Trial statusNot yet recruiting
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

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Zodasiran

    Drug

    By sc injection

Treatment groups

12 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

Locations

This trial has no locations.

Sponsors and contacts

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

Arrowhead Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: