Tests zodasiran injections in teenagers with a rare inherited high cholesterol disease
Tests zodasiran injections in teens with inherited homozygous familial hypercholesterolemia.
Plain-language title generated from the registry record
Registry title: Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Key facts
- Where
- Viborg, DenmarkSee the site
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for teenagers aged 12 to under 18 with homozygous familial hypercholesterolemia (HoFH) whose LDL cholesterol stays high despite their cholesterol-lowering treatment.
- Age
- 12 to under 18 years
- Sex
- All sexes can join.
- Disease stage
- Homozygous familial hypercholesterolemia (HoFH), confirmed by a genetic test or by clinical diagnosis. LDL-C of 116 mg/dL (3 mmol/L) or higher at screening.
- Earlier treatment
- Must be on the highest cholesterol-lowering treatment they can tolerate. Some earlier treatments within a set time before the study cannot be used: a liver-targeted siRNA drug within 365 days (inclisiran is allowed), an antisense oligonucleotide or evinacumab within 3 months, and systemic corticosteroids.
- Biomarker or test result
- A genetic test supporting HoFH, or a clinical diagnosis. LDL-C of 116 mg/dL (3 mmol/L) or higher. HbA1c of 9.5% (80 mmol/mol) or lower. Liver tests within the limits set in the record.
Main reasons for not taking part
- Pregnant or breastfeeding, or planning to become pregnant during the study
- Body weight under 35 kg (77 lb)
- Liver-targeted siRNA drug (other than inclisiran) within 365 days before Day 1
- Antisense oligonucleotide or evinacumab within 3 months before Day 1
- Systemic corticosteroids, unless used as stable replacement therapy for pituitary or adrenal disease
Read by machine on 13 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 17 years old?
Source: NCT07473843, who can take part
Where in Denmark
- Region MidtjyllandViborg, DenmarkTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Arrowhead Pharmaceuticals
- Trial code
- NCT07473843EU 2025-523662-24-00
- Record updated
- 26 October 2026
- Original description
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.Read full original descriptionShow less
Phase 3 Single-Arm Open-Label Study to Evaluate the Efficacy and Safety of Zodasiran in Adolescent Participants (Age 12 to <18 Years) With Homozygous Familial Hypercholesterolemia (SPRUCE)
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.
Inclusion: 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); Screening hemoglobin A1c (HbA1c) ≤9.5%; Total bilirubin <2×upper limit of normal (ULN), unless in previously confirmed cases of Gilbert's syndrome; Alanine aminotransferase or aspartate aminotransferase <3×ULN.
Exclusion: 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); NOTE: Additional inclusion/exclusion criteria may apply per protocol.