BridgeMD
New treatment option for Homozygous Familial Hypercholesterolemia
Official title A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
ClinicalTrials.gov ID: NCT07037771
What this study is testing
What is zodasiran Injection?
zodasiran Injection is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for homozygous familial hypercholesterolemia.
Also referred to as ARO-ANG3.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- 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...
- 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).
- Hemoglobin A1c (HbA1c) ≤9.5%
You likely can't join if
- Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug...
- Use of an antisense oligonucleotide molecule within 3 months before Day 1 (except inclisiran, which is permitted; administration of inclisiran and...
- 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
- 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)
See the full eligibility 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 \ 250 mg/dL [6.5 mmol/L] OR...
- 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).
- 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 to include a maximally tolerated statin, ezetimibe, and a PCSK9 inhibitor
- 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
- 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 NOTE: Additional Inclusion/exclusion criteria may apply per protocol
The study team makes the final eligibility decision.
Where it's taking place
- Park Ridge, Illinois, United States
- New York, New York, United States
- Cincinnati, Ohio, United States
- Pittsburgh, Pennsylvania, United States
- Camperdown, New South Wales, Australia
- Saint Leonards, New South Wales, Australia
- Heidelberg, Victoria, Australia
- Nedlands, Western Australia, Australia
- Innsbruck, Austria
- Linz, Austria
- Vienna, Austria
- La Louvière, Belgium
- Leuven, Belgium
- Cerqueira César, São Paulo, Brazil
- Vancouver, British Columbia, Canada
- Chicoutimi, Quebec, Canada
- Québec, Canada
- Hradec Králové, Czechia
- Paris, France
- Tbilisi, Georgia
+ 20 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Park Ridge, Illinois, United States; New York, New York, United States; Cincinnati, Ohio, United States; Pittsburgh, Pennsylvania, United States; Camperdown, New South Wales, Australia; Saint Leonards, New South Wales, Australia and 34 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.