BridgeMD
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)
EU CTIS ID: 2025-521792-31-01
What this study is testing
To demonstrate reduction of low-density lipoprotein cholesterol (LDL-C) with zodasiran compared with placebo in subjects with HoFH
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study
- 2. Body weight ≥35 kg at Screening as patients could theoretically be <35 kg as the study continues.
- 3. 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)
- 4. LDL-C ≥70 mg/dL (1.8 mmol/L). For adolescents 12 to <18 years of age LDL-C ≥116 mg/dL (3 mmol/L)
- 5. Hemoglobin A1c (HbA1c) ≤9.5%
- 6. Total bilirubin <2xULN, unless in previously confirmed cases of Gilbert's syndrome
You likely can't join if
- 1. 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)
- 2. Use of an antisense oligonucleotide molecule within 3 months before Day 1 (exception: use of inclisiran is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)
- 3. Use of evinacumab within 3 months before Day 1. Evinacumab use is prohibited during the study. In regions where evinacumab is available, the physician must justify and document in source documents and eCRF(s) why a patient is not receiving evinacumab therapy as part of standard of care (eg, safety, tolerability, reimbursement/cost, patient choice, etc.)
- 4. Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses
- 5. Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1
- 6. Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)
See the full eligibility criteria
- 1. Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study
- 2. Body weight ≥35 kg at Screening as patients could theoretically be <35 kg as the study continues.
- 3. 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)
- 4. LDL-C ≥70 mg/dL (1.8 mmol/L). For adolescents 12 to <18 years of age LDL-C ≥116 mg/dL (3 mmol/L)
- 5. Hemoglobin A1c (HbA1c) ≤9.5%
- 6. Total bilirubin <2xULN, unless in previously confirmed cases of Gilbert's syndrome
- 7. Alanine aminotransferase or aspartate aminotransferase <3×ULN
- 8. On standard of care, maximally tolerated lipid-lowering therapy
- 1. 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)
- 2. Use of an antisense oligonucleotide molecule within 3 months before Day 1 (exception: use of inclisiran is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks)
- 3. Use of evinacumab within 3 months before Day 1. Evinacumab use is prohibited during the study. In regions where evinacumab is available, the physician must justify and document in source documents and eCRF(s) why a patient is not receiving evinacumab therapy as part of standard of care (eg, safety, tolerability, reimbursement/cost, patient choice, etc.)
- 4. Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses
- 5. Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1
- 6. Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)
- 7. Estimated glomerular filtration rate <30 mL/min
The study team makes the final eligibility decision.
Where it's taking place
- New Zealand
- Turkey
- Saudi Arabia
- United States
- Israel
- Japan
- Georgia
- Brazil
- Australia
- South Africa
- Canada
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, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New Zealand; Turkey; Saudi Arabia; United States; Israel; Japan and 5 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 EU CTIS; the study team decides eligibility.