ZODASIRAN for Homozygous familial hypercholesterolemia

Inclusion criteria

• {"criterion_text":"- 1. Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study\n- 2. Body weight ≥35 kg at Screening as patients could theoretically be <35 kg as the study continues.\n- 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)\n- 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)\n- 5. Hemoglobin A1c (HbA1c) ≤9.5%\n- 6. Total bilirubin <2xULN, unless in previously confirmed cases of Gilbert's syndrome\n- 7. Alanine aminotransferase or aspartate aminotransferase <3×ULN\n- 8. On standard of care, maximally tolerated lipid-lowering therapy"}

Exclusion criteria

• {"criterion_text":"- 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)\n- 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)\n- 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.)\n- 4. Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses\n- 5. Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1\n- 6. Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)\n- 7. Estimated glomerular filtration rate <30 mL/min"}

Primary endpoints

• {"endpoint_text":"- Percent Change from Baseline to Month 12 in Fasting Low Density Lipoprotein Cholesterol (LDL-C) (Randomized period)","definition_or_measurement_approach":"Percent change from baseline to Month 12 in fasting LDL-C measured during the randomized period (fasting blood measurement; baseline vs Month 12 comparison)."}

Secondary endpoints

• {"endpoint_text":"- 1. Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB) (Randomized Period)\n- 2. Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C) (Randomized Period)\n- 3. Change from Baseline to Month 12 in Fasting LDL-C (Randomized Period)\n- 4. Area Under the Plasma Concentration Versus the Time Curve (AUC) from Baseline to Month 12 for Fasting LDL-C (Randomized Period)\n- 5. Percent Change from Baseline to Month 12 in Fasting Triglycerides (TGs) (Randomized Period)\n- 6. Percent Change from Baseline to Month 12 in Fasting Angiopoietin-like Protein 3 (ANGPTL3) (Randomized Period)\n- 7. Percent Change from Baseline to Month 12 in Fasting Total Cholesterol (Randomized Period)\n- 8. Percent Change from Baseline to Month 12 in Fasting High-Density Lipoprotein Cholesterol (HDL-C) (Randomized Period)\n- 9. Proportion of Participants who Meet European Union (EU) LDL-C Apheresis Eligibility Criteria (per German Apheresis Working Group) at Month 12 (Randomized Period)\n- 10. Proportion of Participants who Meet United States (US) Apheresis Eligibility Criteria (per National Lipid Association) at Month 12 (Randomized Period)\n- 11. Proportion of Participants with Fasting LDL-C <100 mg/dL (2.6 mmol/L) at Month 12 (Randomized Period)\n- 12. Change from Baseline in Fasting LDL-C Over Time (Randomized Period)\n- 13. Percent Change from Baseline in fasting LDL-C Over Time (Randomized Period)\n- 14. Percent Change from Baseline to Month 12 in Fasting Lipoprotein(a) [Lp(a)] (Randomized Period)","definition_or_measurement_approach":"Endpoints are changes or percent-changes from baseline to Month 12 (or over time) in fasting lipid biomarkers (eg, ApoB, non-HDL-C, LDL-C, TGs, ANGPTL3, total cholesterol, HDL-C, Lp(a)) measured by fasting blood samples; several endpoints are proportions meeting predefined LDL-C thresholds or apheresis eligibility at Month 12. AUC endpoint uses plasma concentration vs time for fasting LDL-C from baseline to Month 12."}

Netherlands

Earliest CTIS Part Ii Submission Date

07-10-2025

Latest Decision Or Authorization Date

28-01-2026

Processing Time Days

113

Number Of Sites

1

Number Of Participants

1

France

Earliest CTIS Part Ii Submission Date

14-01-2026

Latest Decision Or Authorization Date

01-04-2026

Processing Time Days

77

Number Of Sites

1

Number Of Participants

1

Spain

Earliest CTIS Part Ii Submission Date

13-01-2026

Latest Decision Or Authorization Date

27-03-2026

Processing Time Days

73

Number Of Sites

3

Number Of Participants

3

Germany

Earliest CTIS Part Ii Submission Date

16-01-2026

Latest Decision Or Authorization Date

27-03-2026

Processing Time Days

70

Number Of Sites

1

Number Of Participants

1

Czechia

Earliest CTIS Part Ii Submission Date

13-01-2026

Latest Decision Or Authorization Date

27-03-2026

Processing Time Days

73

Number Of Sites

1

Number Of Participants

1

Greece

Earliest CTIS Part Ii Submission Date

07-10-2025

Latest Decision Or Authorization Date

24-04-2026

Processing Time Days

199

Number Of Sites

1

Number Of Participants

1

Sweden

Earliest CTIS Part Ii Submission Date

08-01-2026

Latest Decision Or Authorization Date

31-03-2026

Processing Time Days

82

Number Of Sites

1

Number Of Participants

1

Austria

Earliest CTIS Part Ii Submission Date

14-01-2026

Latest Decision Or Authorization Date

28-04-2026

Processing Time Days

104

Number Of Sites

4

Number Of Participants

4

Belgium

Earliest CTIS Part Ii Submission Date

17-12-2025

Latest Decision Or Authorization Date

17-04-2026

Processing Time Days

121

Number Of Sites

2

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

13-01-2026

Latest Decision Or Authorization Date

28-04-2026

Processing Time Days

105

Number Of Sites

3

Number Of Participants

3

Primary sponsor

Full Name

Arrowhead Pharmaceuticals Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Medpace Ellas Monoprosopi I.K.E.

Responsibilities

sponsorDuties codes:1,12

Name

Medpace Finland Oy

Responsibilities

sponsorDuties codes:1,12,13,2,4,5,6

Name

Keystone Bioanalytical Inc.

Responsibilities

sponsorDuties codes:4

Name

Charles River Laboratories Montreal ULC

Responsibilities

sponsorDuties codes:4

Name

Medidata Solutions Inc.

Responsibilities

sponsorDuties codes:7

Name

Almac Clinical Technologies LLC

Responsibilities

sponsorDuties codes:3

Third parties

• {"country":"Greece","full_name":"Medpace Ellas Monoprosopi I.K.E.","duties_or_roles":"codes:1,12","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Keystone Bioanalytical Inc.","duties_or_roles":"codes:4","organisation_type":"Pharmaceutical company"}
• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"codes:1,12,13,2,4,5,6","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Charles River Laboratories Montreal ULC","duties_or_roles":"codes:4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"codes:7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"codes:3","organisation_type":"Pharmaceutical company"}