Severe Familial Hypercholesterolemia Clinical Trial
— TAUSSIGOfficial title:
A Multicenter, Open-label Study to Assess the Long-term Safety, Tolerability, and Efficacy of Evolocumab (AMG145) on LDL-C in Subjects With Severe Familial Hypercholesterolemia
| Verified date | May 2024 |
| Source | Amgen |
| Contact | n/a |
| Is FDA regulated | No |
| Health authority | |
| Study type | Interventional |
A study to assess the long term safety and tolerability of evolocumab (AMG 145) in adolescents and adults with severe familial hypercholesterolemia.
| Status | Completed |
| Enrollment | 300 |
| Est. completion date | May 11, 2018 |
| Est. primary completion date | May 11, 2018 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 12 Years to 80 Years |
| Eligibility | Inclusion Criteria: - Participated in Study 20110233 (NCT01588496) or another qualifying evolocumab parent protocol and have a diagnosis of familial hypercholesterolemia. OR - Have a diagnosis of familial hypercholesterolemia AND - Males and females = 12 to = 80 years of age - Stable low-fat diet and lipid-lowering therapies for at least 4 weeks - Low-density lipoprotein cholesterol (LDL-C) >= 130 mg/dl (3.4 mmol/L) for subjects without diagnosed coronary heart disease (CHD)/CHD risk equivalent OR LDL-C >= 100 mg/dl (2.6 mmol/L) for subjects with diagnosed CHD or CHD risk equivalent OR apheresis patients have no LDL-C entry requirement - Fasting triglycerides = 400 mg/dL(4.5 mmol/L) - Body weight of > 40 kg or greater at screening for subjects less than 18 years of age Exclusion Criteria: - New York Heart Failure Association (NYHA) class III or IV or last known left ventricular ejection fraction < 30% - Myocardial infarction, unstable angina, percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG) or stroke within 3 months of screening - Planned cardiac surgery or revascularization - Uncontrolled cardiac arrhythmia - Uncontrolled hypertension |
| Country | Name | City | State |
|---|---|---|---|
| Australia | Research Site | Hobart | Tasmania |
| Australia | Research Site | Perth | Western Australia |
| Belgium | Research Site | Bruxelles | |
| Belgium | Research Site | La Louvière | |
| Brazil | Research Site | Sao Paulo | São Paulo |
| Brazil | Research Site | São Paulo | |
| Canada | Research Site | Chicoutimi | Quebec |
| Canada | Research Site | London | Ontario |
| Canada | Research Site | Montreal | Quebec |
| Canada | Research Site | Montreal | Quebec |
| Canada | Research Site | Quebec | |
| Czechia | Research Site | Brno | |
| Czechia | Research Site | Hradec Kralove | |
| Czechia | Research Site | Olomouc | |
| Czechia | Research Site | Praha 2 | |
| Czechia | Research Site | Uherske Hradiste | |
| France | Research Site | Dijon | |
| France | Research Site | Paris Cedex 13 | |
| Greece | Research Site | Athens | |
| Hong Kong | Research Site | New Territories | |
| Israel | Research Site | Ramat Gan | |
| Italy | Research Site | Cinisello Balsamo (MI) | |
| Italy | Research Site | Napoli | |
| Italy | Research Site | Pisa | |
| Japan | Research Site | Kanazawa | Ishikawa |
| Japan | Research Site | Suita | Osaka |
| Lebanon | Research Site | Beirut | |
| Netherlands | Research Site | Amsterdam | |
| Netherlands | Research Site | Rotterdam | |
| New Zealand | Research Site | Christchurch | |
| South Africa | Research Site | Johannesburg | Gauteng |
| South Africa | Research Site | Observatory | Western Cape |
| Spain | Research Site | A Coruña | Galicia |
| Spain | Research Site | Barcelona | Cataluña |
| Spain | Research Site | Cordoba | Andalucía |
| Spain | Research Site | Lugo | Galicia |
| Spain | Research Site | Madrid | |
| United Kingdom | Research Site | Manchester | |
| United States | Research Site | Cincinnati | Ohio |
| United States | Research Site | Los Angeles | California |
| United States | Research Site | Nashville | Tennessee |
| United States | Research Site | New York | New York |
| United States | Research Site | New York | New York |
| United States | Research Site | New York | New York |
| Lead Sponsor | Collaborator |
|---|---|
| Amgen |
United States, Australia, Belgium, Brazil, Canada, Czechia, France, Greece, Hong Kong, Israel, Italy, Japan, Lebanon, Netherlands, New Zealand, South Africa, Spain, United Kingdom,
Kasichayanula S, Grover A, Emery MG, Gibbs MA, Somaratne R, Wasserman SM, Gibbs JP. Clinical Pharmacokinetics and Pharmacodynamics of Evolocumab, a PCSK9 Inhibitor. Clin Pharmacokinet. 2018 Jul;57(7):769-779. doi: 10.1007/s40262-017-0620-7. — View Citation
Raal FJ, Hegele RA, Ruzza A, Lopez JAG, Bhatia AK, Wu J, Wang H, Gaudet D, Wiegman A, Wang J, Santos RD. Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies. Arterioscler Thromb Vasc Biol. 2024 May;44(5):1156-1164. doi: 10.1161/ATVBAHA.123.320268. Epub 2024 Mar 28. — View Citation
Raal FJ, Hovingh GK, Blom D, Santos RD, Harada-Shiba M, Bruckert E, Couture P, Soran H, Watts GF, Kurtz C, Honarpour N, Tang L, Kasichayanula S, Wasserman SM, Stein EA. Long-term treatment with evolocumab added to conventional drug therapy, with or without apheresis, in patients with homozygous familial hypercholesterolaemia: an interim subset analysis of the open-label TAUSSIG study. Lancet Diabetes Endocrinol. 2017 Apr;5(4):280-290. doi: 10.1016/S2213-8587(17)30044-X. Epub 2017 Feb 16. — View Citation
Santos RD, Stein EA, Hovingh GK, Blom DJ, Soran H, Watts GF, Lopez JAG, Bray S, Kurtz CE, Hamer AW, Raal FJ. Long-Term Evolocumab in Patients With Familial Hypercholesterolemia. J Am Coll Cardiol. 2020 Feb 18;75(6):565-574. doi: 10.1016/j.jacc.2019.12.020. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Number of Participants With Adverse Events | The severity of each adverse event (AE) was graded according to the National Cancer Institute Common Terminology Criteria for AEs (NCI-CTCAE) grading scale, where grade 1 = mild AE, grade 2 = moderate AE, grade 3 = severe AE, grade 4 = life-threatening AE and grade 5 = death due to AE. | From first dose of study drug in Study 20110271 up to 30 days after the last dose or until the end of study date, whichever was earlier; median duration of treatment was 48.7 months. | |
| Secondary | Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) | Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216 | ||
| Secondary | Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) | Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216 | ||
| Secondary | Percent Change From Baseline in Lipoprotein (a) | Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216 | ||
| Secondary | Percent Change From Baseline in Apolipoprotein B | Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216 | ||
| Secondary | Percent Change From Baseline in Total Cholesterol/HDL-C Ratio | Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216 | ||
| Secondary | Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio | Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216 | ||
| Secondary | Percentage of Participants With a 15% or Greater Reduction in LDL-C | Baseline and weeks 4, 6, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, 180, 192, 204, and 216 |