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Trial Assessing Long Term USe of PCSK9 Inhibition in Subjects With Genetic LDL Disorders (TAUSSIG)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01624142
Recruitment Status : Completed
First Posted : June 20, 2012
Results First Posted : January 28, 2019
Last Update Posted : January 28, 2019
Sponsor:
Information provided by (Responsible Party):
Amgen

Brief Summary:
A study to assess the long term safety and tolerability of evolocumab (AMG 145) in adolescents and adults with severe familial hypercholesterolemia.

Condition or disease Intervention/treatment Phase
Severe Familial Hypercholesterolemia Biological: Evolocumab Phase 2 Phase 3

Detailed Description:

This phase 2/3 open-label extension study was designed to characterize the safety and tolerability of long-term administration of evolocumab to adults and adolescents with severe FH (HoFH or non-HoFH severe FH). Participants not on lipid apheresis at enrollment or within the prior 8 weeks initiated treatment with evolocumab 420 mg once monthly (QM). Participants on lipid apheresis at enrollment initiated treatment with evolocumab 420 mg once every 2 weeks (Q2W). Dose frequency changes (420 mg QM vs 420 mg Q2W) were permitted at week 12, 24, or other visits with Sponsor approval. Participants with < 5% LDL-C reduction from baseline and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) < 100 ng/mL could discontinue evolocumab. If serum unbound PCSK9 was ≥ 100 ng/mL with QM dosing, the participant could switch to evolocumab 420 mg Q2W treatment. Participants on apheresis with ≥ 5% LDL-C reduction from baseline and serum unbound PCSK9 < 100 ng/mL with Q2W treatment could switch to QM dosing.

Participants were to continue to receive open-label evolocumab for up to 5 years or until evolocumab became commercially available in the relevant patient population, whichever occurred first.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 300 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : June 1, 2012
Actual Primary Completion Date : May 11, 2018
Actual Study Completion Date : May 11, 2018

Resource links provided by the National Library of Medicine

Drug Information available for: Evolocumab

Arm Intervention/treatment
Experimental: Evolocumab
Participants received 420 mg evolocumab every month (participants not on lipid apheresis) or every 2 weeks (participants on lipid apheresis) for up to 5 years. Participants could switch dosing regimens at week 12 or 24 based on LDL-C and serum unbound proprotein convertase subtilisin/kexin type 9 (PCSK9) levels.
Biological: Evolocumab
Evolocumab was administered by subcutaneous injection either once a month (QM) or once every two weeks (Q2W).
Other Names:
  • AMG 145
  • Repatha




Primary Outcome Measures :
  1. Number of Participants With Adverse Events [ Time Frame: 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. ]
    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.


Secondary Outcome Measures :
  1. Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) [ Time Frame: 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 ]
  2. Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) [ Time Frame: 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 ]
  3. Percent Change From Baseline in Lipoprotein (a) [ Time Frame: 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 ]
  4. Percent Change From Baseline in Apolipoprotein B [ Time Frame: 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 ]
  5. Percent Change From Baseline in Total Cholesterol/HDL-C Ratio [ Time Frame: 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 ]
  6. Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio [ Time Frame: 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 ]
  7. Percentage of Participants With a 15% or Greater Reduction in LDL-C [ Time Frame: 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 ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   12 Years to 80 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01624142


Locations
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United States, California
Research Site
Los Angeles, California, United States, 90048
United States, New York
Research Site
New York, New York, United States, 10021
Research Site
New York, New York, United States, 10029
Research Site
New York, New York, United States, 10032
United States, Ohio
Research Site
Cincinnati, Ohio, United States, 45227
United States, Tennessee
Research Site
Nashville, Tennessee, United States, 37232
Australia, Tasmania
Research Site
Hobart, Tasmania, Australia, 7000
Australia, Western Australia
Research Site
Perth, Western Australia, Australia, 6000
Belgium
Research Site
Bruxelles, Belgium, 1200
Research Site
La Louvière, Belgium, 7100
Brazil
Research Site
Sao Paulo, São Paulo, Brazil, 04039-030
Research Site
São Paulo, Brazil, 05403-000
Canada, Ontario
Research Site
London, Ontario, Canada, N6A 5K8
Canada, Quebec
Research Site
Chicoutimi, Quebec, Canada, G7H 7K9
Research Site
Montreal, Quebec, Canada, H2W 1R7
Research Site
Montreal, Quebec, Canada, H3A 1A1
Canada
Research Site
Quebec, Canada, G1V 4M6
Czechia
Research Site
Brno, Czechia, 656 91
Research Site
Hradec Kralove, Czechia, 500 05
Research Site
Olomouc, Czechia, 775 20
Research Site
Praha 2, Czechia, 128 08
Research Site
Uherske Hradiste, Czechia, 686 01
France
Research Site
Dijon, France, 21000
Research Site
Paris Cedex 13, France, 75651
Greece
Research Site
Athens, Greece, 17674
Hong Kong
Research Site
New Territories, Hong Kong
Israel
Research Site
Ramat Gan, Israel, 52621
Italy
Research Site
Cinisello Balsamo (MI), Italy, 20092
Research Site
Napoli, Italy, 80131
Research Site
Pisa, Italy, 56124
Japan
Research Site
Kanazawa, Ishikawa, Japan, 920-8641
Research Site
Suita, Osaka, Japan, 565-8565
Lebanon
Research Site
Beirut, Lebanon, 0000
Netherlands
Research Site
Amsterdam, Netherlands, 1105 AZ
Research Site
Rotterdam, Netherlands, 3045 PM
New Zealand
Research Site
Christchurch, New Zealand, 8011
South Africa
Research Site
Johannesburg, Gauteng, South Africa, 2193
Research Site
Observatory, Western Cape, South Africa, 7925
Spain
Research Site
Cordoba, Andalucía, Spain, 14004
Research Site
Barcelona, Cataluña, Spain, 08036
Research Site
A Coruña, Galicia, Spain, 15001
Research Site
Lugo, Galicia, Spain, 27003
Research Site
Madrid, Spain, 28040
United Kingdom
Research Site
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Amgen
Investigators
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Study Director: MD Amgen
  Study Documents (Full-Text)

Documents provided by Amgen:
Study Protocol  [PDF] December 2, 2015
Statistical Analysis Plan  [PDF] May 26, 2018


Additional Information:
Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT01624142    
Other Study ID Numbers: 20110271
2011-005400-15 ( EudraCT Number )
First Posted: June 20, 2012    Key Record Dates
Results First Posted: January 28, 2019
Last Update Posted: January 28, 2019
Last Verified: January 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amgen:
Hypercholesterolemia
Elevated Cholesterol
High Cholesterol
Homozygous Familial Hypercholesterolemia
PCSK9 mutations
Severe Familial Hypercholesterolemia
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type II
Hypercholesterolemia
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Hyperlipoproteinemias
Evolocumab
Antibodies, Monoclonal
Anticholesteremic Agents
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents
Immunologic Factors
Physiological Effects of Drugs