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

Primary Purpose

Severe Familial Hypercholesterolemia

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Evolocumab

About this trial

This is an interventional treatment trial for Severe Familial Hypercholesterolemia focused on measuring Hypercholesterolemia, Elevated Cholesterol, High Cholesterol, Homozygous Familial Hypercholesterolemia, PCSK9 mutations, Severe Familial Hypercholesterolemia

Eligibility Criteria

12 Years - 80 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

- Participated in Study 20110233 (NCT01588496) or another qualifying evolocumab parent protocol and have a diagnosis of familial hypercholesterolemia.

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

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Evolocumab

Arm Description

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)

Percent Change From Baseline in Non-high-density Lipoprotein Cholesterol (Non-HDL-C)

Percent Change From Baseline in Lipoprotein (a)

Percent Change From Baseline in Apolipoprotein B

Percent Change From Baseline in Total Cholesterol/HDL-C Ratio

Percent Change From Baseline in Apolipoprotein B/Apolipoprotein A1 Ratio

Percentage of Participants With a 15% or Greater Reduction in LDL-C

Full Information

NCT ID

NCT01624142

First Posted

June 5, 2012

Last Updated

November 3, 2022

Sponsor

Amgen

1. Study Identification

Unique Protocol Identification Number

NCT01624142

Brief Title

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

Acronym

TAUSSIG

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

Study Type

Interventional

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Completed

Study Start Date

June 1, 2012 (Actual)

Primary Completion Date

May 11, 2018 (Actual)

Study Completion Date

May 11, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Amgen

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

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

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Severe Familial Hypercholesterolemia

Keywords

Hypercholesterolemia, Elevated Cholesterol, High Cholesterol, Homozygous Familial Hypercholesterolemia, PCSK9 mutations, Severe Familial Hypercholesterolemia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

300 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Evolocumab

Arm Type

Experimental

Arm Description

Intervention Type

Biological

Intervention Name(s)

Evolocumab

Other Intervention Name(s)

AMG 145, Repatha

Intervention Description

Evolocumab was administered by subcutaneous injection either once a month (QM) or once every two weeks (Q2W).

Primary Outcome Measure Information:

Title

Number of Participants With Adverse Events

Description

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.

Secondary Outcome Measure Information:

Title

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C)

Time Frame