Back to Search Results
Study Complete
Sponsor ID
20120123

Trial Assessing Efficacy, Safety and Tolerability of Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) Inhibition in Paediatric Subjects With Genetic Low-Density Lipoprotein (LDL) DisordersHAUSER-RCT

Sponsor ID
20120123
Clinicaltrials.gov ID
NCT02392559
EUCTIS ID
N/A
EudraCT ID
2014-002277-11
Contact Us

Study Details

A study to assess safety and efficacy of evolocumab (AMG-145) in paediatric subjects aged 10-17 years diagnosed with heterozygous familial hypercholesterolemia.

Additional Study Details

Phase
Phase 3
Product
Evolocumab
Type
Interventional
Masking
Triple (Participant, Care Provider, Investigator)
Enrollment
158
Additional Study Details

Protocol Summary

Primary Outcome Measures

Percent Change From Baseline to Week 24 in LDL-C

Timeframe: Baseline, Week 24

Secondary Outcome Measures

Mean Percent Change from Baseline to Mean of Weeks 22 and 24 in LDL-C

Timeframe: Baseline, Week 22, Week 24

Change From Baseline to Week 24 in LDL-C

Timeframe: Baseline, Week 24

Percent Change From Baseline to Week 24 in Non-HDL-C

Timeframe: Baseline, Week 24

Percent Change From Baseline to Week 24 in Apoliprotein-B (ApoB)

Timeframe: Baseline, Week 24

Percent Change From Baseline to Week 24 in Total Cholesterol/HDL-C Ratio

Timeframe: Baseline, Week 24

Percent Change From Baseline to Week 24 in ApoB:ApoA1 Ratio

Timeframe: Baseline, Week 24

Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious TEAEs, TEAEs Leading to Discontinuation (DC), Fatal TEAEs, and Device-Related TEAEs

Timeframe: From first dose of study drug through Week 24 (±3 days)

Number of Participants With Maximum Post-Baseline Laboratory Toxicities of Grade ≥ 3

Timeframe: Week 24

Change From Baseline Over Time in Systolic Blood Pressure

Timeframe: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24

Change From Baseline Over Time in Diastolic Blood Pressure

Timeframe: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24

Change From Baseline Over Time in Heart Rate

Timeframe: Baseline, Week 4, Week 12, Week 20, Week 22, Week 24

Serum Evolocumab Concentrations Over Time

Timeframe: Week 12, Week 22, Week 24

Study Documents

Study Synopsis
Available Language(s): English
Download Document(s)

Locations

Location
Status
Contact Us
Location
Research Site
Cincinnati, OH, United States, 45227
Status
Recruiting
Location
Research Site
Praha 5, Czech Republic, 150 06
Status
Completed
Location
Research Site
Svitavy, Czech Republic, 568 25
Status
Recruiting
Location
Research Site
Ostrava-Poruba, Czech Republic, 708 52
Status
Completed
Location
Research Site
Kuopio, Finland, 70029
Status
Completed
Location
Research Site
Helsinki, Finland, 00029
Status
Completed
Showing {first} - {last} of {total} Results
Study Info
Age
10 - 17 Years
Condition
  • Cholesterol
    • Sex
    All
    Trial Dates
    Mar 2016 - Nov 2019
    Inclusion and Exclusion Criteria
    Inclusion Criteria:
    - Male or female ≥ 10 to ≤ 17 years of age (before 18th birthday) - Diagnosis of heterozygous familial hypercholesterolemia - On an approved statin with stable optimized dose for ≥ 4 weeks - Other lipid-lowering therapy stable for ≥ 4 weeks (fibrates must be stable for ≥ 6 weeks) - Fasting LDL-C ≥ 130 mg/dL (3.4 mmol/L) - Fasting triglycerides ≤ 400 mg/dL (4.5 mmol/L)
    Exclusion Criteria:
    - Type 1 diabetes, or type 2 diabetes that is or poorly controlled - Uncontrolled hyperthyroidism or hypothyroidism - Cholesterylester transfer protein (CETP) inhibitor in the last 12 months, or mipomersen or lomitapide in the last 5 months - Previously received evolocumab or any other investigational therapy to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9). - Lipid apheresis within the last 12 weeks prior to screening. - Homozygous familial hypercholesterolemia