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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03409744




Registration number
NCT03409744
Ethics application status
Date submitted
8/01/2018
Date registered
24/01/2018
Date last updated
30/04/2024

Titles & IDs
Public title
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Scientific title
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Secondary ID [1] 0 0
2017-003170-13
Secondary ID [2] 0 0
R1500-CL-1719
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Homozygous Familial Hypercholesterolemia 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - evinacumab

Experimental: evinacumab -


Treatment: Drugs: evinacumab
Intravenous (IV) administration
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216
Timepoint [1] 0 0
Up to 216 weeks
Secondary outcome [1] 0 0
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time
Timepoint [1] 0 0
Up to 120 weeks
Secondary outcome [2] 0 0
Absolute Change in LDL-C Over Time
Timepoint [2] 0 0
Up to 120 weeks
Secondary outcome [3] 0 0
Percent Change in Apolipoprotein B (Apo B) Over Time
Timepoint [3] 0 0
Up to 120 weeks
Secondary outcome [4] 0 0
Absolute Change in Apo B Over Time
Timepoint [4] 0 0
Up to 120 weeks
Secondary outcome [5] 0 0
Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time
Timepoint [5] 0 0
Up to 120 weeks
Secondary outcome [6] 0 0
Absolute Change in Non-HDL-C Over Time
Timepoint [6] 0 0
Up to 120 weeks
Secondary outcome [7] 0 0
Percent Change in Total Cholesterol (TC) Over Time
Timepoint [7] 0 0
Up to 120 weeks
Secondary outcome [8] 0 0
Absolute Change in TC Over Time
Timepoint [8] 0 0
Up to 120 weeks
Secondary outcome [9] 0 0
Percent Change in Triglycerides (TGs) Over Time
Timepoint [9] 0 0
Up to 120 weeks
Secondary outcome [10] 0 0
Absolute Change in TGs Over Time
Timepoint [10] 0 0
Up to 120 weeks

Eligibility
Key inclusion criteria
Key

1. Completion of the parent study in which they participated
2. Able to understand and complete study-related questionnaires

Key
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Significant protocol deviation in the parent study based on the investigator's judgment, such as non-compliance by the patient
2. Concomitant medications that have not been stable prior to the baseline visit
3. Adverse event leading to permanent discontinuation from parent study
4. Any new condition or worsening of an existing condition, which in the opinion of the investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study
5. Member of the clinical site study team and/or his/her immediate family
6. Pregnant or breastfeeding women
7. Women of childbearing potential who are unwilling to practice highly effective contraception prior to the initial dose/start of the first treatment, during the study, and for at least 24 weeks after the last dose of study drug
8. Men who are sexually active with women of childbearing potential and are unwilling to use the following forms of medically acceptable birth control during the study drug treatment period and for 24 weeks after the last injection of study drug: vasectomy with medical assessment of surgical success OR consistent use of a condom.

Note: Other protocol defined Inclusion/Exclusion criteria apply

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/03/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
13/04/2023
Sample size
Target
Accrual to date
Final
116
Recruitment in Australia
Recruitment state(s)
NSW,WA
Recruitment hospital [1] 0 0
Clinical Trial Site - Camperdown
Recruitment hospital [2] 0 0
Clinical Trial Site - Perth
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
6000 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
New York
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
United States of America
State/province [6] 0 0
Oregon
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
Austria
State/province [9] 0 0
Wien
Country [10] 0 0
Austria
State/province [10] 0 0
Innsbruck
Country [11] 0 0
Canada
State/province [11] 0 0
Quebec
Country [12] 0 0
Czechia
State/province [12] 0 0
Katerinská
Country [13] 0 0
France
State/province [13] 0 0
Cedex 13
Country [14] 0 0
France
State/province [14] 0 0
Cedex 5
Country [15] 0 0
France
State/province [15] 0 0
Cedex
Country [16] 0 0
Greece
State/province [16] 0 0
Athens
Country [17] 0 0
Greece
State/province [17] 0 0
Ioannina
Country [18] 0 0
Italy
State/province [18] 0 0
Campania
Country [19] 0 0
Italy
State/province [19] 0 0
Rome
Country [20] 0 0
Japan
State/province [20] 0 0
Aichi
Country [21] 0 0
Japan
State/province [21] 0 0
Fukuoka
Country [22] 0 0
Japan
State/province [22] 0 0
Hyogo
Country [23] 0 0
Japan
State/province [23] 0 0
Ishikawa
Country [24] 0 0
Japan
State/province [24] 0 0
Osaka
Country [25] 0 0
Netherlands
State/province [25] 0 0
Amsterdam
Country [26] 0 0
South Africa
State/province [26] 0 0
Gauteng
Country [27] 0 0
South Africa
State/province [27] 0 0
Western Cape
Country [28] 0 0
Ukraine
State/province [28] 0 0
Ivano-Frankivsk
Country [29] 0 0
Ukraine
State/province [29] 0 0
Kharkiv
Country [30] 0 0
Ukraine
State/province [30] 0 0
Kyiv

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Regeneron Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The primary objectives of the study are:

* To evaluate the long-term safety and tolerability of evinacumab in patients with Homozygous Familial Hypercholesterolemia (HoFH)
* To evaluate the long-term safety and tolerability of evinacumab in adolescent patients with HoFH

The secondary objectives of the study are:

* To evaluate the effect of evinacumab on lipid parameters in patients with HoFH
* To evaluate the effect of evinacumab on lipid parameters in adolescent patients with HoFH
* To evaluate the potential development of anti-evinacumab antibodies
Trial website
https://clinicaltrials.gov/study/NCT03409744
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trial Management
Address 0 0
Regeneron Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03409744