Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
- Conditions
- Homozygous familial hypercholesterolemiaMedDRA version: 20.0Level: LLTClassification code 10057080Term: Homozygous familial hypercholesterolemiaSystem Organ Class: 100000004850Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
- Registration Number
- EUCTR2017-003170-13-IT
- Lead Sponsor
- REGENERON PHARMACEUTICALS, INC.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 116
1. Male and female patients =12 years of age with HoFH. Patients aged =
12 years old will be enrolled only in countries where permitted by the
Regulatory Agency and Institutional Review Board (IRB) or Ethics Committee (EC).
2. Diagnosis of functional HoFH by at least 1 of the following genetic or
clinical criteria:
a. Documented functional mutation or mutations in both LDLR alleles
Note: patients who have null receptor mutations on both LDLR alleles, ie,
double null, are eligible
b. Presence of homozygous or compound heterozygous mutations in Apo
B or PCSK9
Note: patients who are double heterozygous, ie, mutations on different
genes (eg, LDLR/PCSK9) and patients with homozygous LDLRAP1
mutations are eligible
c. Untreated TC >500 mg/dL (12.93 mmol/L) and TG <300 mg/dL (3.39
mmol/L) AND both parents with documented TC >250 mg/dL (6.47
mmol) OR cutaneous or tendinous xanthoma before the age of 10 years
3. For patients who have participated in a previous evinacumab or
alirocumab study: completion of the study in which they participated.
4. Willing and able to comply with clinic visits and study-related
procedures.
5. Provide signed informed consent.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 94
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Concomitant medications and procedures that have not been stable prior to the baseline visit 2. 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.
3. History of a MI, unstable angina leading to hospitalization, coronary artery bypass graft surgery, percutaneous coronary intervention, uncontrolled cardiac arrhythmia, carotid surgery or stenting, stroke, transient ischemic attack, valve replacement surgery, carotid revascularization, endovascular procedure or surgical intervention for peripheral vascular disease within 3 months prior to the baseline visit 4. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins 5. Newly diagnosed (within 3 months prior to screening visit diabetes mellitus or poorly controlled (HbA1c >9%) diabetes
6. Use of systemic corticosteroids, unless used as replacement therapy for pituitary/adrenal disease with a stable regimen for at least 6 weeks prior to screening visit
7. Use of estrogen or testosterone therapy unless the regimen has been stable 6 weeks prior to the screening visit and no plans to change the regimen during the study 8. Systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg at the screening visit 9. History of cancer within the past 5 years, except for adequately treated basal cell skin cancer, squamous cell skin cancer, or in situ cervical cancer 10. History of New York Heart Association (NYHA) Class IV heart failure within 12 months before screening 11. Laboratory findings during the screening period (applies to patients undergoing Screening): • Alanine aminotransferase or aspartate aminotransferase >3 x upper limit of normal (ULN)(1 repeat lab is allowed) • CPK >3 x ULN (1 repeat lab is allowed) • Positive serum beta-human chorionic gonadotropin or urine pregnancy test in WOCBP • TSH >1.5 x ULN of the central laboratory (1 repeat lab is allowed) for patients not on thyroid replacement therapy
• Positive test for hepatitis B surface antigen and/or hepatitis C antibody (associated with a positive HCV RNA polymerase chain reaction) • eGFR <30 mL/min/1.73 m2 (calculated by central lab) • Postmenopausal status will be confirmed by measurement of folliclestimulating hormone (FSH) 12. Member of the clinical site study team and/or his/her immediate family. 13. Pregnant of breastfeeding women 14. Women of child bearing potential (see protocol for more information) 15. Men who are sexually active with WOCBP and are unwilling to
consistently use condoms during the study drug treatment period and for 24 weeks after the last injection of study drug, regardless of vasectomy status. Sperm donation is prohibited during the study and for up to 24 weeks after the last injection of study drug.
16. LDL-C level <40 mg/dL at screening visit 17. Use of any active investigational drugs (except alirocumab) within 1 month or 5 half lives prior to the screening visit, whichever is longer. 18. Age <12 years at the screening visit 19. Tanner stage <2 at the screening visit Exclusion Criteria for Patients from a Previous Evinacumab Study
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method