Efficacy and Safety of REGN727/SAR236553 in patients with primary hypercholesterolemia who are intolerant to statins

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 250

Inclusion Criteria

1. Patients with primary hypercholesterolemia (heFH* or non-FH) with moderate, high or very high CV risk** and a history of statin intolerance***
* Diagnosis of heFH must be made either by genotyping or by clinical criteria. For patients who are not genotyped, the clinical diagnosis must be a certain/definite diagnosis and may be based on either the Simon Broome criteria or the WHO/Dutch Lipid Network criteria.
** Moderate, high, and very high CV risk as defined in section 4.2.
*** Definition of statin intolerance: Inability to tolerate at least 2 previous statins at the lowest approved daily dose due to skeletal muscle-related symptoms, other than those due to strain or trauma, such as pain, aches, weakness, or cramping, that began or increased during statin therapy and stopped when statin therapy was discontinued.
2. Provide signed informed consent
Are the trial subjects under 18? no
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 250
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion Criteria

1. Calculated serum LDL-C <70 mg/dL (1.81 mmol/L) and very high CV risk at the screening visit
2. Calculated serum LDL-C <100 mg/dL (2.59 mmol/L) and high or moderate CV risk at the screening visit
3. A 10-year fatal CVD risk SCORE < 1% at the screening visit
4. Use of a statin that is at or above the lowest approved daily dose within 4 weeks prior to the screening visit
5. Experienced skeletal muscle-related adverse event(s), other than those due to strain or trauma during the 4-week single-blind placebo run-in
6. Experiencing a skeletal muscle-related adverse event(s), other than those due to strain or trauma at the time of screening, start of single-blind placebo run-in period, or day 1/week 0
7. Not on a stable dose of LMT for at least 4 weeks and/or fenofibrate for at least 6 weeks, as applicable, prior to the screening visit or from screening to randomization, as applicable
8. Use of fibrates, other than fenofibrate, within 6 weeks of the screening visit
9. Use of nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/amount that has not been stable for at least 4 weeks prior to the screening visit or between the screening and randomization visits
10. Use of red yeast rice from the screening visit to the end-of-study visit
11. Use of analgesics for which the dose is not planned to be stable from the screening visit to the end-of-study visit
12. Diagnosis of fibromyalgia
13. History of severe neuropathic pain
14. History of rheumatological disease associated with symptoms that may be confounded with symptoms of statin intolerance, e.g., rheumatoid arthritis
15. History of myalgia or myopathy that began or increased during treatment with lipid modifying therapy (LMT), other than statin therapy, and stopped when the LMT was discontinued
16. Known history of seizure disorder
17. History of previous transplant surgery
18. Use of medications that require intramuscular administration, or planned intramuscular injections during the study
19. Known history of myopathy, other than statin-associated myopathy
20. History of rhabdomyolysis (defined as evidence of organ damage with creatine kinase (CK) > 10,000 IU/L)

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

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