A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Evinacumab in Patients with Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

Phase 2

Completed

• Conditions: elevated fat levels in the blood; Severe Hypertriglyceridemia; 10013317

• Registration Number: NL-OMON52186
• Lead Sponsor: Regeneron Pharmaceuticals, Inc.

Brief Summary

Trial ended prematurely

Detailed Description

Not available

Study Timeline

• Results First Posted: 2023-09-21
• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 8

Inclusion Criteria

A patient must meet the following criteria to be eligible for inclusion in the
study:
1. Adults 18 to 80 years of age without FCS due to LPL loss of function
mutations
2. Documented history of 1 HTG-associated AP episode within 24 months of
screening (can be determined by study investigator; does not need to be
confirmed by independent adjudication committee).
3. Fasting serum TG value >880 mg/dL (10 mmol/L) on 2 occasions at least 2 days
apart determined during the screening period. Triglyceride measurement can be
repeated once for values >500 mg/dL (5.6 mmol/L) but <880 mg/dL (10 mmol/L)
OR
Fasting serum TG value >500 mg/dL (5.6 mmol/L) determined during the screening
period in patients with a history of 2 or more HTGassociated AP episodes within
24 months of screening
OR
Fasting serum TG value >500 mg/dL (5.6 mmol/L) determined during the screening
period and a documented fasted serum TG value >1000 mg/dL (11.3 mmol/L) and a
history of 1 or more HTG-associated AP episode(s) within 24 months of screening.
4. Stable dose of lipid-lowering therapy (>=8 weeks) and willingness to maintain
a stable regimen throughout the study
5. Body mass index >=18.0 and <=45.0 kg/m2
6. Compliance with a stable diet and exercise regimen at screening and
willingness to continue the diet through the end of the study
7. Willing and able to comply with clinic visits and study-related procedures
8. Provide informed consent signed by study patient or legally acceptable
representative
9. Able to understand and complete study-related questionnaires

Exclusion Criteria

A patient who meets any of the following criteria will be excluded from the
study:
1. Hospitalization for AP within 4 weeks of screening
2. Known genetic FCS defined as homozygous or compound heterozygous LoF
mutations in LPL, as documented by prior genotype result or determined from FCS
genotyping at screening (see Section 9.2.7).
3. Symptomatic gallstone disease within 6 months prior to screening. Incidental
and/or asymptomatic gallstones are permitted. Patients with symptomatic
gallstone disease in the past 6 months who have undergone cholecystectomy >3
months prior to screening are permitted.
4. Use of any medication or nutraceutical known to alter serum lipids which has
not been part of a stable therapeutic regimen for at least 8 weeks, and there
are no plans to change the regimen during the study
5. Presence of any clinically significant, uncontrolled endocrine disease known
to influence serum lipids, including but not limited to:
a. Newly diagnosed (within 3 months) diabetes by medical history, including
screening value glycosylated hemoglobin (HbA1c) >6.5% without a prior history
of diabetes
b. Diabetes with HbA1c >10.0%
c. Thyroid disease with thyroid-stimulating hormone (TSH) normal (LLN) or >1.5x ULN
d. Thyroid replacement therapy that has not been stable for at least 12 weeks

Note: For laboratory values, 1 repeat measurement is allowed. Other laboratory
values that meet the inclusion/exclusion criteria do not need to be repeated.
6. Use of estrogen or testosterone therapy unless the regimen has been stable
in the past 6 weeks and there are no plans to change the regimen during the
study
7. Any clinically significant abnormality identified at the time of screening
that, in the judgment of the investigator or any sub-investigator, would
preclude safe completion of the study or constrain endpoints assessment; eg,
major systemic diseases, patients with short life expectancy, or considered by
the investigator or any sub-investigator as inappropriate for this study for
any reason, including but not limited to:
a. Deemed unable to meet specific protocol requirements, such as scheduled
visits
b. Deemed unable to tolerate injections, as per the patient or the investigator
c. Part of a vulnerable population such as the institutionalized
d. Presence of any other conditions (eg, geographic or social), either actual
or anticipated, that the investigator feels would restrict or limit the
patient*s participation for the duration of the study
8. Laboratory findings (for the reason that patients with these findings, who
have a higher likelihood of liver, muscle, or kidney adverse events regardless
of treatment assignment, are expected to be rare, they may not be evenly
distributed across treatment groups and thus may confound the analysis of
safety):
a. Estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m2 according to
4 variable Modification of Diet in Renal Disease study equation (MDRD,
calculated by central laboratory)
b. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >3x ULN at
screening
c. Creatine phosphokinase (CPK) >3x ULN at screening

Note: For all laboratory values, 1 repeat measurement is allowed.
9. Systolic blood pressure >160 mmHg or diastolic blood pressure >100 mmHg at
the screening

Study & Design

• Study Type: Interventional
• Study Design: Not specified