Open-label study to evaluate the safety, tolerability, and efficacy of LOmitapide for the treatment of patients with Familial CHylomicroNEmia Syndrome - LOCHNES

PRM|212180 sites20 target enrollmentMay 4, 2020

ConditionsFamilial Chylomicronemia Syndrome MedDRA version: 20.0Level: PTClassification code 10058108Term: DyslipidaemiaSystem Organ Class: 10027433 - Metabolism and nutrition disorders Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

DrugsLojuxta

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Familial Chylomicronemia Syndrome
Sponsor: PRM|21218
Enrollment: 20
Status: Active, not recruiting
Last Updated: 5 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

May 4, 2020

End Date

TBD

Last Updated

5 years ago

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

PRM|21218

Eligibility Criteria

Inclusion Criteria

•1\.Written informed consent
•2\.Age \= 18 years at time of informed consent
•3\.History of chylomicronemia as evidenced by documentation of lactescent serum or documentation of fasting TG measurement \= 885 mg/dl (10 mmol/L)
•4\.A diagnosis of Familial Chylomicronemia Syndrome by documentation of at least one of the following: confirmed homozygote, compound heterozygote or double heterozygote for known loss\-of\-function mutations in genes causing FCS
•5\.Fasting TG \= 750 mg/dl (8\.4 mmol/L) at screening
•6\.History of pancreatitis
•7\.Willing to follow a diet comprising \= 20 g fat per day during the study con quanto riportato a pag. 21 Screening procedures include also dietary counseling with regard to low\-fat diet (\=20% energy from fat) and to avoid alcohol completely while receiving Lomitapide
•8\.Satisfy one of the following:
•Females: non\-pregnant and non\-lactating; surgically sterile, post\-menopausal, abstinent, or if engaged in sexual relations of child\-bearing potential, patient is using an acceptable contraceptive method
•Males: Surgically sterile, abstinent or if engaged in sexual relations with a female of child\-bearing potential, patient is utilizing an acceptable contraceptive method

Exclusion Criteria

•1\.Diabetes mellitus with any of the following: newly diagnosed within 12 weeks of screening; HbA1c \= 9\.0% at screening; recent change in anti\-diabetic pharmacotherapy; anticipated need to change dose or type of medication during the treatment period of the study; current use of GLP\-1 agonists.
•2\.Severe hypertriglyceridemia other than due to FCS.
•3\.Active pancreatitis within 4 weeks prior to screening.
•4\.History within 6 months of screening of acute or unstable cardiac ischemia, stroke, transient ischemic attack or unstable congestive cardiac failure requiring a change in medication or major surgery within 3 months of screening.
•5\.Any of the following laboratory values at screening: total bilirubin \> upper limit of normal (ULN); AST \> 2\.0 x ULN; ALT \> 2\.0 x ULN; persistently positive for protein on urine dipstick; persistently positive for blood or urine dipstick; estimated creatinine clearance calculated according to the formula of Cockcroft and Gault \< 50 mL/min.
•6\.Uncontrolled hypertension (BP \> 160/100 mmHg).
•7\.History of bleeding diathesis or coagulopathy or clinically significant abnormality in coagulation parameters at screening.
•8\.History of heart failure with NYHA greater than Class II.
•9\.Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to begin treatment with Lomitapide (Baseline Visit).
•10\.Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B.