Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)

Akcea Therapeutics

Status and phase

Completed

Phase 2

Conditions

Familial Partial Lipodystrophy

Treatments

Drug: AKCEA-ANGPTL3-LRx

Study type

Interventional

Funder types

Industry

Identifiers

NCT03514420

ISIS 703802-CS5

Details and patient eligibility

About

This is a single-center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of fasting triglycerides in participants with familial partial lipodystrophy.

Enrollment

4 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

Must give written informed consent to participate in the study.
Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia.
Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with hemoglobin A1c (HbA1c) ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as defined in study protocol.
Hypertriglyceridemia as defined by fasting triglycerides (TG) levels ≥ 500 milligrams per deciliter (mg/dL) at both Screening and Qualification visits. Participants with the clinical diagnosis of FPL and with fasting TG levels ≥ 200 (≥ 2.26 millimoles per liter [mmol/L]) to < 500 mg/dL (≥ 5.7 mmol/L) who meet the genetic or family history criteria for study inclusion may be further screened and enrolled in the study.
Presence of hepatosteatosis (fatty liver), as evidenced by a Screening magnetic resonance imaging (MRI) indicating a hepatic fat fraction (HFF) ≥ 6.4%.

Key Exclusion Criteria:

Diagnosis of generalized lipodystrophy.
Diagnosis of acquired partial lipodystrophy (APL).
Acute pancreatitis within 4 weeks of Screening.
Acute coronary syndrome within 6 months of Screening.
Major surgery within 3 months of Screening.
Have any other conditions in the opinion of the investigator which could interfere with the participant participating in or completing the study.