Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With With Familial Partial Lipodystrophy (FPL)
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Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
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 HbA1c ≥ 7% to ≤ 12% at Screening and on anti-diabetic therapy as defined in study protocol
Hypertriglyceridemia as defined by fasting TG levels ≥ 500 mg/dL at both Screening and Qualification visits. Patients with the clinical diagnosis of FPL and with fasting TG levels ≥ 200 (≥ 2.26 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 MRI indicating a hepatic fat fraction (HFF) ≥ 6.4%
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 patient participating in or completing the study