Overview
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
Status:
Completed
Completed
Trial end date:
2019-08-21
2019-08-21
Target enrollment:
0
0
Participant gender:
All
All
Summary
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.Phase:
Phase 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Akcea TherapeuticsCollaborator:
Ionis Pharmaceuticals, Inc.
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.