Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL) (NCT03514420) | Clinical Trial Compass
CompletedPhase 2
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
United States4 participantsStarted 2018-06-15
Plain-language 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.
Who can participate
Age range18 Years
SexALL
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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.
What they're measuring
1
Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)
Timeframe: Baseline and End of the Treatment (Week 27)