Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Akcea Therapeutics
With
Ionis Pharmaceuticals, Inc.
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Must give written informed consent to participate in the study (signed and dated) and any authorizations required by law.
Clinical diagnosis of familial partial lipodystrophy (FPL) plus diagnosis of type 2 diabetes mellitus, hypertriglyceridemia, and fatty liver.
Diagnosis of FPL is based on deficiency of subcutaneous body fat in a partial fashion assessed by physical examination and low skinfold thickness in anterior thigh by caliper measurement: men (less than or equal to \[≤\] 10 millimeters \[mm\]) and women (≤ 22 mm), and at least 1 of the following:
1. Genetic diagnosis of FPL OR
2. Family history of FPL or of similar abnormal fat distribution plus 1 Minor Criteria OR
3. In the absence of FPL-associated genetic variant or family history, 2 Minor Criteria and body mass index (BMI) less than (\<) 35 kilogram per meter square (kg/m\^2).
Diabetes not well controlled on antidiabetic therapy with glycated hemoglobin (Hb) HbA1c more than or equal to (≥) 7 percentage (%) to ≤ 12% at Screening.
Hypertriglyceridemia with fasting triglycerides (TG) levels greater than or equal to (≥) 500 milligrams per deciliter (mg/dL) (≥ 5.7 millimoles per liter \[mmol/L\]) at Screening and Qualification visit, or Fasting TG levels ≥ 200 (≥ 2.26 mmol/L) at both Screening and Qualification Visits for participants who meet the genetic or family history criteria.
Presence of hepatosteatosis (fatty liver), as evidenced by a screening magnetic resonance imaging (MRI) indicating a hepatic fat fraction (HFF) ≥ 6.4%.
You may not be if
A diagnosis of generalized lipodystrophy.
A diagnosis of acquired partial lipodystrophy.
Acute pancreatitis within 4 weeks of Screening.
History within 6 months of Screening of acute or unstable cardiac condition.
Low-density lipoprotein cholesterol (LDL-C) more than (\>) 130 mg/dL on maximal tolerated statin therapy.
Platelet count \< lower limit of normal (LLN).
Treatment with metreleptin within the last 3 months prior to Screening.
Clareo Health | The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy
