CompletedPhase 2

Efficacy, Safety and Tolerability of ISIS 304801 in People With Partial Lipodystrophy With an Open-Label Extension

United States5 participantsStarted 2015-12-23

Plain-language summary

Background: Partial lipodystrophy is a deficiency of body fat in parts of the body (usually the arms and legs). People with partial lipodystrophy often get high blood triglyceride (fat) level, insulin resistance, diabetes and other problems. Researchers think the new drug ISIS 304801 can help treat health problems caused by partial lipodystrophy. Objective: To see if ISIS 304801 will improve blood fat (triglyceride levels), diabetes, and liver disease, and reduce some risks for heart disease caused by partial lipodystrophy. Eligibility: Adults at least 18 years old with partial lipodystrophy. Design: Participants will be screened during a 1-week stay at NIH. They will have: Blood and urine tests Physical exam. Assignment to get either the study drug or placebo. Instructions for how to inject the drug. Body measurements. Heart tests. Participants will give themselves injections of the drug or placebo once a week at home. Some may test blood sugar by finger pricks. They will have monthly phone calls and nurse visits to take blood tests. After 4 months, participants may continue the study for 1 year. All participants will get the study drug. Participants will have study visits at NIH every 4 months. These may include: Insulin sensitivity measurement: Insulin and sugar will be infused through 2 intravenous (IV) lines in the arms. Blood will be drawn. Sugar and fat metabolism measured by IV infusions and blood tests. Special x-ray scan to measure body fat. Liquid meal then blood collected by IV catheter in the arm. Magnetic resonance imaging scans. Neck ultrasound. Questionnaires. Liver biopsy (optional) Injection of heparin (a blood thinner) before a blood test. After finishing the drug, participants will have 1 nurse visit and 1 visit to NIH. ...

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

* INCLUSION CRITERIA: * Age greater than or equal to 18 years at enrollment (the time of informed consent, week -1) * Fasting triglyceride (TG) levels greater than or equal to 500 mg/dL (greater than or equal to 5.7 mmol/L) at enrollment. If the fasting TG value is \<500 mg/dL (\<5.7 mmol/L) but greater than or equal to 350 mg/dL (greater than or equal to 4.0 mmol/L) up to two additional tests may be performed in order to qualify, and a single level greater than or equal to 500 mg/dL will permit enrollment. OR * Fasting TG levels greater than or equal to 200 mg/DL (2.6 mmol/L) with a hemoglobin A1C over 7%. * Willing to maintain their customary physical activity level and to follow a diet moderate in carbohydrates and fats with a focus on complex carbohydrates and replacing saturated for unsaturated fats * Clinical diagnosis of lipodystrophy 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 10mm) and women (less than or equal to 22mm), plus one of the following: * Genetic diagnosis of familial PL (e.g., mutations in LMNA, PPARG, AKT2, or PLIN1 genes) OR * Family history of familial PL or abnormal and similar fat distribution plus 1 minor criterion (below), OR * 2 minor criteria (below) in the absence of genetic diagnosis of family history * MINOR Criteria * Diabetes mellitus with requirement for high doses of i…

What they're measuring

Change in Log 10 Fasting Triglycerides.

Timeframe: Baseline and 16 weeks

Trial details

NCT IDNCT02639286

SponsorNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2019-09-26

Results submitted2020-09-21