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

Completed · Phase 2 · Has a placebo group

Conditions studied: Lipodystrophy

In brief

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. ...

Key facts

Study ID

NCT02639286

Run by

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

People needed

5

Starts

2015-12-23

Expected to finish

2019-09-26

Last updated by the study team

2020-10-20

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• A diagnosis of generalized lipodystrophy
• Current or history of autoimmune diseases (even with a diagnosis of PL) unless approved by the Investigator and Sponsor Medical Monitor
• Acute pancreatitis within 4 weeks of enrollment
• History within 6 months of enrollment of acute or unstable cardiac ischemia (myocardial infarction, acute coronary syndrome, new onset angina), stroke, transient ischemic attack, or unstable congestive heart failure requiring a change in medication
• Major surgery within 3 months of enrollment
• History of heart failure with New York Heart Association functional classification (NYHA) greater than Class II
• Uncontrolled hypertension (blood pressure [BP] >160/100 mm Hg)
• Any of the following laboratory values at enrollment:
• Cardiac troponin T > upper limit of normal (ULN)
• Measured or estimated (in case of triglycerides > 400 mg/dL) LDL-C >130 mg/dL on maximal tolerated statin therapy
• Hemoglobin HbA1c greater than or equal to 9.5%
• Hepatic:
• Total bilirubin >ULN
• Alanine transaminase (ALT) >3.0 x ULN. Higher levels will be permitted after a safety review by a hepatologist, that includes no evidence of cirrhosis.
• Aspartate aminotransferase ( (AST) >3.0 x ULN. Higher levels will be permitted after a safety review by a hepatologist, that includes no evidence of cirrhosis.
• Renal:
• Persistently positive (2 out of 3 tests greater than or equal to trace positive) for blood on urine dipstick. In the event of a positive test eligibility may be confirmed with urine microscopy showing less than or equal to 5 red blood cells per high power field (urine will be screened at admission and repeated if abnormal). If red blood cell counts (RBC) are high every effort will be made to determine if the source is renal or benign, such as from menstrual bleeding. If the presence of RBC is determined to be from a non-renal source, the PI will make the decision to proceed with protocol testing and/or randomization.
• Two out of three consecutive tests greater than or equal to 1+ for protein on urine dipstick. In the event of a positive test eligibility may be confirmed by either a spot urine albumin to creatinine ratio <1000mg/g or a quantitative total urine albumin measurement of < 1g/24 hrs (urine will be screened at admission and repeated if abnormal)
• Estimated creatinine clearance calculated according to the formula of Cockcroft and Gault <60 mL/min
• Platelet count below 140,000 K/uL
• Clinically significant (as determined by the Investigator or Sponsor) abnormalities on laboratory examination that will increase risk to the patient or interfere with data integrity
• Uncontrolled hypothyroidism (abnormal thyroid function tests should be approved by the Investigator)
• History within 6 months of enrollment of screening of drug or alcohol abuse
• History of bleeding diathesis or coagulopathy or clinically significant abnormality in coagulation parameters at enrollment
• Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to enrollment

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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