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

NCT02639286 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2020-10-20

Study results available
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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.

...

Conditions

  • Lipodystrophy

Interventions

DRUG

ISIS 304801

300 mg of ISIS 304801 administered as SC

DRUG

Placebo

Placebo administered via SC

Sponsors & Collaborators

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

    lead NIH

Principal Investigators

  • Rebecca J Brown, M.D. · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-12-23
Primary Completion
2019-09-26
Completion
2019-09-26
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02639286 on ClinicalTrials.gov