Efficacy, Safety and Tolerability of ISIS 304801 in People With Partial Lipodystrophy With an Ope… (NCT02639286) | Clinical Trial Compass
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 range
18 Years
Sex
ALL
See this in plain English?
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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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)