Open studies conducted by NIDDK Principal Investigators appear below. Study statuses may include the following:
Open: Recruiting - Currently recruiting participants and open to everyone who meets eligibility criteria.
Open: Active, Not Recruiting - Participants are receiving an intervention or being examined, however new participants
are not being recruited or enrolled.
Open: Enrolling by Invitation - People in a particular population were selected in advance and invited to participate.
The study is not open to everyone who meets the eligibility criteria.
Open: Available for Expanded Access - Patients who are not participants in the clinical study may be able to
gain access to the drug, biologic, or medical device being studied.
Background:
- Generalized lipodystrophy can cause high blood fat levels and resistance to insulin. This
can lead to health problems including diabetes. Researchers have found that the drug
metreleptin improves health in people with this disease.
Objective:
- To test the safety and effectiveness of metreleptin.
Eligibility:
- People ages 6 months and older with generalized lipodystrophy who:
- have received metreleptin through NIH studies AND
- cannot get it through approved or compassionate use mechanisms in their home country.
Design:
- Participants will come to NIH approximately every 6 months during year one, then every 1
2 years. Financial assistance may be available for travel within the U.S.
- At visits, participants will get a supply of metreleptin to take home for daily
injections. They will have:
- plastic catheter placed in an arm vein.
- blood tests, urine collection, and physical exam.
- oral glucose tolerance test, drinking a sweet liquid.
- ultrasound of the heart, liver, uterus, and ovaries. A gel and a probe are placed on the
skin and pictures are taken of the organs.
- echocardiogram, which takes pictures of the heart with sound waves.
- Resting Metabolic Rate taken. A plastic hood is worn over the head while the oxygen they
breathe is measured.
- Participants will have up to 3 DEXA scan x-rays per year.
- Participants may have:
- annual bone x-rays.
- liver biopsies every few years. A needle will be inserted into the liver to obtain a
small piece. Participants will sign a separate consent for this.
- Participants must be seen regularly by their local doctors and have blood tests at least
every 3 6 months at home.
The trial is Open with a status of Enrolling by invitation.
Background:
- Partial lipodystrophy can cause high blood fat levels and resistance to insulin. This can
lead to health problems including diabetes. Researchers have found that the drug metreleptin
improves health in people with this disease.
Objective:
- To test the safety and effectiveness of metreleptin.
Eligibility:
- People age 6 months and older with partial lipodystrophy who
- have received metreleptin through NIH studies and shown improvement AND
- cannot get metreleptin other ways.
Design:
- Participants will come to NIH approximately every 6 months during year one, then every 1
2 years. Financial assistance may be available for travel within the U.S.
- At visits, participants will get a supply of metreleptin to take home for daily
injections, or it can be shipped to them inside the U.S. They will have:
- plastic catheter placed in an arm vein.
- blood tests, urine collection, and physical exam.
- oral glucose tolerance test, drinking a sweet liquid.
- ultrasound of the heart, liver, uterus, and ovaries. A gel and a probe are placed on the
skin and pictures are taken of the organs.
- echocardiogram, which takes pictures of the heart with sound waves.
- Resting Metabolic Rate taken. A plastic hood is worn over the head while the oxygen they
breathe is measured.
- Participants will have up to 3 DEXA scan x-rays per year.
- Participants may have:
- annual bone x-rays.
- liver biopsies every few years. A needle will be inserted into the liver to obtain a
small piece. Participants will sign a separate consent for this.
- Participants must be seen regularly by their local doctors and have blood tests at least
every 3-6 months at home.
The trial is Open with a status of Enrolling by invitation.
Background:
- Lipodystrophy is a condition where people do not have enough fat in the body. People with
lipodystrophy can have problems such as diabetes or an enlarged liver. Researchers are
looking at how leptin, a hormone produced by fat cells, can help people with these problems.
Leptin helps control appetite and how the body stores food. Taking leptin can help people
with lipodystrophy eat less food, which may help treat diabetes and other problems. To better
understand how leptin works, researchers want to do an inpatient study on leptin treatment in
people with lipodystrophy.
Objectives:
- To study how leptin treatment affects lipodystrophy.
Eligibility:
- Individuals between 14 and 70 years of age who have lipodystrophy.
Design:
- All participants will have a 19-day stay at the National Institutes of Health Clinical
Center. One group of participants will have tests for 5 days before starting to take
leptin. They will then take leptin for 2 weeks, and have more tests. The other group of
participants will have tests for 5 days while taking leptin. They will then take stop
taking leptin for 2 weeks, and have more tests, and then they will start taking leptin
again.
- Participants will have regular blood and urine tests during the visit. Some of the blood
tests will look at insulin levels. Some will look at how the body metabolizes sugar and
fat. Other tests will check hormone levels, especially of reproductive hormones.
- During the visit, participants will spend 3 separate days in a metabolic chamber, a
special room that measures how many calories the body uses. Urine samples will be
collected during these stays.
- Participants will also have several body imaging studies, including magnetic resonance
imaging and a body composition scan.
- Physical activity will be tested with an exercise bicycle and an electronic activity
monitor.
- Participants will be asked questions about hunger and comfort levels throughout the
stay.
Leptin is a hormone produced by the fat cells that researchers have shown to play a role in
controlling appetite. Certain people with severe insulin resistance have little or no leptin.
The purpose of this study is to investigate people whose leptin levels have been found to be
lower than 85 percent of the general population. Researchers will determine whether insulin
levels in these participants improve when they are treated with genetically engineered
leptin.
Study participants must be age 8 years or older and must have severe insulin resistance
syndrome and leptin deficiency. During an initial 7-day visit to the Clinical Center,
researchers will evaluate participants' metabolic parameters, such as insulin responsiveness,
lipid levels, appetite, and hormone levels. After taking these tests, participants will
self-inject doses of leptin once or twice a day and will be monitored for treatment outcomes
as well as side effects via follow-up visits. These inpatient follow-up visits will involve
both blood tests and imaging studies at the Clinical Center at 4, 8, and 12 months after the
initial visit, and every 6 months thereafter.
The study will allow researchers to obtain blood, plasma, DNA, and RNA for genetic studies of
insulin. There will be a focus on the causes of insulin resistance and diabetes mellitus.
Insulin is a hormone found in the body that controls the level of sugar in the blood. Insulin
resistance refers to conditions like diabetes when insulin does not work properly. In this
study researchers would like to compare patients with diabetes and other forms of insulin
resistance to normal individuals. The study will investigate how insulin attaches to cells.
Researchers will take 4 to 6 ounces (100-150 ml) of blood from adult patients and may request
up to 12 ounces (one unit) of blood if necessary. Skin samples may be taken for a biopsy if
further genetic testing is necessary. In addition some patients may be asked not to eat for
up to 72 hours prior to testing.
