Protocol Details
Compassionate Use of Metreleptin in Previously-Treated Patients with Generalized Lipodystrophy
This study is NOT currently recruiting participants.
Summary
Number | 15-DK-0003 |
Sponsoring Institute | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
Recruitment Detail | Type: No longer recruiting/follow-up only
Gender: Male & Female
Min Age: 6 mo
Max Age: 98 Years |
Referral Letter Required | Yes |
Population Exclusion(s) | None |
Keywords | Diabetes;
Leptin;
Lipodystrophy;
Hypertriglyceridemia |
Recruitment Keyword(s) | None |
Condition(s) | Lipodystrophy;
Diabetes;
Hyperlipidemia |
Investigational Drug(s) | Metreleptin
|
Investigational Device(s) | None |
Intervention(s) | Drug: Metreleptin
|
Supporting Site | National Institute of Diabetes and Digestive and Kidney Diseases |
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.
Eligibility
INCLUSION CRITERIA:
1. Age greater than or equal to 6 months.
2. Generalized lipodystrophy (either congenital or acquired).
3. Those who cannot obtain metreleptin through approved or compassionate use mechanisms in their home country.
EXCLUSION CRITERIA:
1. Availability of metreleptin to the patient either as an approved drug, or through local compassionate use or expanded access programs.
2. Known HIV infection or HIV-associated lipodystrophy.
3. Any medical condition or medication that will increase risk to the subject.
4. Current alcohol or substance abuse.
5. Subjects who have a known hypersensitivity to E. coli derived proteins (as leptin is derived from such proteins).
Citations:
Malandrino N, Reynolds JC, Brychta RJ, Chen KY, Auh S, Gharib AM, Startzell M, Cochran EK, Brown RJ. Visceral fat does not contribute to metabolic disease in lipodystrophy. Obes Sci Pract. 2019 Jan 24;5(1):75-82. doi: 10.1002/osp4.319. eCollection 2019 Feb. Meral R, Ryan BJ, Malandrino N, Jalal A, Neidert AH, Muniyappa R, Ak nc B, Horowitz JF, Brown RJ, Oral EA. "Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy: When a Picture Is Worth a Thousand Numbers. Diabetes Care. 2018 Oct;41(10):2255-2258. doi: 10.2337/dc18-0978.
Contacts:
Clinical Trials Number:
NCT02262832Protocol Details
Compassionate Use of Metreleptin in Previously-Treated Patients with Generalized Lipodystrophy
This study is NOT currently recruiting participants.
Summary
Number | 15-DK-0003 |
Sponsoring Institute | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
Recruitment Detail | Type: No longer recruiting/follow-up only
Gender: Male & Female
Min Age: 6 mo
Max Age: 98 Years |
Referral Letter Required | Yes |
Population Exclusion(s) | None |
Keywords | Diabetes;
Leptin;
Lipodystrophy;
Hypertriglyceridemia |
Recruitment Keyword(s) | None |
Condition(s) | Lipodystrophy;
Diabetes;
Hyperlipidemia |
Investigational Drug(s) | Metreleptin
|
Investigational Device(s) | None |
Intervention(s) | Drug: Metreleptin
|
Supporting Site | National Institute of Diabetes and Digestive and Kidney Diseases |
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.
Eligibility
INCLUSION CRITERIA:
1. Age greater than or equal to 6 months.
2. Generalized lipodystrophy (either congenital or acquired).
3. Those who cannot obtain metreleptin through approved or compassionate use mechanisms in their home country.
EXCLUSION CRITERIA:
1. Availability of metreleptin to the patient either as an approved drug, or through local compassionate use or expanded access programs.
2. Known HIV infection or HIV-associated lipodystrophy.
3. Any medical condition or medication that will increase risk to the subject.
4. Current alcohol or substance abuse.
5. Subjects who have a known hypersensitivity to E. coli derived proteins (as leptin is derived from such proteins).
Citations:
Malandrino N, Reynolds JC, Brychta RJ, Chen KY, Auh S, Gharib AM, Startzell M, Cochran EK, Brown RJ. Visceral fat does not contribute to metabolic disease in lipodystrophy. Obes Sci Pract. 2019 Jan 24;5(1):75-82. doi: 10.1002/osp4.319. eCollection 2019 Feb. Meral R, Ryan BJ, Malandrino N, Jalal A, Neidert AH, Muniyappa R, Ak nc B, Horowitz JF, Brown RJ, Oral EA. "Fat Shadows" From DXA for the Qualitative Assessment of Lipodystrophy: When a Picture Is Worth a Thousand Numbers. Diabetes Care. 2018 Oct;41(10):2255-2258. doi: 10.2337/dc18-0978.
Contacts:
Clinical Trials Number:
NCT02262832