Short-term Effects of Leptin in People With Lipodystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01778556
• Date of registration: 26/01/2013
• Prospective Registration: Yes
• Primary sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
• Public title: Short-term Effects of Leptin in People With Lipodystrophy
• Scientific title: Short Term Effects of Leptin Withdrawal or Initiation in Lipodystrophy Independent of Energy Intake
• Date of first enrolment: January 26, 2013
• Target sample size: 25
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01778556
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

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

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

- Age 14-70 years (children under age 18 will only be enrolled in the leptin-naive arm
of the study

- Clinically-significant lipodystrophy as defined in protocol 02-DK-0022 (Long Term
Efficacy of Leptin Replacement in the Treatment of Lipodystrophy). Relevant inclusion
criteria for enrollment in protocol 02-DK-0022 are (summarized):

- Lipodystrophy identified by the study physician during physical examination as an
absence of fat outside the range of normal

- Circulating leptin levels < 12.0 ng/mL in females and < 8.0 ng/mL in males

- Presence of at least one of the following metabolic abnormalities:

1. Diabetes as defined by the 2007 American Diabetes Association criteria

2. Fasting insulin >30 microU/mL

3. Fasting hypertriglyceridemia >200 mg/dL

- Co-enrolled in protocol 02-DK-0022 and either:

- Leptin naive, with plans to initiate leptin treatment during the current study.
For the purpose of this study, leptin naive will be defined as having received no
exogenous leptin in the 4 months prior to study participation. Thus, subjects who
previously received leptin therapy, discontinued, and wish to restart are
eligible.

Or

--Leptin treated, meaning the subject has taken a stable dose of exogenous leptin for a
minimum of 4 months (adults over age 18, only)

EXCLUSION CRITERIA:

In leptin treated subjects only, the following exclusion criteria apply:

- Poorly controlled diabetes at study entry (hemoglobin A1c greater than or equal to 9%)

- Poorly controlled hypertriglyceridemia at study entry (serum triglycerides > 800
mg/dL)

- Extreme hypertriglyceridemia prior to leptin (triglycerides greater than 2000 mg/dL at
initiation of leptin treatment)

- History of chronic or recurrent acute pancreatitis (> 1 episode), or a single episode
of pancreatitis while receiving leptin treatment

- Lipase greater than the upper limit of normal (491 units/L) at study entry

In all subjects (leptin treated and leptin naive), the following exclusion criteria apply:

- Known HIV infection or HIV-associated lipodystrophy

- History of diabetic ketoacidosis

- Active inflammatory disease (e.g. dermatomyositis)

- Change in diabetes or lipid-lowering medications within the past 6 weeks

- Estimated glomerular filtration rate < 30 mL/minute

- Current or recent (past 2 weeks) use of systemic glucocorticoids

- Inadequately controlled hypothyroidism (TSH < 0.4 or >4 mcIU/L) or change in thyroid
medication in the past 8 weeks.

- Pregnancy or breast-feeding

- Psychiatric disorder impeding competence or compliance

- Any medical condition or medication that will increase risk to the subject (e.g.
ischemic heart disease, decompensated liver disease) or that will interfere with
interpretation of study data (e.g. Cushing s syndrome).



Age minimum: 14 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Lipodystrophy

Intervention(s)

Biological: Metreleptin

Primary Outcome(s)

Total Body Insulin Sensitivity [Time Frame: Intervention 1 (5 days), Intervention 2 (14 days), and Long-term follow-up (6 months)]

Secondary Outcome(s)

Endogenous Rate of Appearance of Palmitate [Time Frame: Intervention 1 (5 days), Intervention 2 (14 days), and Long-term follow-up (6 months)]

Insulin-mediated Suppression of Hepatic Glucose Production [Time Frame: Intervention 1 (5 days), Intervention 2 (14 days), and Long-term follow-up (6 months)]

Secondary ID(s)

130057

13-DK-0057

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 13/11/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01778556