HypoleptinemiaPhase 2/3NCT00896298
Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
A Phase 2/3 study testing Leptin in people with Hypoleptinemia and Generalized Lipodystrophy, run by University of Texas Southwestern Medical Center at 1 site in United States.
Trial summary
Lipodystrophies represent a therapeutic challenge with regards to insulin resistance, hypertriglyceridemia and fatty liver which often is coupled with significant adipose tissue loss. The purpose of the study is to examine the safety and efficacy of Leptin on subjects with lipodystrophy. Read more about this study
- Who is it for?
- Ages 6 to 70 years
- Conditions the study is for
- Hypoleptinemia, Generalized Lipodystrophy, Partial Lipodystrophy
- What is being tested?
- Leptin
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2006to Nov 2014
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- age > 6 years
- Partial and generalized lipodystrophy
- Serum leptin levels less than the 7th percentile of normal values reported by the 3rd National Health and Nutrition Examination survey (less than 7.0 ng/mL in females and less than 3/0 ng/mL in males)
- Presence of at least one of the following metabolic abnormalities:
- Type 2 Diabetes Mellitus
- Fasting serum insulin >20 uU/mL
- Fasting serum triglycerides > 300 mg/dL
- Previous participation in leptin trial (Amgen 991265, GCRC 660) - for Part B of the Study.
Who cannot join
- Known liver disease due to causes other than non-alcoholic steatohepatitis.
- Hematocrit of less than 30%.
- Current alcohol or substance abuse.
- Use of anorexigenic drugs, anabolic steroids, GH and thiazolidinediones
- Active tuberculosis
- Psychiatric disorder impeding competence or compliance
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- Malignancies
- HIV infection
- Subjects who have a known hypersensitivity to E. Coli derived proteins
- Other condition, which in the opinion of the clinical investigators would impede completion of the study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
Lipodystrophies represent a therapeutic challenge with regards to insulin resistance, hypertriglyceridemia and fatty liver which often is coupled with significant adipose tissue loss. The purpose of the study is to examine the safety and efficacy of Leptin on subjects with lipodystrophy.
Read the detailed description
The mechanism by which leptin improves glucose and lipid control is not clear. We will examine the possible mechanisms of leptin action by studying the effects of leptin administration on food intake, insulin resistance, insulin secretory response, hepatic and intramuscular triglyceride stores in a large sample of patients with lipodystrophy.
Hypothesis: Leptin replacement in patients with generalized and partial lipodystrophy and hypoleptinemia will be safe and efficacious in improving the metabolic abnormalities associated with insulin resistance.
- Being tested
- Leptin, Placebo
- Keywords
- Lipodystrophy, Insulin secretory response
- Medical subject headings
- Lipodystrophy, Congenital Generalized, Lipodystrophy, Partial, Acquired, Insulin Resistance, Lipodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LeptinDrug
Also called Recombinant-methionyl Human Leptin., r-metHuLeptin
Leptin injected subcutaneously twice a day, to be administered at 100% of the estimated replacement dose during the first month, which is a dosage of 0.03mg/kg for female children, 0.04mg/kg for adult females, and0.02 mg/kg for all males, and then at 200% subsequently, or placebo.
PlaceboDrug
Sugar pill
| Study group | Role | What they receive |
|---|---|---|
| 1 Leptin | Active comparator | Active Comparator for 4 months, then for 8 months. Receives: Drug: Leptin |
| 2 Sugar pill | Placebo comparator | Placebo for 4 months, then active comparator for 8 months. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Fasting Serum Triglycerides
Measured: 4 months
HbA1c
Measured: 4 months
Secondary outcomes (2)
Fasting Serum Glucose
Measured: 4 months
Body Weight
Measured: 4 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 May 2009 (estimated)
- Study startApr 2006
- Primary completionNov 2014
- Study completionNov 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for HypoleptinemiaStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Abhimanyu Garg, University of Texas Southwestern Medical Center
- Investigators
- Abhimanyu Garg, MD, Principal investigator, University of Texas Southwestern Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00896298
- Sponsor's protocol ID
- 0502-294
- Status last verified
- Mar 2024
- First submitted
- 8 May 2009
- Last updated
- 27 Mar 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-Blind, Placebo-Controlled Trial of Leptin Replacement Therapy in Patients With Lipodystrophy
- ClinicalTrials.gov ID
- NCT00896298
- Acronym / sponsor ID
- 0502-294
- Keywords
- Lipodystrophy, Insulin secretory response
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Study start
- Apr 2006
- Primary completion
- Nov 2014 (actual)
- Study completion
- Nov 2014 (actual)
- First posted
- 11 May 2009 (estimated)
- Results
- Posted 5 Mar 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Lipodystrophies represent a therapeutic challenge with regards to insulin resistance, hypertriglyceridemia and fatty liver which often is coupled with significant adipose tissue loss. The purpose of the study is to examine the safety and efficacy of Leptin on subjects with lipodystrophy.
Detailed description
The mechanism by which leptin improves glucose and lipid control is not clear. We will examine the possible mechanisms of leptin action by studying the effects of leptin administration on food intake, insulin resistance, insulin secretory response, hepatic and intramuscular triglyceride stores in a large sample of patients with lipodystrophy.
Hypothesis: Leptin replacement in patients with generalized and partial lipodystrophy and hypoleptinemia will be safe and efficacious in improving the metabolic abnormalities associated with insulin resistance.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 Leptin | Active comparator | Drug: LeptinActive Comparator for 4 months, then for 8 months. |
| 2 Sugar pill | Placebo comparator | Drug: PlaceboPlacebo for 4 months, then active comparator for 8 months. |
Who may qualify
- Ages
- 6 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- age > 6 years
- Partial and generalized lipodystrophy
- Serum leptin levels less than the 7th percentile of normal values reported by the 3rd National Health and Nutrition Examination survey (less than 7.0 ng/mL in females and less than 3/0 ng/mL in males)
- Presence of at least one of the following metabolic abnormalities:
Key exclusion criteria (10 listed)
- Known liver disease due to causes other than non-alcoholic steatohepatitis.
- Hematocrit of less than 30%.
- Current alcohol or substance abuse.
- Use of anorexigenic drugs, anabolic steroids, GH and thiazolidinediones
- Active tuberculosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LeptinAlso: Recombinant-methionyl Human Leptin., r-metHuLeptinLeptin injected subcutaneously twice a day, to be administered at 100% of the estimated replacement dose during the first month, which is a dosage of 0.03mg/kg for female children, 0.04mg/kg for adult females, and0.02 mg/kg for all males, and then at 200% subsequently, or placebo. | Drug | 1 Leptin |
| PlaceboSugar pill | Drug | 2 Sugar pill |
Primary outcome
Fasting Serum Triglycerides
Time frame: 4 months
HbA1c
Time frame: 4 months
2 secondary outcomes
Fasting Serum Glucose
Time frame: 4 months
Body Weight
Time frame: 4 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Abhimanyu Garg, MD principal investigator
University of Texas Southwestern Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Leptin in people with Hypoleptinemia and Generalized Lipodystrophy, run by University of Texas Southwestern Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 5 Mar 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Fasting Serum Triglycerides (time frame: 4 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Mar 2024.