A Phase 4 interventional study of Metreleptin in Generalized Lipodystrophy, sponsored by Aegerion Pharmaceuticals, Inc.. Completed at 8 sites in United States. Open to participants aged 1 Year and older. Per ClinicalTrials.gov, last updated 2025-11-26.
Sponsored by Aegerion Pharmaceuticals, Inc. (part of Chiesi Farmaceutici S.p.A.) · Phase 4, Interventional, and Treatment
MYALEPT™ (metreleptin) has been approved as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy (MYALEPT Prescribing Information). This study is a multicenter, open-label, Phase 4 trial to provide an assessment of the immunogenicity associated with metreleptin and of any major potential risks due to development of antibodies to metreleptin. The study is being conducted to comply with a postmarketing requirement.
139 studies on the registry are indexed under Lipodystrophy; 10 are open to participants now.
This study's enrollment of 11 is below the median of 46 across 97 interventional studies indexed under Lipodystrophy.
Browse Lipodystrophy studies →Aegerion Pharmaceuticals, Inc. is the lead sponsor of 17 studies on the registry; none are open to participants now.
First submitted before Aegerion Pharmaceuticals, Inc. became part of Chiesi Farmaceutici S.p.A..
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
\<30 mL/min using institutional standards:
e.g., calculated using Cockcroft-Gault formula for patients ≥18 years of age; calculated using Schwartz equation for patients \<18 years of age.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subjects will receive prescribed dosage of metreleptin as indicated in the USPI Patients (males and females) ≤ 40 kg: 0.06mg/kg Male patients \> 40 kg: 2.5mg Female patients \> 40 kg: 5mg
Drug: Metreleptin
Subjects will receive prescribed dosage of metreleptin as indicated in the USPI
Evaluate the Immunogenicity Associated With Daily Subcutaneous (SC) Metreleptin Treatment in Patients With Congenital Generalized Lipodystrophy (CGL) or Acquired Generalized Lipodystrophy (AGL).
Anti-metreleptin, anti-human leptin (HuL) binding antidrug antibody (ADA) titers over time. Category of in vitro cell-based neutralizing antibody (NAb) activity to metreleptin, and titer in the receptor blocking (RB) NAb assay in ADA positive samples over time.
Time frame: 36 months
Assess 2 Methods of Measuring in Vitro NAb Activity to Metreleptin.
The percent (standard error) of patients with a positive result was compared for the RB NAb assay and the cell-based NAb assay over time.
Time frame: 36 months
Evaluate the Safety and Tolerability in Relation to the Development of or Absence of Anti-metreleptin and Anti-HuL Binding ADAs, and/or in Vitro NAb Activity to Metreleptin in Patients With CGL or AGL
Serious adverse events (SAEs), AEs leading to discontinuation, loss of response (as assessed by glycated hemoglobin (HbA1c) and serum triglycerides), severe infections and/or sepsis, standard laboratory tests, and vital signs over time.
Time frame: 36 months
Evaluate the Efficacy With Daily Metreleptin in Patients With GL
Change from Baseline in HbA1c over time.
Time frame: 36 months
Evaluate the Efficacy With Daily Metreleptin in Patients With GL
Percent change from Baseline in fasting triglycerides over time.
Time frame: 36 months
Patients were recruited from 4 centers in the US. Prescribing Investigators for this study were those who were certified under REMS for MYALEPT prescription and able to ensure prescription of metreleptin was as per the USPI.
| Milestone | Metreleptin |
|---|---|
| Started | 11 |
| Completed | 7 |
| Not completed | 4 |
The percent (standard error) of patients with a positive result was compared for the RB NAb assay and the cell-based NAb assay over time.
| Participants | Metreleptin |
|---|---|
| NAb cell based+ | 1 |
| NAb receptor blocking+ | 10 |
Serious adverse events (SAEs), AEs leading to discontinuation, loss of response (as assessed by glycated hemoglobin (HbA1c) and serum triglycerides), severe infections and/or sepsis, standard laboratory tests, and vital signs over time.
| Participants | Metreleptin |
|---|---|
| All AE — AEs leading to discomntinuation | 11 |
| SAE — AEs leading to discomntinuation | 7 |
| AEs leading to discontinuation — AEs leading to discomntinuation | 1 |
| Severe infections and/or sepsis — AEs leading to discomntinuation | 2 |
| Blood Triglycerides increased — AEs leading to discomntinuation | 3 |
| Glycosylated haemoglobin increased — AEs leading to discomntinuation | 1 |
Change from Baseline in HbA1c over time.
| Percentage | Metreleptin |
|---|---|
| Absolute change in HbA1c from baseline to Month 12 | -0.3 (-6.5 to 2.3) |
| Absolute change in HbA1c from baseline to Month 24 | -0.5 (-7.2 to 1.8) |
| Absolute change in HbA1c from baseline to Month 36 | -0.7 (-7.3 to 1.9) |
Anti-metreleptin, anti-human leptin (HuL) binding antidrug antibody (ADA) titers over time. Category of in vitro cell-based neutralizing antibody (NAb) activity to metreleptin, and titer in the receptor blocking (RB) NAb assay in ADA positive samples over time.
| Participants | Metreleptin |
|---|---|
| ADA positive — ADA+ | 10 |
| NAb cell based + — ADA+ | 1 |
| NAb receptor blocking+ — ADA+ | 10 |
Percent change from Baseline in fasting triglycerides over time.
| Percent Change | Metreleptin |
|---|---|
| Precent change in triglycerides from baseline to Month 12 | -49.68 (-91.26 to 174.77) |
| Precent change in triglycerides from baseline to Month 24 | -46.72 (-92.25 to 593.01) |
| Precent change in triglycerides from baseline to Month 36 | -58.12 (-98.55 to -22.16) |
Collected over From signing the ICF up until either the last scheduled Follow-up Visit (36 months) or at least 4 weeks after the last dose of study medication in the case of an early termination. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Metreleptin | 1/11 (9.1%) | 7/11 (63.6%) | 11/11 (100%) |
| Event | Metreleptin |
|---|---|
| COVID-19Infections and infestations | 1/11 |
| Autoimmune hepatitisHepatobiliary disorders | 1/11 |
| Pancreatitis relapsingGastrointestinal disorders | 1/11 |
| OsteomyelitisInfections and infestations | 1/11 |
| SepsisInfections and infestations | 1/11 |
| Abortion spontaneousPregnancy, puerperium and perinatal conditions | 1/11 |
| ConstipationGastrointestinal disorders | 1/11 |
| Event | Metreleptin |
|---|---|
| COVID-19Infections and infestations | 4/11 |
| Abdominal painGastrointestinal disorders | 3/11 |
| Abdominal painGastrointestinal disorders | 3/11 |
| Maternal exposure during pregnancyInjury, poisoning and procedural complications | 2/11 |
| Blood triglycerides increasedInvestigations | 2/11 |
| AnemiaBlood and lymphatic system disorders | 1/11 |
| Cardiac failureCardiac disorders | 1/11 |
| Cerumen impactionEar and labyrinth disorders | 1/11 |
| CataractEye disorders | 1/11 |
| DiarrheaGeneral disorders | 1/11 |
| Age, Categorical(Participants) | Metreleptin |
|---|---|
| <=18 years | 3 |
| Between 18 and 65 years | 8 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Metreleptin |
|---|---|
| Female | 9 |
| Male | 2 |
| Race (NIH/OMB)(Participants) | Metreleptin |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 1 |
| White | 9 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
| Ethnicity (NIH/OMB)(Participants) | Metreleptin |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 9 |
| Unknown or Not Reported | 0 |
| Lipodystrophy diagnosis(Participants) | Metreleptin |
|---|---|
| Congenital Generalised Lipodystrophy | 6 |
| Acquired Generalised Lipodystrophy | 5 |
| Medical History(Participants) | Metreleptin |
|---|---|
| Hypertriglyceridemia | 8 |
| Diabetes | 7 |
| Pancreatitis | 5 |
| Hypertension | 4 |
| Hepatic steatosis | 3 |
| Polycystic ovary syndrome | 2 |
| Proteinuric | 2 |
| Hepatic cirrhosis | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — only IPD that underly results in a publication
Supporting information: Study protocol, Informed consent form
Time frame: A copy of data generated at the study site will be provided to individual sites after the overall publication by the sponsor is completed and submitted
Ran from Nov 2018 to primary completion in Oct 2024, 71 months.
Results were first posted in Nov 2025, 1 year after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Nov 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Metreleptin
Aegerion Pharmaceuticals, Inc. →