Generalized LipodystrophyPhase 4NCT04026178
Immunogenicity of Metreleptin in Patients With Generalized Lipodystrophy
A Phase 4 study testing Metreleptin in people with Generalized Lipodystrophy, run by Aegerion Pharmaceuticals, Inc. at 8 sites in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Generalized Lipodystrophy
- What is being tested?
- Metreleptin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 8 sitesUnited States
- Study dates
- 14 Nov 2018to 31 Oct 2024
- Phase
- Phase 4
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Provision of informed consent prior to any study specific procedures. If <18 years of age, has a parent or guardian able to read, understand, and sign the Informed Consent Form (ICF) and a Child Assent form, communicate with the Investigator, and understand and comply with protocol requirements. Adolescent patients must also read and understand the Child Assent Form. If the child is too young or unable to read, then the Child Assent form must be explained to the child.
- Female and/or male patients ≥1 years of age.
- Physician-confirmed diagnosis of congenital or acquired generalized lipodystrophy and will begin treatment with MYALEPT for the first time.
- Negative pregnancy test (urine or serum) for female patients of childbearing potential.
- Female patients of childbearing potential must be 1 year postmenopausal, surgically sterile, or be willing to use an acceptable method of contraception (an acceptable method of contraception is defined as a barrier method in conjunction with a spermicide) for the duration of the study (from the time they sign consent). In addition, oral contraceptives, approved contraceptive implant, long-term injectable contraception, intrauterine device, or tubal ligation are allowed. Oral contraception alone is not acceptable; additional barrier methods in conjunction with spermicide must be used.
- Male patients must be surgically sterile or be willing to use an acceptable method of contraception (defined as barrier methods in conjunction with spermicides) for the duration of the study (from the time they sign consent).
- Patients who are blood donors should not donate blood during the study and for 3 months following their last dose of metreleptin.
Who cannot join
- Involvement in the planning and/or conduct of the study (applies to both Aegerion staff and/or staff at the study site.)
- Previous treatment with metreleptin.
- Participation in another clinical study with an investigational product during the last 6 months.
- Patients with prior severe hypersensitivity reactions to metreleptin or to any of the product components.
- Known to have tested positive for human immunodeficiency virus, are immunocompromised, or are receiving immunomodulatory drugs.
- Known history of drug or alcohol abuse within 1 year of screening.
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- Creatinine clearance <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.
- For women only - currently pregnant (confirmed with positive pregnancy test) or breast-feeding.
- Any condition where, in the opinion of the Investigator, participation in this study may pose a significant risk to the patient or could render the patient unable to successfully complete the study.
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
University of Michigan
Ann Arbor, Michigan, 48109
Univ. Alabama-Birmingham
Birmingham, Alabama, 35294
Endocrinology Research Associates
Columbus, Ohio, 43201
Ohio State University
Columbus, Ohio, 43203
University Texas Southwestern INT
Dallas, Texas, 75390
Ochsner Clinic
New Orleans, Louisiana, 70121
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Seattle Children's Hospital
Seattle, Washington, 98105
About this study
Official title: A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily SC Metreleptin Treatment in Patients With Generalized Lipodystrophy
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.
- Conditions
- Generalized Lipodystrophy
- Being tested
- Metreleptin
- Keywords
- Immunogenicity, Antibodies, Lipodystrophy
- Medical subject headings
- Lipodystrophy, Congenital Generalized, Lipodystrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetreleptinDrug
Subjects will receive prescribed dosage of metreleptin as indicated in the USPI
| Study group | Role | What they receive |
|---|---|---|
| Metreleptin | Experimental | 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 Receives: Drug: Metreleptin |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 36 months
Secondary outcomes (2)
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.
Measured: 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.
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jul 2019
- Study start14 Nov 2018
- Primary completion31 Oct 2024
- Study completion31 Oct 2024
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 Generalized LipodystrophyStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Janet Boylan, Study director, Aegerion Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04026178
- Sponsor's protocol ID
- AEGR-734-401
- Status last verified
- Nov 2025
- First submitted
- 30 Oct 2018
- Last updated
- 26 Nov 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: only IPD that underly results in a publication
Study at a glance
From the registry record, last updated by the sponsor on 26 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 36-Month, Multicenter, Open Label Phase 4 Study to Evaluate the Immunogenicity of Daily SC Metreleptin Treatment in Patients With Generalized Lipodystrophy
- ClinicalTrials.gov ID
- NCT04026178
- Acronym / sponsor ID
- AEGR-734-401
- Conditions
- Generalized Lipodystrophy
- Keywords
- Immunogenicity, Antibodies, Lipodystrophy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Aegerion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Nov 2018 (actual)
- Primary completion
- 31 Oct 2024 (actual)
- Study completion
- 31 Oct 2024 (actual)
- First posted
- 19 Jul 2019 (actual)
- Results
- Posted 26 Nov 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Metreleptin | Experimental | Drug: MetreleptinSubjects 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 |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Provision of informed consent prior to any study specific procedures. If <18 years of age, has a parent or guardian able to read, understand, and sign the Informed Consent Form (ICF) and a Child Assent form, communicate with the Investigator, and understand and comply with protocol requirements. Adolescent patients must also read and understand the Child Assent Form. If the child is too young or unable to read, then the Child Assent form must be explained to the child.
- Female and/or male patients ≥1 years of age.
- Physician-confirmed diagnosis of congenital or acquired generalized lipodystrophy and will begin treatment with MYALEPT for the first time.
- Negative pregnancy test (urine or serum) for female patients of childbearing potential.
- Female patients of childbearing potential must be 1 year postmenopausal, surgically sterile, or be willing to use an acceptable method of contraception (an acceptable method of contraception is defined as a barrier method in conjunction with a spermicide) for the duration of the study (from the time they sign consent). In addition, oral contraceptives, approved contraceptive implant, long-term injectable contraception, intrauterine device, or tubal ligation are allowed. Oral contraception alone is not acceptable; additional barrier methods in conjunction with spermicide must be used.
Key exclusion criteria (10 listed)
- Involvement in the planning and/or conduct of the study (applies to both Aegerion staff and/or staff at the study site.)
- Previous treatment with metreleptin.
- Participation in another clinical study with an investigational product during the last 6 months.
- Patients with prior severe hypersensitivity reactions to metreleptin or to any of the product components.
- Known to have tested positive for human immunodeficiency virus, are immunocompromised, or are receiving immunomodulatory drugs.
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 |
|---|---|---|
| MetreleptinSubjects will receive prescribed dosage of metreleptin as indicated in the USPI | Drug | Metreleptin |
Primary outcome
Evaluate the Immunogenicity Associated With Daily Subcutaneous (SC) Metreleptin Treatment in Patients With Congenital Generalized Lipodystrophy (CGL) or Acquired Generalized Lipodystrophy (AGL).
Time frame: 36 months
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.
2 secondary outcomes
Assess 2 Methods of Measuring in Vitro NAb Activity to Metreleptin.
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
Time frame: 36 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Nov 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. only IPD that underly results in a publication
Related trials for Generalized Lipodystrophy
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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.
Janet Boylan study director
Aegerion Pharmaceuticals, Inc.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Metreleptin in people with Generalized Lipodystrophy, run by Aegerion Pharmaceuticals, Inc. at 8 sites 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 7 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 26 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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 26 Nov 2025). 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?
Evaluate the Immunogenicity Associated With Daily Subcutaneous (SC) Metreleptin Treatment in Patients With Congenital Generalized Lipodystrophy (CGL) or Acquired Generalized Lipodystrophy (AGL). (time frame: 36 months). 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 26 Nov 2025.