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MYALEPTâ„¢ (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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Metreleptin | Drug | Subjects will receive prescribed dosage of metreleptin as indicated in the USPI |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 36 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Evaluate the Efficacy With Daily Metreleptin in Patients With GL | Change from Baseline in HbA1c over time. | 36 months |
| Evaluate the Efficacy With Daily Metreleptin in Patients With GL | Percent change from Baseline in fasting triglycerides over time. |
Inclusion Criteria:
Exclusion Criteria:
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.
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.
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| Name | Affiliation | Role |
|---|---|---|
| Janet Boylan | Aegerion Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Univ. Alabama-Birmingham | Birmingham | Alabama | 35294 | United States | ||
| Ochsner Clinic |
only IPD that underly results in a publication
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
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At Screening, consenting patients were assessed to ensure that they met eligibility criteria. The informed consent and health insurance portability and accountability act Authorization forms were signed prior to performing any Protocol-required procedures. The assessments conducted at the Screening Visit. When all Screening results were available, individuals were notified of their eligibility status.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Metreleptin | 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 |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Metreleptin | 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 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Secondary | 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. | Only ADA+ participants were analysed for neutralizing activity. | Posted | Count of Participants | Participants | 36 months |
|
|
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
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Metreleptin | 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 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID-19 | Infections and infestations | Non-systematic Assessment | COVID-19 infection, not related to metreleptin |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anemia | Blood and lymphatic system disorders | Non-systematic Assessment | Anemia, not related to metreleptin |
Following limitations may have occurred:
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Metreleptin Clinical Program Lead | Chiesi | +3905212791 | clinicaltrials_info@chiesi.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 8, 2018 | Oct 29, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 24, 2025 | Oct 29, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D052497 | Lipodystrophy, Congenital Generalized |
| D008060 | Lipodystrophy |
| ID | Term |
|---|---|
| D008052 | Lipid Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C415771 | metreleptin |
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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. |
| 36 months |
| 36 months |
| New Orleans |
| Louisiana |
| 70121 |
| United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| Endocrinology Research Associates | Columbus | Ohio | 43201 | United States |
| Ohio State University | Columbus | Ohio | 43203 | United States |
| Childrens Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| University Texas Southwestern INT | Dallas | Texas | 75390 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Lipodystrophy diagnosis | 11 GL: 6 Congenital Generalized Lipodystrophy, and 5 Acquired Generalized Lipodystrophy | Count of Participants | Participants |
|
| Medical History | Count of Participants | Participants |
|
| Participants |
|
|
| Secondary | 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. | Posted | Count of Participants | Participants | 36 months |
|
|
|
| Other Pre-specified | Evaluate the Efficacy With Daily Metreleptin in Patients With GL | Change from Baseline in HbA1c over time. | Only subjects who reached the visits (Month 12, 24 ,36) were included in the analysis | Posted | Median | Full Range | Percentage | 36 months |
|
|
|
| Primary | 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. | Only ADA positive participants were analysed for neutralizing activity. | Posted | Jul 2024 | Count of Participants | Participants | 36 months |
|
|
|
| Other Pre-specified | Evaluate the Efficacy With Daily Metreleptin in Patients With GL | Percent change from Baseline in fasting triglycerides over time. | Only subjects who reached the visits (Month 12, 24 ,36) were included in the analysis | Posted | Median | Full Range | Percent Change | 36 months |
|
|
|
| 1 |
| 11 |
| 7 |
| 11 |
| 11 |
| 11 |
|
| Autoimmune hepatitis | Hepatobiliary disorders | Non-systematic Assessment | Exacerbation of autoimmune hepatitis, not related to metreleptin |
|
| Pancreatitis relapsing | Gastrointestinal disorders | Non-systematic Assessment | Recurrent pancreatitis, not related to metreleptin |
|
| Osteomyelitis | Infections and infestations | Non-systematic Assessment | Osteomyelitis (fifth digit of left hand), not related to metreleptin |
|
| Sepsis | Infections and infestations | Non-systematic Assessment | Sepsis, not related to metreleptin |
|
| Abortion spontaneous | Pregnancy, puerperium and perinatal conditions | Non-systematic Assessment | Miscarriage, not related to metreleptin |
|
| Constipation | Gastrointestinal disorders | Non-systematic Assessment | Constipation, not related to metreleptin |
|
|
| Cardiac failure | Cardiac disorders | Non-systematic Assessment | Heart failure, not related to metreleptin |
|
| Cerumen impaction | Ear and labyrinth disorders | Non-systematic Assessment | Bilateral impacted cerumen, not related to metreleptin |
|
| Cataract | Eye disorders | Non-systematic Assessment | Bilateral cataracts, not related to metreleptin |
|
| Abdominal pain | Gastrointestinal disorders | Non-systematic Assessment | Abdominal pain, not related to metreleptin |
|
| Abdominal pain | Gastrointestinal disorders | Non-systematic Assessment |
|
| Diarrhea | General disorders | Non-systematic Assessment |
|
| Pancreatitis chronic | Gastrointestinal disorders | Non-systematic Assessment | Chronic pancreatitis, not related to metreleptin |
|
| Vomiting | Gastrointestinal disorders | Non-systematic Assessment | Vomiting, not related to metreleptin |
|
| Injection site haemorrhage | General disorders | Non-systematic Assessment | Very small blead of blood after one injection, not related to metreleptin |
|
| Peripheral swelling | General disorders | Non-systematic Assessment | Left leg swelling, not related to metreleptin |
|
| Ocular icterus | Hepatobiliary disorders | Non-systematic Assessment | Jaundice eyes, not related to metreleptin |
|
| Bacterial vaginosis | Infections and infestations | Non-systematic Assessment | Bacterial vaginosis, not related to metreleptin |
|
| Chlamydial infection | Infections and infestations | Non-systematic Assessment | Chlamydia, not related to metreleptin |
|
| COVID-19 | Infections and infestations | Non-systematic Assessment | COVID-19, not related to metreleptin |
|
| Croup infectious | Infections and infestations | Non-systematic Assessment | Croup, not related to metreleptin |
|
| Infectious mononucleosis | Infections and infestations | Non-systematic Assessment | Mononucleosis, not related to metreleptin |
|
| Influenza | Infections and infestations | Non-systematic Assessment | Flu, not related to metreleptin |
|
| Respiratory syncytial virus infection | Infections and infestations | Non-systematic Assessment | RSV infection, not related to metreleptin |
|
| Sinusitis | Infections and infestations | Non-systematic Assessment | Sinus infection, not related to metreleptin |
|
| Tonsilitis | Infections and infestations | Non-systematic Assessment | Tonsilitis, not related to metreleptin |
|
| Urinary tract infection | Infections and infestations | Non-systematic Assessment | Urinary tract infection, not related to metreleptin |
|
| Viral infection | Infections and infestations | Non-systematic Assessment | Viral illness, not related to metreleptin |
|
| Fall | Injury, poisoning and procedural complications | Non-systematic Assessment | Fall, not related to metreleptin |
|
| Foot fracture | Injury, poisoning and procedural complications | Non-systematic Assessment | Foot fracture, not related to metreleptin |
|
| Hand fracture | Injury, poisoning and procedural complications | Non-systematic Assessment | LEFT HAND FRACTURES IN 3 PLACES - HAND HIT WALL IN INDOOR SOCCER GAME, not related to metreleptin |
|
| Maternal exposure during pregnancy | Injury, poisoning and procedural complications | Non-systematic Assessment | Pregnancy |
|
| Skin laceration | Injury, poisoning and procedural complications | Non-systematic Assessment | STITCHES IN CHIN FOR LACERATION, not related to metreleptin |
|
| Blood bilirubin increased | Investigations | Non-systematic Assessment | Elevated bilirubin level, not related to metreleptin |
|
| Blood cholesterol increased | Investigations | Non-systematic Assessment | High cholesterol, not related to metreleptin |
|
| Blood pressure increased | Investigations | Non-systematic Assessment | Elevated blood pressure, not related to metreleptin |
|
| Blood triglycerides increased | Investigations | Non-systematic Assessment | Elevated triglyceride level, not related to metreleptin |
|
| Glycosylated haemoglobin increased | Investigations | Non-systematic Assessment | High HbA1c, not related to metreleptin |
|
| Liver function test increased | Investigations | Non-systematic Assessment | Increased liver function tests, not related to metreleptin |
|
| Hyperglycemia | Metabolism and nutrition disorders | Non-systematic Assessment | Hyperglycemia, not related to metreleptin |
|
| Hyperkalemia | Metabolism and nutrition disorders | Non-systematic Assessment | Hyperkalemia, not related to metreleptin |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | Non-systematic Assessment | Left shoulder pain, not related to metreleptin |
|
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Non-systematic Assessment | BASAL CELL CARCINOMA, MULTIPLE (RIGHT SHOULDER, RIGHT UPPER ARM, RIGHT LOWER BACK) NO SIZE PROVIDED, not related to metreleptin |
|
| Hypoesthesia | Nervous system disorders | Non-systematic Assessment | Increasing in leg numbness, not related to metreleptin |
|
| Motor dysfunction | Nervous system disorders | Non-systematic Assessment | Worsening motor dysfunction, not related to metreleptin |
|
| Neuropathy peripheral | Nervous system disorders | Non-systematic Assessment | Worsening neuropathy, not related to metreleptin |
|
| Anxiety | Psychiatric disorders | Non-systematic Assessment | Worsening of anxiety, not related to metreleptin |
|
| Insomnia | Psychiatric disorders | Non-systematic Assessment | Worsening of insomnia, not related to metreleptin |
|
| Mental status changes | Psychiatric disorders | Non-systematic Assessment | Altered mental state, not related to metreleptin |
|
| Stress | Psychiatric disorders | Non-systematic Assessment | Stress, not related to metreleptin |
|
| Acute kidney injury | Renal and urinary disorders | Non-systematic Assessment | Acute kidney injury, not related to metreleptin |
|
| Focal segmental glomerulosclerosis | Renal and urinary disorders | Non-systematic Assessment | FOCAL SEGMENTAL GLOMERULOSCLEROSIS, not related to metreleptin |
|
| Bladder spasm | Renal and urinary disorders | Non-systematic Assessment | Bladder spasms, not related to metreleptin |
|
| Renal impairment | Renal and urinary disorders | Non-systematic Assessment | DETERIORATING KIDNEY FUNCTION WITH RISING CREATININE, not related to metreleptin |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment | Nose bleed, not related to metreleptin |
|
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment | Sinus congestion, not related to metreleptin |
|
| Eczema | Skin and subcutaneous tissue disorders | Non-systematic Assessment | Intermittent scalp eczema, not related to metreleptin |
|
| Dermatitis | Skin and subcutaneous tissue disorders | Non-systematic Assessment | Skin rash - dermatitis unspecified |
|
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment | Rash on bilateral elbows, not related to metreleptin |
|
| Skin lesion | Skin and subcutaneous tissue disorders | Non-systematic Assessment | SKIN LESION EXCISION BY DERMATOLOGY - BENIGN, not related to metreleptin |
|
| Urticaria | Skin and subcutaneous tissue disorders | Non-systematic Assessment | Hives, not related to metreleptin |
|
| Vitiligo | Skin and subcutaneous tissue disorders | Non-systematic Assessment | Increased vitiligo in mouth, not related to metreleptin |
|
| Xanthoma | Skin and subcutaneous tissue disorders | Non-systematic Assessment | Eruptive xanthomas, not related to metreleptin |
|
| Abortion induced | Surgical and medical procedures | Non-systematic Assessment | Abortion induced, not related to metreleptin |
|
| Arteriovenous fistula | Vascular disorders | Non-systematic Assessment | Arteriovenous fistula, not related to metreleptin |
|
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| D012875 | Skin Diseases, Metabolic |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D052439 | Lipid Metabolism Disorders |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Title | Measurements |
|---|---|
| AEs leading to discomntinuation |
|
| Severe infections and/or sepsis |
|
| Blood Triglycerides increased |
|
| Glycosylated haemoglobin increased |
|
|
| Absolute change in HbA1c from baseline to Month 36 |
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|
| NAb receptor blocking+ |
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| Precent change in triglycerides from baseline to Month 36 |
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