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CLINICAL PROTOCOL to Investigate the Long-term Safety and Efficacy of Metreleptin in Various Forms of Partial Lipodystrophy
Phase 2 trial testing Metreleptin in Familial Partial Lipodystrophy in 11 participants. Completed in 14 November 2019.
| Lead sponsor | University of Michigan |
|---|---|
| Phase | Phase 2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 11 |
| Start date | 29 September 2015 |
| Primary completion | 14 November 2019 |
| Estimated completion | 14 November 2019 |
| Sites | 1 location across United States |
University of Michigan
5 and older, any sex, with Familial Partial Lipodystrophy or Nonalcoholic Steatohepatitis. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Fasting triglyceride levels after 5 years on metreleptin, measured in mg/dL are compared to fasting triglyceride levels at baseline). The median percent increase (positive numbers) or decrease from baseline is shown, along with the full range.
| Group | Value | 95% CI |
|---|---|---|
| Metreleptin | 55.76 | -66.79 – 203.80 |
Fasting hemoglobin A1c levels after 5 years on metreleptin, measured in % are compared to fasting Hemoglobin levels at baseline). Thus, for example, a change from 10% to 5% would be a -50% change (or 50% decrease). The median percent increase (positive numbers) or decrease from baseline is shown, along with the full range.
| Group | Value | 95% CI |
|---|---|---|
| Metreleptin | 2.55 | -27.52 – 25.00 |
Time frame: 5 years (from baseline visit to year 5 study visit or date of study participant early termination). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Metreleptin |
|---|---|---|
| pleural effusion | Respiratory, thoracic and mediastinal disorders | — |
| anterior cervical discectomy | Surgical and medical procedures | — |
| labia majora dermatofibrosarcoma protuberans | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | — |
| hyperglycemia | Metabolism and nutrition disorders | — |
| onset of type 1 diabetes | Endocrine disorders | — |
| Onset of hypertriglyceridemia | Metabolism and nutrition disorders | — |
| autoimmune hepatitis | Hepatobiliary disorders | — |
| diabetic ketoacidosis | Endocrine disorders | — |
| hypokalemia | Endocrine disorders | — |
| abdominal pain | Gastrointestinal disorders | — |
| pancreatitis | Endocrine disorders | — |
| metreleptin neutralizing antibodies | Immune system disorders | — |
| acute hypoxia | Respiratory, thoracic and mediastinal disorders | — |
| left shoulder pain | Musculoskeletal and connective tissue disorders | — |
| nonepilogenic seizures | Nervous system disorders | — |
| clinical sepsis | Infections and infestations | — |
| splenic flexure colitis fluid collection | Gastrointestinal disorders | — |
| multiple rib fractures | Musculoskeletal and connective tissue disorders | — |
| splenic laceration | Gastrointestinal disorders | — |
| uterine bleeding | Reproductive system and breast disorders | — |
| shortness of breath | Respiratory, thoracic and mediastinal disorders | — |
| dizziness | Nervous system disorders | — |
| chest pain with elevated troponin | Cardiac disorders | — |
| atrial flutter | Cardiac disorders | — |
| sudden cardiac death | Cardiac disorders | — |
| Reaction | System | Metreleptin |
|---|---|---|
| cold | Infections and infestations | — |
| urinary tract infection | Infections and infestations | — |
| upper respiratory tract infection | Infections and infestations | — |
| abdominal pain | Gastrointestinal disorders | — |
| worsening depression | Psychiatric disorders | — |
| worsening of chronic back pain | Musculoskeletal and connective tissue disorders | — |
| extremity weakness | Musculoskeletal and connective tissue disorders | — |
| cellutitus | Infections and infestations | — |
| reticular rash | Skin and subcutaneous tissue disorders | — |
| hypoglycemia | Endocrine disorders | — |
| bronchitis | Infections and infestations | — |
| elevated blood platelets | Blood and lymphatic system disorders | — |
| hyperesthesia | Nervous system disorders | — |
| sinus infection | Infections and infestations | — |
| weight loss | Endocrine disorders | — |
| elevated liver function tests | Metabolism and nutrition disorders | — |
| acid reflux | Gastrointestinal disorders | — |
| kidney stone | Renal and urinary disorders | — |
| strep throat | Infections and infestations | — |
| nausea | Gastrointestinal disorders | — |
| headache | Nervous system disorders | — |
| worsening vitamin D deficiency | Endocrine disorders | — |
| worsening imbalance | Nervous system disorders | — |
| worsening diabetes | Endocrine disorders | — |
| worsening weakness | Musculoskeletal and connective tissue disorders | — |
| vomiting | Gastrointestinal disorders | — |
| increase in appetite | Endocrine disorders | — |
| cardiac chest pain | Cardiac disorders | — |
| lung nodule | Respiratory, thoracic and mediastinal disorders | — |
| knee pain | Musculoskeletal and connective tissue disorders | — |
| vaginal spotting | Reproductive system and breast disorders | — |
| Spinal pain | Musculoskeletal and connective tissue disorders | — |
| vaginal yeast infection | Infections and infestations | — |
| elevated low density cholesterol | Metabolism and nutrition disorders | — |
| worsening of restless leg syndrome | Musculoskeletal and connective tissue disorders | — |
| low blood iron level | Endocrine disorders | — |
| infected tooth | Infections and infestations | — |
| kidney infection | Infections and infestations | — |
| thyroid nodules | Metabolism and nutrition disorders | — |
| intermittent suicidal ideation | Psychiatric disorders | — |
Most-reported serious reactions: pleural effusion, anterior cervical discectomy, labia majora dermatofibrosarcoma protuberans, hyperglycemia, onset of type 1 diabetes, Onset of hypertriglyceridemia, autoimmune hepatitis, diabetic ketoacidosis.
Data from ClinicalTrials.gov NCT02654977 adverse events section.
The study investigators' aim is to determine the long term safety and efficacy of Metreleptin (Myalept,) in promoting amelioration of metabolic abnormalities in patients with all forms of partial lipodystrophy. Patients will be offered this protocol under the following condition: Subjects have completed University of Michigan research protocol MB002-014 and have shown improved clinical benefit as judged by clinical criteria set forth in this protocol.
1 peer-reviewed publication reference this trial (live from Europe PMC):
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