None listed
Conditions
Interventions
Treatment Arm A Type of group
Treatment Arm A:
This arm consists of 3 sequential 8-week periods in which patients receive the following treatments:
- Placebo
- Low-dose REGN4461
- High-dose REGN4461.
Group name:Treatment Arm B Type of group
Treatment Arm B:
This arm consists of 3 sequential 8-week periods in which patients receive the following treatments:
- Low-dose REGN4461
- High-dose REGN4461
- High-dose REGN4461
Sponsors
Regeneron Pharmaceuticals Inc,
Eligibility
Inclusion criteria
Inclusion criteria: 1. Male or female patients, ≥12 years of age at the screening visit (or lower limit of age approved by Health Authority, EC, and IRB). 2. Clinical diagnosis of congenital or acquired GLD based on history, physical examination, body composition, and metabolic status as described in Brown, 2016. 3. Presence of one or both of the following metabolic abnormalities at screening: a. HbA1c ≥ 7% OR b. Fasting TG ≥250 mg/dL 4. Generally stable diet (based on patient’s recall) and medication regimen (that optimizes treatment for their metabolic disease) for at least 3 months prior to the screening visit. 5. Willing and able to comply with clinic visits and study related procedures (Note: patients incapable of completing PRO assessments or unable to undergo MRI will not be excluded). 6. Provide informed consent signed by study patient or legally acceptable representative.
Exclusion criteria
Exclusion criteria: 1. Treatment with metreleptin within 1 month of the screening visit. 2. Patients with clinically significant hematologic abnormalities such as neutropenia, lymphopenia, or lymphadenopathy at screening. 3. Treatment with over-the-counter or prescription medications for weight loss within 3 months prior to the screening visit. 4. Any malignancy, eg, lymphoma, within the past 1 year, prior to screening visit except for fully treated basal cell or squamous epithelial cell carcinomas of the skin or carcinoma in situ of the cervix or anus. 5. Estimated GFR of <30 mL/min/1.73 m2 based on CKDEpi equation at screening. 6. History of heart failure hospitalization, diagnosis of a myocardial infarction, stroke, transient ischemic attack, unstable angina, percutaneous or surgical revascularization procedure (coronary, carotid, or peripheral vascular), or intracardiac device placement (eg, pacemaker) within 3 months before the screening visit. 7. Advanced heart failure (NYHA Class 3 to 4) or severe and uncontrolled hypertension at screening. 8. History of HIV or HIV seropositivity at screening visit 9. Uncontrolled infection with hepatitis B or hepatitis C infection, or known active tuberculosis at screening. 10. Patient has a history of an anaphylactic reaction to monoclonal antibody therapeutics or investigational products (IP).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Outcome name:fasting TG, fasting glucose, HbA1c, WMG Measure:glycemic endpoint Timepoints:within the first 2 months following initiation | — |
Secondary
| Measure | Time frame |
|---|---|
| Outcome name:FAS (Full analysis set) Measure:HbA1c endpoint Timepoints:44 Weeks ; Outcome name:FAS (Full analysis set) Measure:fasting glucose and fasting TG Timepoints:44 Weeks ; Outcome name:FAS (Full analysis set) Measure:WMG and MMTT Timepoints:16 Weeks | — |
Countries
Peru, Russian Federation, Turkey, United States
Contacts
Public ContactGiselle Vasquez
SYNEOS HEALTH PERU S.R.L.
Outcome results
None listed