NCT03262610

Setmelanotide in a Single Patient With Partial Lipodystrophy

Expanded-access for the Use of Setmelanotide in a Single Patient With Partial Lipodystrophy (LD) Associated With Leptin Deficiency and Multiple Autoimmune Diseases

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Rhythm Pharmaceuticals, Inc. · Industry
• Sex: Female
• Age: 15 Years – 15 Years
• Healthy volunteers: Not accepted

Summary

The objective of this single patient study is to assess the safety and efficacy of setmelanotide as a treatment of severe metabolic abnormalities resulting from LD, especially refractory hypertriglyceridemia leading to recurrent bouts of pancreatitis.

Detailed description

This is a single-patient study to assess the safety and efficacy of setmelanotide in a unique patient with partial LD and severe metabolic abnormalities, most notably refractory hypertriglyceridemia leading to recurrent pancreatitis despite ongoing plasmapheresis treatment.

Conditions

• Hypertriglyceridemia
• Obesity

Interventions

Type	Name	Description
DRUG	Setmelanotide	daily subcutaneous injection

Timeline

• Start date: 2017-08-15
• Primary completion: 2018-01-31
• Completion: 2018-01-31
• First posted: 2017-08-25
• Last updated: 2022-01-04

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study