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

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03262610

Enrollment

Registered

2017-08-25

Start date

2017-08-15

Completion date

2018-01-31

Last updated

2022-01-04

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hypertriglyceridemia, Obesity

Brief 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.

Interventions

DRUGSetmelanotide

daily subcutaneous injection

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

FEMALE

Age

15 Years to 15 Years

Healthy volunteers

Inclusion criteria

1. Signed written informed consent 2. Ability to comply with visits and procedures required by program. 3. Has physician-confirmed partial lipodystrophy and the following characteristics: * Has atypical lipodystrophy. * Has presence of neutralizing antibody to metreleptin * Patient has life threatening hypertriglyceridemia and has had \>8 episodes of pancreatitis, requiring weekly plasmapheresis * Has Type 1 diabetes mellitus with HbA1c \> 10%. 4. Female, under the age of 18 years

Exclusion criteria

1. Diagnosis of schizophrenia, bipolar disorder, personality disorder or other Diagnostic and Statistical Manual of Mental Disorders (DSM-III) disorders that the investigator believes will interfere significantly with study compliance. Neurocognitive disorders affecting ability to consent will not be disqualifying as long as an appropriate guardian able to give consent has been appointed. 2. History or close family history (parents or siblings) of skin cancer or melanoma, or patient history of ocular-cutaneous albinism. 3. Significant dermatologic findings relating to melanoma or pre-melanoma skin lesions, determined as part of a screening comprehensive skin evaluation performed by a qualified dermatologist. 4. Significant hypersensitivity to study drug. 5. Inability to comply with QD injection regimen.

Design outcomes

Primary

Measure	Time frame	Description
Fasting Triglycerides (TG) levels	12 to 20 weeks	The mean change from baseline in fasting triglycerides after setmelanotide treatment.

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 18, 2026