Current partner codePEPTIDESDE
NCT03262610·Phase 2·INTERVENTIONAL

Setmelanotide in a Single Patient With Partial Lipodystrophy

Status

Completed

Phase

Phase 2

Enrollment

1

Locations

1

Results

Not posted

Publications

0

Study summary

What the protocol is testing.

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.

Full 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

Treatment arms and agents.

DRUG

Setmelanotide

daily subcutaneous injection

Timeline

From registration to results.

  1. First posted

    Aug 25, 2017

  2. Study start

    Aug 15, 2017

  3. Primary completion

    Jan 31, 2018

  4. Study completion

    Jan 31, 2018

  5. Results posted

    Not reported

  6. Registry updated

    Jan 4, 2022

Outcomes

What the study measures.

Primary outcomes

Fasting Triglycerides (TG) levels

Time frame · 12 to 20 weeks

The mean change from baseline in fasting triglycerides after setmelanotide treatment.

Secondary outcomes

Not reported in the indexed record.

Eligibility

Who can take part.

Minimum age
15 Years
Maximum age
15 Years
Sex
FEMALE
Healthy volunteers
No

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.

Study locations

1 registered sites.

United States. Showing up to 24 locations stored in the fast local snapshot.

University of Michigan

Ann Arbor, Michigan, United States

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

Related trials

More studies on Setmelanotide.

Related PeptideStat pages

Put the record in context.

Research pages describe evidence. Vendor pages, where available, describe independently tracked research-product listings and are not clinical recommendations.