DRUG
Setmelanotide
daily subcutaneous injection
Status
Completed
Phase
Phase 2
Enrollment
1
Locations
1
Results
Not posted
Publications
0
Study 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.
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
DRUG
daily subcutaneous injection
Timeline
First posted
Aug 25, 2017
Study start
Aug 15, 2017
Primary completion
Jan 31, 2018
Study completion
Jan 31, 2018
Results posted
Not reported
Registry updated
Jan 4, 2022
Outcomes
Fasting Triglycerides (TG) levels
Time frame · 12 to 20 weeks
The mean change from baseline in fasting triglycerides after setmelanotide treatment.
Not reported in the indexed record.
Eligibility
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
United States. Showing up to 24 locations stored in the fast local snapshot.
University of Michigan
Ann Arbor, Michigan, United States
Publications
No PMID-linked publications were present in this registry snapshot.
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