Status:
COMPLETED
Setmelanotide in a Single Patient With Partial Lipodystrophy
Lead Sponsor:
Rhythm Pharmaceuticals, Inc.
Conditions:
Hypertriglyceridemia
Obesity
Eligibility:
FEMALE
15-15 years
Phase:
PHASE2
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 hypertriglyce...
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 l...
Eligibility Criteria
Inclusion
- Signed written informed consent
- Ability to comply with visits and procedures required by program.
- 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%.
- Female, under the age of 18 years
Exclusion
- 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.
- History or close family history (parents or siblings) of skin cancer or melanoma, or patient history of ocular-cutaneous albinism.
- 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.
- Significant hypersensitivity to study drug.
- Inability to comply with QD injection regimen.
Key Trial Info
Start Date :
August 15 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 31 2018
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT03262610
Start Date
August 15 2017
End Date
January 31 2018
Last Update
January 4 2022
Active Locations (1)
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1
University of Michigan
Ann Arbor, Michigan, United States, 48105