CompletedPhase 2

Setmelanotide in a Single Patient With Partial Lipodystrophy

United States1 participantsStarted 2017-08-15

Plain-language 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.

Who can participate

Age range15 Years – 15 Years

SexFEMALE

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Inclusion criteria

✓. Signed written informed consent
✓. Ability to comply with visits and procedures required by program.
✓. Has physician-confirmed partial lipodystrophy and the following characteristics:
✓. Female, under the age of 18 years

Exclusion criteria

✕. 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.

What they're measuring

Fasting Triglycerides (TG) levels

Timeframe: 12 to 20 weeks

Trial details

NCT IDNCT03262610

SponsorRhythm Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-01-31

View on ClinicalTrials.gov More Hypertriglyceridemia trials