Expanded-access for the Use of Setmelanotide in a Single Patient With Partial Lipodystrophy (LD) Associated With Leptin Deficiency and Multiple Autoimmune Diseases
| Registry | NCT03262610 |
|---|---|
| Status | Completed |
| Phase | Phase 2 |
| Study type | Interventional; na, single group, masking: none, treatment |
| Enrollment | 1 (Actual) |
| Sponsor | Rhythm Pharmaceuticals, Inc. (Industry) |
| Conditions | Hypertriglyceridemia, Obesity |
| Interventions | Setmelanotide (Drug) |
| Dates | start 2017-08-15, primary completion 2018-01-31, completion 2018-01-31, first posted 2017-08-25, last updated 2022-01-04 |
| Locations | 1 site in United States |
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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.
| Arm | Type | Interventions | Description |
|---|---|---|---|
| Setmelanotide daily subcutaneous injection | Experimental | Drug: Setmelanotide | Up to 18 weeks setmelanotide treatment. |
Sex: Female, ages: 15 Years to 15 Years
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.
| Facility | City | Country | Status |
|---|---|---|---|
| University of Michigan | Ann Arbor, Michigan | United States |