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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT03262610 |
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Date of registration:
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22/08/2017 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Setemelanotide in a Single Patient With Partial Lipodystrophy
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Scientific title:
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Expanded-access for the Use of Setemelanotide in a Single Patient With Partial Lipodystrophy (LD) Associated With Leptin Deficiency and Multiple Autoimmune Diseases |
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Date of first enrolment:
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February 17, 2017 |
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Target sample size:
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Recruitment status: |
No longer available |
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URL:
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https://clinicaltrials.gov/show/NCT03262610 |
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Study type:
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Expanded Access |
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Study design:
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Phase:
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Countries of recruitment
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United States
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Contacts
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Name:
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Liz Stoner |
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Address:
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Telephone:
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Email:
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Affiliation:
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Rhythm Pharmceuticals, Inc |
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Key inclusion & exclusion criteria
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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 plasmapaheresis
- Has Type 1 diabetes melltus with HbA1c > 10%. Age and Reproductive Status
4. Female, under the the age of 18 years • The patient has primary amenorrhea at this
time
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. Any concerning lesions identified during the screening
period will be biopsied and results known to be benign prior to enrollment. If the
pre-treatment biopsy results are of concern, the patient may need to be excluded from
the study.
4. Significant hypersensitivity to study drug. 5. Inability to comply with QD
injection regimen.
Age minimum:
15 Years
Age maximum:
15 Years
Gender:
Female
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Health Condition(s) or Problem(s) studied
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Hypertriglyceridemia
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Intervention(s)
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Drug: Setmelanotide
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Secondary ID(s)
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RM-493-019
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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