Setmelanotide in a Single Patient With Partial Lipodystrophy

Obesity Clinical Trial

Official title:

Expanded-access for the Use of Setmelanotide in a Single Patient With Partial Lipodystrophy (LD) Associated With Leptin Deficiency and Multiple Autoimmune Diseases

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03262610
• Other study ID #: RM-493-019
• Status: Completed
• Phase: Phase 2
• Start date: August 15, 2017
• Est. completion date: January 31, 2018

Study information

• Verified date: December 2021
• Source: Rhythm Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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 leading to recurrent pancreatitis despite ongoing plasmapheresis treatment.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: January 31, 2018
• Est. primary completion date: January 31, 2018
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 15 Years to 15 Years

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.

Related Conditions & MeSH terms

• Hypertriglyceridemia
• Lipodystrophy
• Obesity

Intervention

Drug:
• Setmelanotide
daily subcutaneous injection

Locations

Country	Name	City	State
United States	University of Michigan	Ann Arbor	Michigan

Sponsors (1)

Lead Sponsor	Collaborator
Rhythm Pharmaceuticals, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Fasting Triglycerides (TG) levels	The mean change from baseline in fasting triglycerides after setmelanotide treatment.	12 to 20 weeks