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Setmelanotide in a Single Patient With Partial Lipodystrophy

Primary Purpose

Hypertriglyceridemia, Obesity

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Setmelanotide
Sponsored by
Rhythm Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hypertriglyceridemia

Eligibility Criteria

15 Years - 15 Years (Child)FemaleDoes not accept healthy volunteers

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.

Sites / Locations

  • University of Michigan

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Setmelanotide daily subcutaneous injection

Arm Description

Up to 18 weeks setmelanotide treatment.

Outcomes

Primary Outcome Measures

Fasting Triglycerides (TG) levels
The mean change from baseline in fasting triglycerides after setmelanotide treatment.

Secondary Outcome Measures

Full Information

First Posted
August 22, 2017
Last Updated
December 13, 2021
Sponsor
Rhythm Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03262610
Brief Title
Setmelanotide in a Single Patient With Partial Lipodystrophy
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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2021
Overall Recruitment Status
Completed
Study Start Date
August 15, 2017 (Actual)
Primary Completion Date
January 31, 2018 (Actual)
Study Completion Date
January 31, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Rhythm Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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 hypertriglyceridemia leading to recurrent bouts of pancreatitis.
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 leading to recurrent pancreatitis despite ongoing plasmapheresis treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypertriglyceridemia, Obesity

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
1 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Setmelanotide daily subcutaneous injection
Arm Type
Experimental
Arm Description
Up to 18 weeks setmelanotide treatment.
Intervention Type
Drug
Intervention Name(s)
Setmelanotide
Other Intervention Name(s)
RM-493
Intervention Description
daily subcutaneous injection
Primary Outcome Measure Information:
Title
Fasting Triglycerides (TG) levels
Description
The mean change from baseline in fasting triglycerides after setmelanotide treatment.
Time Frame
12 to 20 weeks

10. Eligibility

Sex
Female
Gender Based
Yes
Gender Eligibility Description
This is a single patient study
Minimum Age & Unit of Time
15 Years
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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: 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 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Linda Shapiro, M.D., Ph.D.
Organizational Affiliation
Chief Medical Officer, Rhythm Pharmaceuticals, Inc
Official's Role
Study Chair
Facility Information:
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States

12. IPD Sharing Statement

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Setmelanotide in a Single Patient With Partial Lipodystrophy

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