Setmelanotide in a Single Patient With Partial Lipodystrophy

December 13, 2021 updated by: Rhythm Pharmaceuticals, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

1

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Michigan
      • Ann Arbor, Michigan, United States, 48105
        • University of Michigan

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

15 years to 15 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Female

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Setmelanotide daily subcutaneous injection
Up to 18 weeks setmelanotide treatment.
Drug: Setmelanotide
daily subcutaneous injection
Other Names:
  • RM-493

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Fasting Triglycerides (TG) levels
Time Frame: 12 to 20 weeks
The mean change from baseline in fasting triglycerides after setmelanotide treatment.
12 to 20 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Rhythm Pharmaceuticals, Inc.

Investigators

  • Study Chair: Linda Shapiro, M.D., Ph.D., Chief Medical Officer, Rhythm Pharmaceuticals, Inc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 15, 2017

Primary Completion (Actual)

January 31, 2018

Study Completion (Actual)

January 31, 2018

Study Registration Dates

First Submitted

August 22, 2017

First Submitted That Met QC Criteria

August 22, 2017

First Posted (Actual)

August 25, 2017

Study Record Updates

Last Update Posted (Actual)

January 4, 2022

Last Update Submitted That Met QC Criteria

December 13, 2021

Last Verified

December 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RM-493-019

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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