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CompletedNCT03262610Updated Jan 4, 2022

Setmelanotide in a Single Patient With Partial Lipodystrophy

A Phase 2 interventional study of Setmelanotide in Hypertriglyceridemia and Obesity, sponsored by Rhythm Pharmaceuticals, Inc.. Completed at 1 site in United States. Open to female participants aged 15 Years to 15 Years. Per ClinicalTrials.gov, last updated 2022-01-04.

Sponsored by Rhythm Pharmaceuticals, Inc. · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
1
Allocation
Not applicable
Ages
15 Years to 15 Years
Sex
Female
01

Study 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.

Read the 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.

02

Conditions studied

  • Hypertriglyceridemia
  • Obesity
03

Who can participate

Ages eligible
15 Years to 15 Years
Sexes eligible
Female
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
1 participant (actual)

Study arms

  • Experimental
    Setmelanotide daily subcutaneous injection

    Up to 18 weeks setmelanotide treatment.

    Drug: Setmelanotide

Interventions

  • DrugSetmelanotide

    daily subcutaneous injection

    Also known as: RM-493

05

What researchers measure

Primary outcomes

  1. Fasting Triglycerides (TG) levels

    The mean change from baseline in fasting triglycerides after setmelanotide treatment.

    Time frame: 12 to 20 weeks

06

Study locations

1 site
  • University of Michigan
    Ann Arbor, Michigan 48105, United States
07

Registry details

Key details

Study ID
NCT03262610
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Aug 25, 2017
Start date
Aug 15, 2017
Primary completion
Jan 31, 2018
Completion
Jan 31, 2018
Last update
Jan 4, 2022

Study contacts

Linda Shapiro, M.D., Ph.D.
study chair · Chief Medical Officer, Rhythm Pharmaceuticals, Inc

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Dec 2021. You cannot join it, but the record below documents what was studied.

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