Setmelanotide in a Single Patient With Partial Lipodystrophy

NCT03262610 | Completed Phase 2 FDA Drug

• 1 other identifier: RM-493-019
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

Hypertriglyceridemia; Obesity

Outcome Measures

Primary Outcomes (1)

• Fasting Triglycerides (TG) levels

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

  12 to 20 weeks

Study Arms (1)

Setmelanotide daily subcutaneous injection

EXPERIMENTAL

Up to 18 weeks setmelanotide treatment.

Drug: Setmelanotide

Interventions

Setmelanotide DRUG

daily subcutaneous injection

Also known as: RM-493

Setmelanotide daily subcutaneous injection

Eligibility Criteria

• Age: 15 Years - 15 Years
• Gender Eligibility Details: This is a single patient study
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Rhythm Pharmaceuticals, Inc.: lead

Study Sites (1)

University of Michigan

Ann Arbor, Michigan, 48105, United States

Location

MeSH Terms

Conditions

Hypertriglyceridemia; Obesity

Interventions

setmelanotide

Condition Hierarchy (Ancestors)

Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Overweight; Overnutrition; Nutrition Disorders; Body Weight; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Linda Shapiro, M.D., Ph.D.

  Chief Medical Officer, Rhythm Pharmaceuticals, Inc

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 22, 2017
• First Posted: August 25, 2017
• Study Start: August 15, 2017
• Primary Completion: January 31, 2018
• Study Completion: January 31, 2018
• Last Updated: January 4, 2022

Record last verified: 2021-12

Locations

US (1)