Patients with MSL for Lipomatosis, Multiple Symmetrical

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
University of Michigan, Ann Arbor, MI
Lipomatosis, Multiple Symmetrical+2 More
Metreleptin - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

Patients (homozygous MFN2 [gene that provides instructions to produce the Mitofusin 2 protein] R707W) will be treated with Metreleptin, and effects on body composition, metabolic parameters and safety will be assessed over a 6 month intervention period.

Eligible Conditions

  • Lipomatosis, Multiple Symmetrical

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Lipomatosis, Multiple Symmetrical

Study Objectives

2 Primary · 0 Secondary · Reporting Duration: Baseline, Week 24

Baseline, Week 24
Change in total adiposity
Change in truncal adiposity

Trial Safety

Safety Progress

1 of 3

Other trials for Lipomatosis, Multiple Symmetrical

Side Effects for

Metreleptin
15%Musculoskeletal pain
12%Weight loss
10%Hypoglycemia
9%Nausea
8%Abdominal pain
7%Infection
7%Fatigue
6%tumor, benign
6%Anemia
6%Anxiety
5%Diarrhea
5%Insomnia
5%Constipation
4%Depression
4%Headache
4%Joins pain
4%Decreased appetite
4%Iron deficiency
2%Exacerbations of heart failure
2%Pneumonia
1%Abdominal pain requiring hospitalization
1%Osteomyelitis
1%Acute exacerbation of pancreatitis
1%Group B streptococcus bacteremia
1%Ovarian cyst requiring bilateral oophorectomy and total abdominal hysterectomy
1%Cellulitis
1%Miscarriage
1%Severe acute bronchitis
This histogram enumerates side effects from a completed 2015 Phase 2 trial (NCT00025883) in the Metreleptin ARM group. Side effects include: Musculoskeletal pain with 15%, Weight loss with 12%, Hypoglycemia with 10%, Nausea with 9%, Abdominal pain with 8%.

Trial Design

1 Treatment Group

Patients with MSL
1 of 1
Experimental Treatment

2 Total Participants · 1 Treatment Group

Primary Treatment: Patients with MSL · No Placebo Group · Phase 1

Patients with MSL
Drug
Experimental Group · 1 Intervention: Metreleptin · Intervention Types: Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Metreleptin
2001
Completed Phase 2
~170

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, week 24

Trial Background

Prof. Elif Oral, Professor of Internal Medicine
Principal Investigator
University of Michigan
Closest Location: University of Michigan · Ann Arbor, MI
Photo of university of michigan comprehensive cancer center  1Photo of university of michigan comprehensive cancer center  2Photo of university of michigan comprehensive cancer center  3
2022First Recorded Clinical Trial
1 TrialsResearching Lipomatosis, Multiple Symmetrical
1003 CompletedClinical Trials

Eligibility Criteria

Age < 65 · All Participants · 8 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are younger than 60 years of age.\n
A negative pregnancy test (human chorionic gonadotropin, beta subunit) at baseline.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.