Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)

Sponsor

University of Michigan (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05351164

Collaborator

Amryt Pharma (Industry)

Enrollment

Location

Arm

2.1

Anticipated Duration (Months)

0.9

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Lipomatosis, Multiple Symmetrical	Drug: Metreleptin	Phase 1

Study Design

Study Type:

Interventional

Anticipated Enrollment :

2 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)

Anticipated Study Start Date :

Jul 28, 2022

Anticipated Primary Completion Date :

Oct 1, 2022

Anticipated Study Completion Date :

Oct 1, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Patients with MSL	Drug: Metreleptin Study drug for injection is supplied in a carton containing 30 vials for reconstitution. Each vial contains 11.3 mg of the study drug as a sterile, white, solid, lyophilized cake or powder to deliver 5 mg/mL of the study drug when reconstituted with 2.2 mL of water for injection (WFI).

Arm

Intervention/Treatment

Experimental: Patients with MSL

Drug: Metreleptin

Study drug for injection is supplied in a carton containing 30 vials for reconstitution. Each vial contains 11.3 mg of the study drug as a sterile, white, solid, lyophilized cake or powder to deliver 5 mg/mL of the study drug when reconstituted with 2.2 mL of water for injection (WFI).

Outcome Measures

Primary Outcome Measures

Change in truncal adiposity [Baseline, Week 24]
Measured using a Dual-energy X-ray absorptiometry (DEXA) scan
Change in total adiposity [Baseline, Week 24]
Measured using a Dual-energy X-ray absorptiometry (DEXA) scan

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Have the clinical diagnosis of MSL and being followed at University of Michigan (cohort to be studied in this proof-of-concept study is already available at Michigan).
Willing and able to tolerate the study procedures.
Willing and able to tolerate blood sampling.
Having no condition that may impede successful data collection or interfere with testing parameters.
<60 years of age.
If female of childbearing potential:
Not breastfeeding.
Negative pregnancy test (human chorionic gonadotropin, beta subunit) at baseline.
Can read, understand and sign approved informed consent form, communicate with study physician, and study team, and understand and comply with protocol requirements.

Exclusion Criteria:

Presence of advanced liver disease (abnormal synthetic function, prothrombin time [PT], or albumin) in medical records
Evidence of other etiologies of viral hepatitis in medical records
Presence of active hematologic, bone marrow or other abnormalities that may increase risk of bleeding in medical records.
Presence of HIV infection in medical records.
Presence of End-stage renal disease (ESRD), active cancer, or >class 2 congestive heart failure based on medical history and physical examination.
Active chronic infection (e.g., known chronic osteomyelitis or Tuberculosis [TB]). May have transient infections but must be free of active infection for two weeks prior to study visits.
Unable to ambulate or tolerate trips to the University of Michigan Clinical Research Unit.
Clinically relevant Coronary artery disease (CAD): history of stent or Coronary artery bypass graft surgery (CABG) with cardiologist confirmed angina.
Presence of autoimmune disease.
Hypersensitivity to metreleptin.
General obesity not associated with congenital leptin deficiency.
Any other condition that, in our opinion, may impede successful data collection.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Michigan	Ann Arbor	Michigan	United States	48109

Sponsors and Collaborators

University of Michigan
Amryt Pharma

Investigators

Principal Investigator: Elif Oral, University of Michigan

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Elif Oral, Professor of Internal Medicine, University of Michigan

ClinicalTrials.gov Identifier:

NCT05351164

Other Study ID Numbers:

HUM00206598

First Posted:

Apr 28, 2022

Last Update Posted:

Apr 28, 2022

Last Verified:

Apr 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Product Manufactured in and Exported from the U.S.:

Yes

Keywords provided by Elif Oral, Professor of Internal Medicine, University of Michigan

Metreleptin

Additional relevant MeSH terms:

Lipoma

Lipomatosis

Lipomatosis, Multiple Symmetrical

Study Results

No Results Posted as of Apr 28, 2022