Trial Outcomes & Findings for Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL) (NCT NCT05351164)

Last Updated: 2025-06-12

Results Overview

A dual-energy X-ray absorptiometry (DEXA) scan was used to measure truncal adiposity at baseline and week 24. Truncal adiposity is the amount of fat a person has in the trunk area, and this outcome measure looked at the change in the fat in this area of the body in relation to the total fat in the participant's body. Results represent the change in the percentage of fat tissue from the trunk area to the total body.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

Baseline, Week 24

Results posted on

2025-06-12

Participant Flow

Participant milestones

Participant milestones
Measure	Patients With MSL 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).
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Patients With MSL n=4 Participants 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).
Age, Continuous	38.5 years STANDARD_DEVIATION 19.3 • n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 24

Outcome measures

Outcome measures
Measure	Patients With MSL n=4 Participants 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).
Change in Truncal Adiposity Baseline	22.8 percentage of truncal body fat Standard Deviation 3.2
Change in Truncal Adiposity Week 24	16.0 percentage of truncal body fat Standard Deviation 6.0

PRIMARY outcome

Timeframe: Baseline, Week 24

A dual-energy X-ray absorptiometry (DEXA) scan was used to measure total adiposity at baseline and week 24. Total adiposity is the amount of fat participants had in the body compared to their total weight. Results represent the change in the percentage of fat tissue from the total body to the total weight.

Outcome measures

Outcome measures
Measure	Patients With MSL n=4 Participants 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).
Change in Total Adiposity Baseline	21.9 percentage of body weight Standard Deviation 2.3
Change in Total Adiposity Week 24	16.7 percentage of body weight Standard Deviation 4.7

Adverse Events

Patients With MSL

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Patients With MSL n=4 participants at risk 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).
Injury, poisoning and procedural complications fall from recreational vehicle	25.0% 1/4 • 24 weeks
Metabolism and nutrition disorders weight loss	25.0% 1/4 • 24 weeks
Surgical and medical procedures planned orthopedic surgery	25.0% 1/4 • 24 weeks
Cardiac disorders palpitations and chest discomfort	25.0% 1/4 • 24 weeks
Gastrointestinal disorders Diarrhea or nausea	50.0% 2/4 • 24 weeks
General disorders less stamina and strength	25.0% 1/4 • 24 weeks
Musculoskeletal and connective tissue disorders Musculoskeletal pain	50.0% 2/4 • 24 weeks
Renal and urinary disorders Hematuria (self resolved)	25.0% 1/4 • 24 weeks
Infections and infestations viral and non-serious bacterial infections	75.0% 3/4 • 24 weeks
Blood and lymphatic system disorders high blood pressure	25.0% 1/4 • 24 weeks
General disorders high body temperature and intermittent shivering	25.0% 1/4 • 24 weeks
Cardiac disorders Chest discomfort and pain	25.0% 1/4 • 24 weeks
General disorders Syncope and collapse	25.0% 1/4 • 24 weeks
General disorders Nocturnal hunger	25.0% 1/4 • 24 weeks
General disorders Painful lipoma	25.0% 1/4 • 24 weeks

Additional Information

Elif Oral

University of Michigan

Phone: 734-615-7271

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place