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NCT05351164·Phase 2·INTERVENTIONAL

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

Status

Active, not recruiting

Phase

Phase 2

Enrollment

4

Locations

1

Results

Posted

Publications

0

Study summary

What the protocol is testing.

Participants (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. Additional safety will be assessed for 1 more year (up to 1.5 years total) in which adverse event data will be collected.

Full detailed description

In 2024, the IRB granted an amendment that allowed for continued data collection, including adverse events, up to 5 years per participant.

Interventions

Treatment arms and agents.

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).

Timeline

From registration to results.

  1. First posted

    Apr 28, 2022

  2. Study start

    Aug 1, 2023

  3. Primary completion

    Apr 8, 2024

  4. Study completion

    Oct 2028

  5. Results posted

    Jun 12, 2025

  6. Registry updated

    Jun 12, 2025

Outcomes

What the study measures.

Primary outcomes

Change in Truncal Adiposity

Time frame · Baseline, Week 24

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.

Change in Total Adiposity

Time frame · 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.

Secondary outcomes

Not reported in the indexed record.

Eligibility

Who can take part.

Minimum age
Not reported
Maximum age
60 Years
Sex
ALL
Healthy volunteers
No

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.

Study locations

1 registered sites.

United States. Showing up to 24 locations stored in the fast local snapshot.

University of Michigan

Ann Arbor, Michigan, United States

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

Related trials

More studies on Metreleptin.

Related PeptideStat pages

Put the record in context.

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