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

Plain-language summary

This study evaluates the safety and efficacy of a drug called Metreleptin in patients with multiple symmetric lipomatosis (MSL). It enrolls patients under 60 years of age who have a clinical diagnosis of MSL. The study is in Phase 2 and is currently active but not recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Phase 2

Status

Active, not recruiting

Sponsor

University of Michigan

Intervention

Metreleptin

Start date

8/1/2023

Estimated completion

10/1/2028

Countries

United States

Related genes

Locations

• Ann Arbor, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.