Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric… (NCT05351164) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)
United States4 participantsStarted 2023-08-01
Plain-language summary
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.
Who can participate
Age range60 Years
SexALL
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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…