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

Primary Purpose

Lipomatosis, Multiple Symmetrical

Status
Enrolling by invitation
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Metreleptin
Sponsored by
University of Michigan
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Lipomatosis, Multiple Symmetrical focused on measuring Metreleptin

Eligibility Criteria

undefined - 60 Years (Child, Adult)All SexesDoes not accept 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.

Sites / Locations

  • University of Michigan

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Patients with MSL

Arm Description

Outcomes

Primary Outcome Measures

Change in truncal adiposity
Measured using a Dual-energy X-ray absorptiometry (DEXA) scan
Change in total adiposity
Measured using a Dual-energy X-ray absorptiometry (DEXA) scan

Secondary Outcome Measures

Full Information

First Posted
April 22, 2022
Last Updated
April 6, 2023
Sponsor
University of Michigan
Collaborators
Amryt Pharma
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1. Study Identification

Unique Protocol Identification Number
NCT05351164
Brief Title
Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)
Official Title
Evaluation of Safety and Efficiency of Metreleptin Treatment for Patients With Multiple Symmetric Lipomatosis (MSL)
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
May 2023 (Anticipated)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Michigan
Collaborators
Amryt Pharma

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Lipomatosis, Multiple Symmetrical
Keywords
Metreleptin

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Patients with MSL
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Metreleptin
Intervention Description
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).
Primary Outcome Measure Information:
Title
Change in truncal adiposity
Description
Measured using a Dual-energy X-ray absorptiometry (DEXA) scan
Time Frame
Baseline, Week 24
Title
Change in total adiposity
Description
Measured using a Dual-energy X-ray absorptiometry (DEXA) scan
Time Frame
Baseline, Week 24

10. Eligibility

Sex
All
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elif Oral
Organizational Affiliation
University of Michigan
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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