Back to resultsEvaluating the Efficacy and Safety of Metformin in Vitiligo
Primary Purpose
Vitiligo
Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Metformin Hydrochloride
Sponsored by
About this trial
This is an interventional treatment trial for Vitiligo focused on measuring vitiligo, metformin, systemic treatment, stable vitiligo, segmental vitiligo, non-segmental vitiligo
Eligibility Criteria
Inclusion Criteria: Adults 18 years and older with stable vitiligo Stable vitiligo is defined as no new spots of depigmentation or expansion of any existing spots for one year; Willingness to participate in the study; Willingness to undergo suction blistering; Non-English speaking adults may be enrolled with the assistance of an interpreter and the use of an IRB-approved short form in the subject's language; Informed consent document signed by the subject; Exclusion Criteria: Adults unable to consent (adults lacking capacity); Active vitiligo defined by presence of confetti lesions, trichrome lesions, and Koebner's phenomenon; Individuals who are not yet adults (infants, children, teenagers); Pregnant women and/or breastfeeding, or those who have recently delivered a baby within the past 6 months; Prisoners; Systemic immunosuppressive medication (oral corticosteroids) within prior 4 weeks; Topical steroids within the prior 2 weeks; Currently undergoing UVB light therapy or history of light therapy within the past 8 weeks; Unable to return for follow-up visits; Enrolled in a clinical study of any other investigational drug or device; Diabetes, liver disease, or kidney disease; Hypoglycemia as defined by fasting blood glucose <70 mg/dL assessed at a fasting study visit; Prescription medication or cosmetics containing: retinoids, glycolic acid, salicylic acid, or any other remedies that might affect the healing process. Non-medicated moisturizers are allowed. If the person is unsure, they can bring in any products for our review; Self-reported history of chronic alcohol or drug abuse within 12 months prior to screening, or any condition associated with poor compliance as judged by the investigator; Any other condition or laboratory value that would, in the professional opinion of the investigators, potentially affect the subject's response or the integrity of the data or would pose an unacceptable risk to the subject.
Sites / Locations
- UMass Chan Medical SchoolRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Oral Metformin
Arm Description
Treatment with metformin will be started at 500 mg twice daily and increased to 1000 mg twice daily only after they have tolerated the treatment.
Outcomes
Primary Outcome Measures
F-VASI 50
The primary outcome will be 50% of subjects who achieve a facial VASI50 (F-VASI50) after 6 months of treatment. Comparisons will be made from scores at week 24 to baselines scores.
Secondary Outcome Measures
Safety and tolerability of metformin use in patients with vitiligo.
Proportion of adverse events in patients with vitiligo receiving metformin at week 24.
Expression of enzyme markers involved in metabolic pathways of interest
Determination of the effects of metformin treatment on expression of enzyme markers involved in metabolic pathways of interest, as well as definition of the subpopulations of skin cells using protein surface markers at a single cell level., autoreactive CD8+ T cells in the blood of patients with vitiligo treated with metformin.
Gene expression and ion abundance following metformin treatment.
Two-fold decrease in gene expression and ion abundance following metformin treatment.
Full Information
NCT ID
NCT05607316
First Posted
October 31, 2022
Last Updated
May 31, 2023
Sponsor
University of Massachusetts, Worcester
1. Study Identification
Unique Protocol Identification Number
NCT05607316
Brief Title
Evaluating the Efficacy and Safety of Metformin in Vitiligo
Official Title
Metformin as a Novel Treatment for Vitiligo by Targeting CD8+ T Cell Metabolism
Study Type
Interventional
2. Study Status
Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 1, 2023 (Actual)
Primary Completion Date
July 1, 2027 (Anticipated)
Study Completion Date
November 1, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of Massachusetts, Worcester
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
Metformin modulates metabolism in multiple cell types and is currently used to reduce glucose levels and insulin resistance in diabetic patients. The investigators hypothesize that oral metformin can regulate the metabolism of CD8+ T cells, reduce their cytotoxic activity and thus serve as a novel treatment for vitiligo.
Detailed Description
Metformin modulates metabolism in multiple cell types and is currently used to reduce glucose levels and insulin resistance in diabetic patients. Bae et al. reported that the use of metformin correlated with a lower risk of developing vitiligo, suggesting that metformin could potentially mitigate the disease. The investigators found that treating mouse T cells with metformin during activation reduced their mitochondrial respiration and proliferation, while mice treated with metformin reversed their vitiligo. Therefore, the investigators hypothesize that regulation of CD8+ T cell metabolism in vitiligo patients by metformin will reduce their proliferation and cytotoxic activity, resulting in skin repigmentation and thus serve as a novel treatment.
The investigators plan to treat approximately 30 subjects with stable vitiligo.
Metformin is FDA-approved for use with dosing from 500-2000 mg/day. It has a rare risk of lactic acidosis, which can be meaningful in patients with risk factors such as renal insufficiency. This risk is directly proportional to the dose given; therefore, participants will be started at a lower dose (500 mg twice daily) with follow-up to monitor any arising symptoms. Per current clinical recommendations, participants will only be increased to higher-dose metformin (1000 mg twice daily) if the initial dose is tolerated.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Vitiligo
Keywords
vitiligo, metformin, systemic treatment, stable vitiligo, segmental vitiligo, non-segmental vitiligo
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
Open-label (unmasked) study
Masking
None (Open Label)
Masking Description
Open-label (unmasked)
Allocation
N/A
Enrollment
30 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Oral Metformin
Arm Type
Experimental
Arm Description
Treatment with metformin will be started at 500 mg twice daily and increased to 1000 mg twice daily only after they have tolerated the treatment.
Intervention Type
Drug
Intervention Name(s)
Metformin Hydrochloride
Other Intervention Name(s)
Glucophage, Glumetza, Riomet
Intervention Description
Consistent with previous studies and clinical recommendations, subjects will initiate treatment at metformin 500 mg twice daily and increase to 1000 mg twice daily only after they have tolerated the treatment (details below).
Study Visit 1 (Week 1) - Study Visit 2 (Week 2) Subjects will be directed to take metformin 500 mg twice daily.
Study Visit 2 (Week 2) - Study Visit 5 (Week 24) If initial metformin dose is tolerated, subjects will be directed to increase the dose to 1000 mg by mouth twice daily at bedtime for the remainder of the study.
Primary Outcome Measure Information:
Title
F-VASI 50
Description
The primary outcome will be 50% of subjects who achieve a facial VASI50 (F-VASI50) after 6 months of treatment. Comparisons will be made from scores at week 24 to baselines scores.
Time Frame
Week 24
Secondary Outcome Measure Information:
Title
Safety and tolerability of metformin use in patients with vitiligo.
Description
Proportion of adverse events in patients with vitiligo receiving metformin at week 24.
Time Frame
Week 24
Title
Expression of enzyme markers involved in metabolic pathways of interest
Description
Determination of the effects of metformin treatment on expression of enzyme markers involved in metabolic pathways of interest, as well as definition of the subpopulations of skin cells using protein surface markers at a single cell level., autoreactive CD8+ T cells in the blood of patients with vitiligo treated with metformin.
Time Frame
Week 24
Title
Gene expression and ion abundance following metformin treatment.
Description
Two-fold decrease in gene expression and ion abundance following metformin treatment.
Time Frame
Week 24
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Adults 18 years and older with stable vitiligo
Stable vitiligo is defined as no new spots of depigmentation or expansion of any existing spots for one year;
Willingness to participate in the study;
Willingness to undergo suction blistering;
Non-English speaking adults may be enrolled with the assistance of an interpreter and the use of an IRB-approved short form in the subject's language;
Informed consent document signed by the subject;
Exclusion Criteria:
Adults unable to consent (adults lacking capacity);
Active vitiligo defined by presence of confetti lesions, trichrome lesions, and Koebner's phenomenon;
Individuals who are not yet adults (infants, children, teenagers);
Pregnant women and/or breastfeeding, or those who have recently delivered a baby within the past 6 months;
Prisoners;
Systemic immunosuppressive medication (oral corticosteroids) within prior 4 weeks;
Topical steroids within the prior 2 weeks;
Currently undergoing UVB light therapy or history of light therapy within the past 8 weeks;
Unable to return for follow-up visits;
Enrolled in a clinical study of any other investigational drug or device;
Diabetes, liver disease, or kidney disease;
Hypoglycemia as defined by fasting blood glucose <70 mg/dL assessed at a fasting study visit;
Prescription medication or cosmetics containing: retinoids, glycolic acid, salicylic acid, or any other remedies that might affect the healing process. Non-medicated moisturizers are allowed. If the person is unsure, they can bring in any products for our review;
Self-reported history of chronic alcohol or drug abuse within 12 months prior to screening, or any condition associated with poor compliance as judged by the investigator;
Any other condition or laboratory value that would, in the professional opinion of the investigators, potentially affect the subject's response or the integrity of the data or would pose an unacceptable risk to the subject.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Nuria Martinez Gutierrez, PhD
Phone
(774)4554758
Email
nuria.martinezgutierrez@umassmed.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John E Harris, MD, PhD
Organizational Affiliation
Chair, Department of Dermatology
Official's Role
Principal Investigator
Facility Information:
Facility Name
UMass Chan Medical School
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01605
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nuria Martinez, PhD
Phone
774-455-4758
Email
nuria.martinezgutierrez@umassmed.edu
First Name & Middle Initial & Last Name & Degree
John E Harris, MD PhD
12. IPD Sharing Statement
Citations:
PubMed Identifier
24523323
Citation
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Evaluating the Efficacy and Safety of Metformin in Vitiligo
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