Trial Outcomes & Findings for Clinical Trial of Simvastatin to Treat Generalized Vitiligo (NCT NCT01517893)

Last Updated: 2018-11-14

Results Overview

Number of participants with 33% decrease in the Vitiligo Area Scoring Index (VASI) from baseline to the last available study visit. Decrease in VASI score means improvement. Minimum value is 0, that means no vitiligo. maximum value is 100, that means 100% of the body surface area has vitiligo (total body surface area).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

Assessed at baseline and final study visit, 6 months after randomization

Results posted on

2018-11-14

Participant Flow

Participant milestones

Participant milestones
Measure	Intervention Arm Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Study STARTED	8	7
Overall Study COMPLETED	5	7
Overall Study NOT COMPLETED	3	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Intervention Arm Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Overall Study Withdrawal by Subject	3	0

Baseline Characteristics

Clinical Trial of Simvastatin to Treat Generalized Vitiligo

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Intervention Arm n=8 Participants Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=7 Participants Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Total n=15 Participants Total of all reporting groups
Age, Continuous	43.9 years n=93 Participants	39 years n=4 Participants	41.6 years n=27 Participants
Sex: Female, Male Female	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Sex: Female, Male Male	8 Participants n=93 Participants	7 Participants n=4 Participants	15 Participants n=27 Participants
Vitiligo area scoring index	14.81 percentage n=93 Participants	23.09 percentage n=4 Participants	19.23 percentage n=27 Participants

PRIMARY outcome

Timeframe: Assessed at baseline and final study visit, 6 months after randomization

Population: Overall number of participants for intervention (5) differs from enrollment (8) due to withdraw of 3 participants on intervention arm.

Outcome measures

Outcome measures
Measure	Intervention Arm n=5 Participants Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=7 Participants Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Number of Participants With a Decrease in Vitiligo Area Scoring Index (VASI) Score	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Assessed at baseline and final study visit, 6 months after randomization

Population: For placebo arm, only 6 out of 7 participants reported data.

Increase in Investigator Global Assessment Scores of 30% or more from baseline to last available visit. Increase in score means improvement. 0% is no improvement at all. 100% is complete recovery.

Outcome measures

Outcome measures
Measure	Intervention Arm n=5 Participants Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=6 Participants Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Number of Participants With Increase in Investigator's Global Assessment Score	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Assessed at baseline, then monthly until final study visit, six months after randomization.

The number of participants who experienced toxicity based upon monitored lab values (Liver Function Test) and patient symptoms for evidence of simvastatin toxicity

Outcome measures

Outcome measures
Measure	Intervention Arm n=5 Participants Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=7 Participants Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Number of Participants Experiencing Toxicity From of High-dose Simvastatin .	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Assessed at baseline and final study visit, 6 months after randomization

Change in percent depigmentation of sentinel patch lesion from baseline to last available study visit ( 6 months after randomization). positive numbers mean increase or worsening of sentinel patch area negative numbers mean decrease or improvement of sentinel patch area

Outcome measures

Outcome measures
Measure	Intervention Arm n=5 Participants Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=7 Participants Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Change in Sentinel Patch Area	-0.2272 cm2 Standard Deviation 3.463	3.8571 cm2 Standard Deviation 9.17

SECONDARY outcome

Timeframe: Assessed at baseline and final study visit, 6 months after randomization

The aim of this questionnaire is to measure how much your skin problem has affected your life. We measured change in questionnaire score from baseline to end of study (at 6 months after randomization) of subjects randomized to treatment with simvastatin versus placebo. Change was measured as a drop in score at the end of 6 months of treatment. Minimum score is 0, maximum is 30. Higher value means worse score.

Outcome measures

Outcome measures
Measure	Intervention Arm n=5 Participants Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=7 Participants Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Change in Quality of Life Score by Using DERMATOLOGY LIFE QUALITY INDEX (DLQI)	3.4 units on a scale Standard Deviation 4.159326869	2.285714286 units on a scale Standard Deviation 2.690370837

SECONDARY outcome

Timeframe: Assessed at baseline and final study visit, 6 months after randomization

Population: Placebo arm reports 6 instead of enrolled 7 due to failure of participant to complete assessment.

Increase in Patient's Global Assessment Scores of 30% or more from baseline to last available visit Increase means improvement. minimum is 0% and maximum is 100%

Outcome measures

Outcome measures
Measure	Intervention Arm n=5 Participants Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=6 Participants Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Number of Participants With an Increase in Patient's Global Assessment Score	0 Participants	1 Participants

SECONDARY outcome

Timeframe: Assessed at baseline and final study visit, 6 months after randomization

Population: Serum CXCL10 levels from the first and last available clinic visits were measured via ELISA. The mean and SEM are reported here. One participant in intervention group was not included because participant withdrew before a second CXCL10 level was obtained. Other withdrawn participants were included, using the CXCL10 from their final visits.

Determination of the effects of simvastatin treatment on Serum CXCL10 levels from the first and last available clinic visits were measured via ELISA in the blood of patients with vitiligo treated with simvastatin versus placebo

Outcome measures

Outcome measures
Measure	Intervention Arm n=7 Participants Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=7 Participants Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Serum CXCL10 Levels From the First and Last Available Clinic Visits Were Measured Via ELISA	0.9148 Fold change of baseline CXCL10 level Standard Error 0.263	0.6176 Fold change of baseline CXCL10 level Standard Error 0.1685

SECONDARY outcome

Timeframe: Assessed prior to treatment and periodically while on treatment

Population: No data collected as this outcome was abandoned due to budget constraints and strength of other study results.

Determination of the effects of simvastatin treatment on CXCR3 expression in melanocyte-specific, autoreactive CD8+ T cells in the blood of patients with vitiligo treated with simvastatin versus placebo

Outcome measures

Outcome data not reported

Adverse Events

Intervention Arm

Serious events: 1 serious events

Other events: 4 other events

Deaths: 0 deaths

Placebo Arm

Serious events: 0 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Intervention Arm n=8 participants at risk Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=7 participants at risk Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Skin and subcutaneous tissue disorders Burn	12.5% 1/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	0.00% 0/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.

Other adverse events

Additional Information

John E. Harris, MD, PhD

University of Massachusetts Medical School

Phone: 508-856-1982

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Intervention Arm n=8 participants at risk Sig: Simvastatin 40 mg, increased to 80 mg after 1 month if initial dose tolerated Simvastatin: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated	Placebo Arm n=7 participants at risk Placebo: Sig: 40 mg PO daily for 1 month, increased to 80 mg PO daily for 5 months if low dose tolerated
Musculoskeletal and connective tissue disorders back pain	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Musculoskeletal and connective tissue disorders leg twinge	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Gastrointestinal disorders diarrhea	12.5% 1/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	28.6% 2/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Gastrointestinal disorders abdominal pain	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Ear and labyrinth disorders tinnitus	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Gastrointestinal disorders flatulence	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Gastrointestinal disorders stool odor	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Musculoskeletal and connective tissue disorders pulled muscle	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Musculoskeletal and connective tissue disorders neck pain	12.5% 1/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	0.00% 0/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Musculoskeletal and connective tissue disorders myalgia	25.0% 2/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	0.00% 0/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Musculoskeletal and connective tissue disorders arthrlagia	25.0% 2/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	0.00% 0/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Respiratory, thoracic and mediastinal disorders URI symptoms	12.5% 1/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	28.6% 2/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Skin and subcutaneous tissue disorders xerosis	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Respiratory, thoracic and mediastinal disorders cough	0.00% 0/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	14.3% 1/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Renal and urinary disorders urine color change	12.5% 1/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	0.00% 0/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Nervous system disorders sleep disturbance	12.5% 1/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	0.00% 0/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.
Infections and infestations dental infection	12.5% 1/8 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.	0.00% 0/7 • 6 months (baseline to end of treatment) Safety lab values and questionnaires collected for outcomes.