Efficacy of Autologous Outer-Root-Sheath Melanocytes Transplantation in the Treatment of Vitiligo

Double Blind Within-Subject Controlled Study of Autologous Hair Follicle Outer-Root-Sheath Melanocytes Transplantation in the Treatment of Vitiligo

The purpose of this study is to evaluate the hypothesis that, in patients with stable vitiligo unresponsive to conventional treatments including traditional phototherapy and/or topical steroids, the application of a non-cultured autologous hair follicle outer-root-sheath melanocytes suspension in the area affected by the disease followed by targeted ultraviolet B (UVB) phototherapy can lead to a significant skin repigmentation. To assess the effect of the proposed treatment, a within-subject controlled study involving selected symmetric lesion areas localized to the back of the hands will be conducted.

Study Overview

• Status: Terminated
• Conditions: Vitiligo
• Intervention / Treatment: Biological: Outer-Root-Sheath Melanocytes Suspension; Biological: Placebo

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Phase 2

Contacts and Locations

Study Locations

• Italy
  • Bergamo, Italy, 24127
    • Papa Giovanni XXIII Hospital
  • Brescia, Italy, 25123
    • Spedali Civili

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Presence of symmetric lesions of vitiligo at the back of the hands with an extension >5cm²
• Vitiligo lasting at least one year at the backs of both hands
• Stable vitiligo, defined as vitiligo that presents no evident evolution (appearance of new lesions or increase in the extension of lesions already present) for at least 6 months
• Suspension for at least two months prior to the enrollment date of any systemic drug for vitiligo such as immunosuppressive treatment (cyclosporine, systemic steroids) or psoralen + ultraviolet A therapy, phototherapy with ultraviolet B, and of any anticoagulant drug
• Suspension of topical medications for at least 15 days prior to the enrollment date

Exclusion Criteria:

• Presence of active vitiligo or Koebner phenomenon
• Difference of more than 10% in the extension of symmetrical areas of vitiligo
• Presence of systemic infections or infections localized to the tissues intended for transplantation
• History of infections to the tissues intended for transplantation (herpes simplex, human papillomavirus infections, pityriasis versicolor, pityriasis alba)
• Presence or history of malignancy
• Chemotherapy or radiation therapy in progress
• History of allergies or adverse reactions to local anesthetics
• Presence of transmissible diseases (human immunodeficiency virus, hepatitis B and C, human T-lymphotropic virus type I and II, syphilis, cytomegalovirus, Creutzfeldt-Jacob, tuberculosis)
• Women who are pregnant or intend to become pregnant during the study period (including breastfeeding women)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Outer-Root-Sheath Melanocytes Suspension; This arm includes all patients sides (left or right) treated with autologous outer-root-sheath melanocytes suspension followed by targeted UVB phototherapy	Biological: Outer-Root-Sheath Melanocytes Suspension; The treatment consists in depositing a suspension of autologous outer-root-sheath melanocytes, derived from patient's plucked hair follicles, and patient's own serum on the area affected by vitiligo, after properly preparation with dermabrasion. Afterwards a targeted UVB phototherapy will be carried out by patients at home for 3 days / week from the beginning of the 5th week after transplantation till the end of the study period (12th week).
Placebo Comparator: Placebo; This arm includes all patients sides (left or right) treated with placebo followed by targeted UVB phototherapy	Biological: Placebo; The treatment consists in depositing a solution made of saline and patient's own serum on the area affected by vitiligo, after properly preparation with dermabrasion. Afterwards a targeted UVB phototherapy will be carried out by patients at home for 3 days / week from the beginning of the 5th week after transplantation till the end of the study period (12th week).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Repigmentation equal to or greater than 50% of the treated areas from baseline as assessed by image analysis	12 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Any repigmentation of the treated areas from baseline as assessed by physician according to an ordinal 6-points scale	6 weeks, 12 weeks
Any repigmentation of the treated areas from baseline as assessed by patient according to an ordinal 6-points scale	6 weeks, 12 weeks
Overall patient satisfaction to the proposed therapy as assessed by visual analogue scale	6 weeks, 12 weeks
Any repigmentation of the treated areas from baseline as assessed by image analysis	6 weeks, 12 weeks
Any repigmentation of other areas affected by vitiligo from baseline as assessed by physician according to an ordinal 6-points scale (evaluation of a possible systemic effect)	6 weeks, 12 weeks

Collaborators and Investigators

• Sponsor: Centro Studi Gised
• Collaborators: ARIV Onlus
• Investigators: Principal Investigator: Luigi Naldi, MD, Centro Studi Gised

Study record dates

Study Major Dates

• Study Start: January 1, 2014
• Primary Completion (Actual): June 1, 2014
• Study Completion (Actual): June 1, 2014

Study Registration Dates

• First Submitted: August 9, 2013
• First Submitted That Met QC Criteria: August 13, 2013
• First Posted (Estimate): August 15, 2013

Study Record Updates

• Last Update Posted (Estimate): January 27, 2016
• Last Update Submitted That Met QC Criteria: January 26, 2016
• Last Verified: January 1, 2016

More Information

Terms related to this study

• Keywords: Vitiligo; Melanocytes; Outer-Root-Sheath; Hair Follicles
• Additional Relevant MeSH Terms: Skin Diseases; Pigmentation Disorders; Hypopigmentation; Vitiligo
• Other Study ID Numbers: VIT-FOL1