Clinical Study to Investigate the Safety and Effectiveness of RECELL for Repigmentation of Stable Vitiligo Lesions

A Prospective Blinded Within-Subject Randomized Controlled Clinical Study to Investigate the Safety and Effectiveness of RECELL for Repigmentation of Stable Vitiligo Lesions

This is a prospective blinded within-subject randomized study to evaluate the application of Spray-On Skin™ Cells, prepared using the RECELL® Device for safe and effective repigmentation of ablated stable vitiligo lesions.

Study Overview

• Status: Completed
• Conditions: Vitiligo
• Intervention / Treatment: Device: Control Intervention (UVB); Device: Investigational Interventions (Ablative Laser Resurfacing + RECELL + UVB)

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • California
    • Irvine, California, United States, 92697
      • University of California, Irvine
    • Sacramento, California, United States, 95817
      • University of California, Davis
    • San Diego, California, United States, 92121
      • West Dermatology
  • Connecticut
    • Westport, Connecticut, United States, 06880
      • DMR Research
  • Florida
    • Hollywood, Florida, United States, 33021
      • Skin Care Research, Llc
    • Miami, Florida, United States, 33173
      • Miami Dermatology and Laser Institute
  • Massachusetts
    • Worcester, Massachusetts, United States, 01581
      • University of Massachusetts
  • Michigan
    • Detroit, Michigan, United States, 48202
      • Henry Ford Health System
  • North Carolina
    • Charlotte, North Carolina, United States, 28207
      • Dermatology, Laser & Vein Specialists of the Carolinas
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Texas
    • Austin, Texas, United States, 78712
      • University of Texas at Austin Dell Medical School
    • Houston, Texas, United States, 77008
      • Heights Dermatology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Focal, segmental or generalized (i.e., nonsegmental) stable vitiligo, defined as no new depigmented areas nor any depigmented areas that have expanded in size within the preceding 12 months, regardless of whether the areas are intended to be used as study areas.

1a. Photo documentation (current and at least 12 months prior) of the patient's depigmented areas have been evaluated as stable by an independent Screening Committee.

2. The patient has not undergone topical treatment (e.g., steroids, tacrolimus) for the study areas within the past 90 days.

3. The patient has not undergone phototherapy (e.g., NB-UVB) for the study areas within the past 90 days.

4. The patient is a candidate for surgical intervention of a depigmented area, defined as a patient who has previously been compliant with but has not satisfactorily responded to both

a. topical therapy and b. a minimum of 3 months of phototherapy.

5. The patient must have two study areas available for treatment that:

1. are of similar size (±50%),
2. are between 16cm2 and 456cm2 (contiguous),
3. are similarly sun exposed,
4. have the same extent of leukotrichia, and

5. are judged clinically as ≥90% depigmented (by area).

   6. The patient is 18 years of age or older.

   7. The patient is willing and able to comply with post-treatment at-home phototherapy and all follow-up evaluations required by the study protocol.

   8. The patient agrees to abstain from any other treatment of the study areas for the duration of his/her participation in the study (52 weeks).

   9. The patient agrees to abstain from enrollment in any other interventional clinical trial for the duration of his/her participation in the study (52 weeks).

   10. In the opinion of the investigator, the patient must be able to:

a. Understand the full nature and purpose of the study, including possible risks and adverse events, b. Understand instructions, and c. Provide voluntary written informed consent.

Exclusion Criteria:

1. The study areas selected have concomitant dermatologic conditions other than vitiligo.
2. The study area selected for treatment includes the lips, eyelids, plantar surface of feet, palmar surface of hands, fingertips, wrists, ankles, elbows, or knees.
3. The patient is unable to undergo the treatment area preparation.
4. Patients who are pregnant.

5. Patients with:

   1. universalis vitiligo,
   2. depigmented areas over >30% of their body surface area,
   3. depigmented lips and fingertips (lip-tip vitiligo), or
   4. > 3 depigmented fingertips, defined as depigmentation of the dorsal aspect of the fingertip from the distal interphalangeal joint to the tip of the digit.

6. Patients with recent history (within previous 12 months) of:

   1. Koebnerization,
   2. confetti-like, or
   3. trichrome lesions.
7. Patients with a history of keloid formation.
8. Patients who have used a tanning salon in the past 60 days.
9. The patient has other concurrent conditions that in the opinion of the investigator may compromise patient safety or study objectives.
10. Current use of medications (e.g., anticoagulants such as heparin or warfarin) that in the investigator's opinion may compromise patient safety or trial objectives.
11. The patient has a known hypersensitivity to trypsin or compound sodium lactate for irrigation (Hartmann's) solution.
12. Life expectancy is less than 1 year.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: All Participants (within patient control); Each participant will serve as their own Control, receiving both Control and Investigational Interventions randomly allocated to treatment of a portion of a depigmented vitiligo lesion.	Device: Control Intervention (UVB); Targeted narrowband-UVB phototherapy in alignment with published Vitiligo Working Group recommendations.; Other Names: Phototherapy; Device: Investigational Interventions (Ablative Laser Resurfacing + RECELL + UVB); Spray-On Skin™ Cells prepared using RECELL will be applied to an area prepared by ablative laser, followed by targeted narrowband UVB phototherapy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of ≥80% Study Area Repigmentation	Repigmentation was evaluated by qualified members of a Central Review Committee.	Week 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Repigmentation Category	Expert Central Review Committee categorization of the amount of area repigmented (0-25%, 26-50%, 51-79% and 80-100%). This is visual estimation of the vitiligo lesion area that has repigmented, with reference to training images. Higher %repigmentation is a better outcome.	Week 24
Color Matching	The Expert Central Review Committee visually judged the quality of repigmentation in terms of how closely it matches with the normal pigmentation seen in the surrounding area. The rating scale consists of poor, moderate, good, and excellent. Color matching can only be evaluated in instances of >0% repigmentation.	Week 24

Collaborators and Investigators

• Sponsor: Avita Medical

Publications and helpful links

General Publications

• Liu JP, Hsueh HM, Hsieh E, Chen JJ. Tests for equivalence or non-inferiority for paired binary data. Stat Med. 2002 Jan 30;21(2):231-45. doi: 10.1002/sim.1012.

Study record dates

Study Major Dates

• Study Start (Actual): September 10, 2020
• Primary Completion (Actual): June 28, 2022
• Study Completion (Actual): January 31, 2023

Study Registration Dates

• First Submitted: September 8, 2020
• First Submitted That Met QC Criteria: September 8, 2020
• First Posted (Actual): September 14, 2020

Study Record Updates

• Last Update Posted (Actual): September 21, 2023
• Last Update Submitted That Met QC Criteria: August 25, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Skin Diseases; Pigmentation Disorders; Hypopigmentation; Vitiligo
• Other Study ID Numbers: CTP009

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes