Vitiligo
Conditions
Brief summary
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.
Interventions
DEVICEControl Intervention (UVB)
Targeted narrowband-UVB phototherapy in alignment with published Vitiligo Working Group recommendations.
DEVICEInvestigational 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.
Sponsors
Avita Medical
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Masking description
The Central Review Committee (CRC) will be blinded to treatment assignment when adjudicating data.
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of ≥80% Study Area Repigmentation | Week 24 | Repigmentation was evaluated by qualified members of a Central Review Committee. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Repigmentation Category | Week 24 | 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. |
| Color Matching | Week 24 | 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. |
Countries
United States
Participant flow
Pre-assignment details
The design of this study is a within-patient comparison. Protocol enrollment is 25 (participants), with 50 interventions (2 per patient).
Participants by arm
| Arm | Count |
|---|---|
| All Participants (Within Patient Control) All subjects will receive both RECELL and NV-UVB. Each subject will serve as their own Control, with a depigmented area receiving no RECELL treatment but receiving the same targeted NB-UVB as the investigational treatment area. Study treatment areas will be randomly assigned as Area A and Area B. Investigational treatment (RECELL) will be randomly allocated to either Area A or Area B.
Spray-On Skin™ Cells 1:20 followed by NB-UVB: Skin cell suspension at expansion ratio 1:20 (donor area : recipient area), prepared using the RECELL System, will be applied to an ablated (de-epithelialized) area of depigmentation, followed by targeted phototherapy using NB-UVB.
NB-UVB only: Each subject will serve as their own control, with a portion (or similar) depigmented lesion receiving the same targeted NB-UVB without application of skin cell suspension. | 25 |
| Total | 25 |
Baseline characteristics
| Characteristic | All Participants (Within Patient Control) |
|---|---|
| Age, Continuous | 41.0 years STANDARD_DEVIATION 12.5 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 6 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 19 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 3 Participants |
| Race (NIH/OMB) Black or African American | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 20 Participants |
| Sex: Female, Male Female | 13 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 25 | 0 / 25 |
| other Total, other adverse events | 1 / 25 | 5 / 25 |
| serious Total, serious adverse events | 0 / 25 | 0 / 25 |
Outcome results
Primary
Incidence of ≥80% Study Area Repigmentation
Repigmentation was evaluated by qualified members of a Central Review Committee.
Time frame: Week 24
Population: ITT
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Control Intervention (UVB) | Incidence of ≥80% Study Area Repigmentation | 0 study areas |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Incidence of ≥80% Study Area Repigmentation | 9 study areas |
p-value: 0.012continuity-corrected method
Secondary
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.
Time frame: Week 24
Population: ITT
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control Intervention (UVB) | Color Matching | Color Matching Category: Poor | 0 study areas with >0% repigmentation |
| Control Intervention (UVB) | Color Matching | Color Matching Category: Moderate | 4 study areas with >0% repigmentation |
| Control Intervention (UVB) | Color Matching | Color Matching Category: Good | 12 study areas with >0% repigmentation |
| Control Intervention (UVB) | Color Matching | Color Matching Category: Excellent | 4 study areas with >0% repigmentation |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Color Matching | Color Matching Category: Excellent | 6 study areas with >0% repigmentation |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Color Matching | Color Matching Category: Poor | 1 study areas with >0% repigmentation |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Color Matching | Color Matching Category: Good | 9 study areas with >0% repigmentation |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Color Matching | Color Matching Category: Moderate | 6 study areas with >0% repigmentation |
p-value: 1Wilcoxon (Mann-Whitney)
Secondary
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.
Time frame: Week 24
Population: ITT
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Control Intervention (UVB) | Repigmentation Category | Repigmentation Category: 100% | 0 depigmented vitiligo lesions |
| Control Intervention (UVB) | Repigmentation Category | Repigmentation Category: 0% | 5 depigmented vitiligo lesions |
| Control Intervention (UVB) | Repigmentation Category | Repigmentation Category: 1 to 25% | 13 depigmented vitiligo lesions |
| Control Intervention (UVB) | Repigmentation Category | Repigmentation Category: 26 to 50% | 4 depigmented vitiligo lesions |
| Control Intervention (UVB) | Repigmentation Category | Repigmentation Category: 51 to 79% | 3 depigmented vitiligo lesions |
| Control Intervention (UVB) | Repigmentation Category | Repigmentation Category: 80 to 99% | 0 depigmented vitiligo lesions |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Repigmentation Category | Repigmentation Category: 51 to 79% | 5 depigmented vitiligo lesions |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Repigmentation Category | Repigmentation Category: 100% | 0 depigmented vitiligo lesions |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Repigmentation Category | Repigmentation Category: 26 to 50% | 2 depigmented vitiligo lesions |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Repigmentation Category | Repigmentation Category: 0% | 3 depigmented vitiligo lesions |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Repigmentation Category | Repigmentation Category: 80 to 99% | 9 depigmented vitiligo lesions |
| Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) | Repigmentation Category | Repigmentation Category: 1 to 25% | 6 depigmented vitiligo lesions |
p-value: <0.001Wilcoxon (Mann-Whitney)