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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All Participants (within patient control) | Experimental | 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. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Control Intervention (UVB) | Device | Targeted narrowband-UVB phototherapy in alignment with published Vitiligo Working Group recommendations. |
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| Measure | Description | Time Frame |
|---|---|---|
| Incidence of ≥80% Study Area Repigmentation | Repigmentation was evaluated by qualified members of a Central Review Committee. | Week 24 |
| 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 |
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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:
are of similar size (±50%),
are between 16cm2 and 456cm2 (contiguous),
are similarly sun exposed,
have the same extent of leukotrichia, and
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:
The study areas selected have concomitant dermatologic conditions other than vitiligo.
The study area selected for treatment includes the lips, eyelids, plantar surface of feet, palmar surface of hands, fingertips, wrists, ankles, elbows, or knees.
The patient is unable to undergo the treatment area preparation.
Patients who are pregnant.
Patients with:
Patients with recent history (within previous 12 months) of:
Patients with a history of keloid formation.
Patients who have used a tanning salon in the past 60 days.
The patient has other concurrent conditions that in the opinion of the investigator may compromise patient safety or study objectives.
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.
The patient has a known hypersensitivity to trypsin or compound sodium lactate for irrigation (Hartmann's) solution.
Life expectancy is less than 1 year.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Irvine | Irvine | California | 92697 | United States | ||
| University of California, Davis |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 11782062 | Background | 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. |
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The design of this study is a within-patient comparison. Protocol enrollment is 25 (participants), with 50 interventions (2 per patient).
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| ID | Title | Description |
|---|---|---|
| FG000 | Control Intervention (UVB) | A portion of each participant's vitiligo lesion is randomly assigned to receive the Control Intervention (within-patient control): Targeted narrowband-UVB (only) |
| FG001 | Investigational Intervention (Ablative Laser Resurfacing + RECELL + UVB) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 8, 2021 |
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The Central Review Committee (CRC) will be blinded to treatment assignment when adjudicating data.
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| Investigational Interventions (Ablative Laser Resurfacing + RECELL + UVB) | Device | Spray-On Skinâ„¢ Cells prepared using RECELL will be applied to an area prepared by ablative laser, followed by targeted narrowband UVB phototherapy. |
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| 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 |
| Sacramento |
| California |
| 95817 |
| United States |
| West Dermatology | San Diego | California | 92121 | United States |
| DMR Research | Westport | Connecticut | 06880 | United States |
| Skin Care Research, LLC | Hollywood | Florida | 33021 | United States |
| Miami Dermatology and Laser Institute | Miami | Florida | 33173 | United States |
| University of Massachusetts | Worcester | Massachusetts | 01581 | United States |
| Henry Ford Health System | Detroit | Michigan | 48202 | United States |
| Dermatology, Laser & Vein Specialists of the Carolinas | Charlotte | North Carolina | 28207 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| University of Texas at Austin Dell Medical School | Austin | Texas | 78712 | United States |
| Heights Dermatology | Houston | Texas | 77008 | United States |
A portion of each participant's vitiligo lesion is randomly allocated to receive the Investigational Treatment: Spray-On Skinâ„¢ Cells prepared using RECELL will be applied to an area prepared by ablative laser, followed by targeted narrowband UVB phototherapy. |
| COMPLETED |
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| NOT COMPLETED |
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Experimental: All Participants (within patient control)
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| ID | Title | Description |
|---|---|---|
| BG000 | 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 Skinâ„¢ 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. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Incidence of ≥80% Study Area Repigmentation | Repigmentation was evaluated by qualified members of a Central Review Committee. | ITT | Posted | Number | study areas | Week 24 | depigmented vitiligo lesions | depigmented vitiligo lesions |
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| 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. | ITT | Posted | Number | depigmented vitiligo lesions | Week 24 | depigmented vitiligo lesions | depigmented vitiligo lesions |
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| 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. | ITT | Posted | Number | study areas with >0% repigmentation | Week 24 | study areas with >0% repigmentation | study areas with >0% repigmentation |
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52 Weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Control Intervention | 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. Control area intervention: NB-UVB only without application of skin cell suspension. | 0 | 25 | 0 | 25 | 1 | 25 |
| EG001 | Investigational Intervention (RECELL) | 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. Investigational Intervention: Spray-On Skinâ„¢ 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. | 0 | 25 | 0 | 25 | 5 | 25 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Scar | Injury, poisoning and procedural complications | Systematic Assessment |
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| Erythema | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Vitiligo | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| SVP Global Clinical Research | AVITA | 1-833-462-8482 | ekirshner@avitamedical.com |
| Apr 29, 2024 |
| Prot_SAP_002.pdf |
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| ID | Term |
|---|---|
| D014820 | Vitiligo |
| ID | Term |
|---|---|
| D017496 | Hypopigmentation |
| D010859 | Pigmentation Disorders |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
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| ID | Term |
|---|---|
| D010789 | Phototherapy |
| ID | Term |
|---|---|
| D013812 | Therapeutics |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| depigmented vitiligo lesions |
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| study areas with >0% repigmentation |
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