| Protocol No: | ECCT/24/04/05 | Date of Protocol: | 21-03-2024 |
| Study Title: |
A Double-Arm, Open Label, Phase III Study to Compare the
Efficacy and Safety of SCENESSE® and Narrow-Band Ultraviolet B (NB-UVB) Light versus NB-
UVB Light Alone in the Treatment of Vitiligo
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| Study Objectives: |
Primary objective
To evaluate the efficacy of SCENESSE® and NB-UVB compared to NB-UVB alone in repigmentation of vitiligo on the body after 20 weeks of treatment using Total Vitiligo Area Scoring Index (T-VASI) (excluding hands and feet)
Secondary objectives
•To determine the safety of SCENESSE® and NB-UVB light treatment in participants with vitiligo
•To evaluate the efficacy of SCENESSE® and NB-UVB compared to NB-UVB alone in repigmentation of vitiligo on the face using F-VASI (Facial VASI) (excluding scalp, ears, neck or lips)
•To compare the maintenance of pigmentation achieved with SCENESSE® and NB-UVB versus NB-UVB alone in participants with vitiligo using T-VASI (excluding hands and feet) and F-VASI (excluding scalp, ears, neck or lips)
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| Laymans Summary: | Vitiligo is the most common depigmentation disorder (colour loss of the skin), is an acquired disorder characterised by a chronic (long-term) and progressive (worsening) ability for cells in the skin, called melanocytes to produce skin pigment. There is still no cure for vitiligo. The purpose of this Phase III study is to evaluate if afamelanotide combined with NB-UVB can improve repigmentation and for how long the pigmentation is maintained after the treatment is complete. Additionally, the study will investigate the safety of afamelanotide in combination with NB-UVB Light usage in participant with vitiligo. Afamelanotide is an artificially produced drug. It is the synthetic form of the human alpha-melanocyte stimulating hormone (α-MSH), a naturally occurring hormone in the body that stimulates your colour producing cells in your skin. Afamelanotide works similarly to the natural hormone; causing skin cells to stimulate production of the pigment and the protective substance called melanin. Afamelanotide increases skin pigmentation faster and more effectively than the natural hormone, providing protection against light and sun, which is also known as photoprotection. Up to 200 participants from different countries will be enrolled in this study. Study treatment is planned for 20 weeks and follow up will take up to six (6) months.
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| Abstract of Study: | Title: A Double-Arm, Open Label, Phase III Study to Compare the Efficacy and Safety of SCENESSE® and Narrow-Band Ultraviolet B (NB-UVB) Light versus NB-UVB Light Alone in the Treatment of Vitiligo Rationale: Vitiligo, the most common depigmentation disorder, affecting people worldwide with a prevalence of 0.1 to 2%, is an acquired disorder characterized by a chronic and progressive loss of functional epidermal and/or hair follicle melanocytes1,2. The disease usually begins in childhood or young adulthood with a peak onset at 10-30 years2,3. Both genders are equally affected, and there are no apparent differences in rates of occurrence according to skin type or race. This double-arm, open label, Phase III study is proposed to compare the efficacy and safety of the SCENESSE® implants administered every three weeks and NB-UVB in patients with vitiligo. Afamelanotide, similar to α-MSH, is unique in that it exclusively targets many cutaneous effector cells including those that play a key role in vitiligo. In addition to resident cells of the epidermis (melanocytes, keratinocytes), MC1R expressing inflammatory cells (neutrophils and lymphocytes) may also be targeted by afamelanotide. Restoring the deficient MC system in vitiligo with afamelanotide is expected to be of a therapeutic value for vitiligo. Indeed, by mimicking the effects of α-MSH, afamelanotide is expected to exert the same effects on the skin cells as the physiological hormone. In addition, afamelanotide has a stronger binding affinity to the MC1R and a slower dissociation rate than the natural occurring α-MSH. Primary Objective: To evaluate the efficacy of SCENESSE® and NB-UVB compared to NB-UVB alone in repigmentation of vitiligo on the body after 20 weeks of treatment using Total Vitiligo Area Scoring Index (T-VASI) (excluding hands and feet) Secondary Objective: To determine the safety of SCENESSE® and NB-UVB light treatment in participants with vitiligo
• To evaluate the efficacy of SCENESSE® and NB-UVB compared to NB-UVB alone in repigmentation of vitiligo on the face using F-VASI (Facial VASI) (excluding scalp, ears, neck or lips)
• To compare the maintenance of pigmentation achieved with SCENESSE® and NB-UVB versus NB-UVB alone in participants with vitiligo using T-VASI (excluding hands and feet) and F-VASI (excluding scalp, ears, neck or lips)
Study design and overal plan for the study: This is a double arm, open label, 20-week Phase III study with three and six-month follow-up periods, in participants with a documented history of generalized vitiligo.
Up to 200 eligible participants will be enrolled globally and randomised in equal numbers to one of the following treatment groups:
• Group A will receive NB-UVB twice weekly from Day 0 (40 treatments in total), and SCENESSE® (one implant administered on Days 0, 21 (±4), 42 (±4), 63 (±4), 84 (±4), 105 (±4) and 126 (±4) (seven implants in total));
• Group B will receive NB-UVB light only (administered twice weekly for 20 weeks, 40 treatments in total).
No. of participants planned: Up to 100 participants per treatment group, with a documented medical history of vitiligo on the face and body
The target recruitment for the KNH is 10 to 20 participants from KNH dermatology clinic.
Study period: approximately 12 months (20 weeks treatment plus three and six-month follow-up) including a screening period of up to 28 days.
Statistical analysis: A formal statistical analysis plan (SAP) will be prepared prior to completion of the study and before data analysis is undertaken. A gatekeeping procedure will be implemented for the primary and key secondary analyses to control the overall Type I error rate at 5%.
Safety Analyses: Treatment-emergent adverse events (TEAEs), including clinically significant changes in haematology, serum chemistry and urinalysis measurements from Screening Visit to Days 140 or Early Termination (if applicable), will be summarized by MedDRA preferred term and body system for each treatment group. TEAEs will be further summarized by intensity, seriousness, outcome and relationship to study drug.
During the follow up phase, on Days 224 and 308, adverse events will be recorded and routine laboratory assessments performed.
Full anterior and posterior photography performed at Screening or Day 0 and Days 140 and 308 or Early Termination (if applicable). Examination of the skin and oral mucosa will be performed on Days 0, 56, 126, 140, 224 and 308 or Early Termination (if applicable).
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