E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10047642 |
E.1.2 | Term | Vitiligo |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The priniciple research question is to compare NB-UVB light therapy to a potent topical steroid ointment, to see which treatment is safer and which treatment is more effective in improving the appearance of vitiligo.
PRIMARY OBJECTIVE To assess the safety and effectiveness of: • NB-UVB light compared to potent-strength topical corticosteroid • The combination of NB-UVB light plus topical corticosteroid, compared to topical corticosteroid alone
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E.2.2 | Secondary objectives of the trial |
SECONDARY OBJECTIVES • To assess whether treatment response (if any) is maintained once the treatment is stopped.
• To compare the cost-effectiveness of the treatments.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patients 5 years of age or over with a diagnosis of non-segmental vitiligo confirmed by a dermatologist
2. Vitiligo limited to less than 10% of body surface area, with at least one patch that is reported by the participant to have been active (new onset or spread) in the last 12 months.
3. No other active therapy for vitiligo (or willing to stop current treatment – no washout period required).
4. Able to administer the intervention safely at home
5. Able and willing to give informed consent (or parental/guardian consent in the case of children).
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E.4 | Principal exclusion criteria |
1. Other types of vitiligo (e.g. segmental or universal vitiligo).
2. Patients with vitiligo limited to areas of the body for which NB-UVB light therapy or potent topical corticosteroids would be inappropriate (e.g. around the genitals).
3. History of skin cancer (ever).
4. History of radiotherapy use (ever).
5. Photosensitivity (e.g. lupus, polymorphic light eruption, solar urticaria, chronic actinic dermatitis, actinic prurigo, porphyria or other photosensitivity disorders e.g. dermatomyositis)
6. Pregnant, breastfeeding or likely to become pregnant during the 9-month treatment period.
7. Current use of immunosuppressive drugs (e.g. e.g. ciclosporin, azathioprine, mycophenolate mofetil, methotrexate, systemic tacrolimus)
8. Allergy or contraindication to mometasone furoate or any of its components (e.g any cutaneous bacterial, viral or fungal infections in the area to be exposed to trial treatments).
9. Current participation in another clinical trial or intervention study
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary outcome measure is participant-reported treatment success, for a target lesion.
Treatment success is defined as participants reporting that their vitiligo is either “a lot less noticeable” or “no longer noticeable” when asked the question:
"Compared to before treatment, how noticeable is the vitiligo now?"
More noticeable (1) As noticeable (2) Slightly less noticeable (3) A lot less noticeable (4) No longer noticeable (5)
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
The primary outcome measure will be assessed at 9 months. |
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E.5.2 | Secondary end point(s) |
1. Investigator-assessed onset of treatment response (including cessation of spread) for each assessed patch of vitiligo
“Compared to before treatment, has there been a change in the vitiligo?”
• Further skin colour lost (1) • No change in skin colour (2) • Some skin colour returned (3)
2. Participant-reported treatment success for each body region: Assessed by noticeability quesiton.
3. Percentage repigmentation of assessed lesions.
4. Quality of life (assessed by the following questionnaires: VitiQOL, Skindex 29, EQ-5D-5L, CHU 9D).
5. Time burden of treatment.
6. Within trial cost-effectiveness analysis. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Secondary Endpoint 1: 3, 6, 9 months.
Secondary Endpoint 2: 3, 6, 9, 12, 15, 18 and 21 months.
Secondary Endpoint 3: 3, 6, 9 months.
Secondary Endpoint 4: 9 and 21 months.
Secondary Endpoint 5: 3, 6, 9 months.
Secondary Endpoint 6: end of trial. |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
Dermfix 1000MX Light Therapy Device (active and dummy devices) |
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E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 15 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The end of the trial is the last 21 month questionnaire of the last participant randomised. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | 31 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 29 |