Efficacy and tolerability of the combination of anifrolumab (300 mg IV) and phototherapy versus phototherapy in adults with progressive vitiligo

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Universitaire De Bordeaux

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17], Diseases [C] - Nervous System Diseases [C10]

Trial duration

27 Nov 2023 → ongoing

Decision date (initial)

2024-09-18

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-512041-17-00

EudraCT number

2022-002003-37

ClinicalTrials.gov

NCT05917561

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate the efficacy of the combination of anifrolumab intravenous (IV) every four weeks + UVB TL01 (twice a week) by the evaluation of the percentage of skin repigmentation after 36 weeks of treatment using the VASI score in the experimental group receiving anifrolumab + UVB TL01.

Secondary objectives 20

1. To evaluate the efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) by the evaluation of the percentage of skin repigmentation after 12 weeks of treatment using the VASI score in the experimental group receiving anifrolumab + UVB TL01.
2. To evaluate safety and tolerability of the combination of anifrolumab IV every four weeks + UVB TL01
3. To evaluate fficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the score Vitiligo European Task Force (VETF).
4. To evaluate efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the Vitiligo Extent Score (VES).
5. To evaluate efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the Face Vitiligo Area Scoring Index (F-VASI).
6. To efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the Vitiligo Signs of Activity Score (VSAS).
7. To evaluate improvement of the quality of life of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the DLQI, SkinDex29 and the Vitiligo Impact Scale (VIPs)
8. To evaluate change in blood and skin biomarkers of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week).
9. To evaluate efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the Vitiligo Noticeability scale (VNS) score.
10. To evaluate efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the Phycisian’s Global Impression of Change-Vitiligo (PhGIC-V)
11. To evaluate efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the Patient’s Global Impression of Change-Vitiligo (PaGIC-V)
12. To evaluate efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the Total-Phycisian’s Global Vitiligo assessment (T-PhGVA)
13. To evaluate efficacy of the combination of anifrolumab IV every four weeks + UVB TL01 (twice a week) using the Total-Patient’s Global Vitiligo Assessment (T-PaGVA-V)
14. To evaluate 48 weeks after inclusion (12 weeks after stopping the experimental procedure) the recurrence of vitiligo using the VASI score.
15. To evaluate 48 weeks after inclusion (12 weeks after stopping the experimental procedure) the recurrence of vitiligo using the Vitiligo European Task Force (VETF) score.
16. To evaluate 48 weeks after inclusion (12 weeks after stopping the experimental procedure) the recurrence of vitiligo using the Vitiligo Extent Score (VES).
17. To evaluate 48 weeks after inclusion (12 weeks after stopping the experimental procedure) the recurrence of vitiligo using the Face Vitiligo Area Scoring Index (F-VASI).
18. To evaluate 48 weeks after inclusion (12 weeks after stopping the experimental procedure) the activity of vitiligo using the Vitiligo Signs of Activity Score (VSAS).
19. To evaluate 48 weeks after inclusion (12 weeks after stopping the experimental procedure) the impact on quality of life of the using the DLQI, SkinDex29 and the Vitiligo Impact Scale (VIPs)
20. To evaluate 48 weeks after inclusion (12 weeks after stopping the experimental procedure) the recurrence of vitiligo using VNS score, PhGIC-V, PaGIC-V, T-PhGVA, T-PaGVA

Conditions and MedDRA coding

Patient with non-segmental (symmetrical) vitiligo with a body surface area involved >5% excluding hands and feet

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. Subject male or female aged ≥ 18 years and ≤ 65 years
2. Subject with body weight > or = 40kg
3. Diagnosis of non-segmental (symmetrical) vitiligo with a body surface area involved >5% excluding hands and feet
4. Active non-segmental vitiligo is defined by: • Non-segmental vitiligo with new patches or extension of old lesions during the last 6 months AND • Presence of hypochromic aspect under Wood’s lamp examination and/or perifollicular hypopigmentation under Wood’s lamp examination.
5. Able to read, understand, and give documented (electronic or paper signature) informed consent
6. Affiliated or beneficiary of the French Social Security
7. Agree to discontinue the use of the following excluded medications/treatments for at least 4 weeks prior to randomization (Visit 2): phototherapy.
8. Agree to discontinue the use of the following excluded medications/treatments for at least 4 weeks prior to randomization and throughout the study: systemic steroids, methotrexate, cyclosporine, mycophenolate mofetil, azathioprine.
9. Agree to discontinue the use of the following excluded medications for at least 2 weeks prior to randomization and throughout the study: • TCS or topical immune modulators (e.g., tacrolimus or pimecrolimus) • Topical phosphodiesterase type 4 (PDE-4) inhibitor (crisaborole) • Topical JAK inhibitor (e.g., tofacitinib or ruxolitinib) and/or any other investigational topical treatments.

Exclusion criteria 7

1. Segmental or mixed vitiligo
2. Are currently experiencing or have a history of other concomitant skin conditions (e.g., psoriasis or lupus erythematosus) that would interfere with evaluations of the effect of study medication on vitiligo.
3. Patients who are currently experiencing a skin infection that requires treatment, or who are currently being treated, with topical or systemic antibiotics. Note: Patients may not be rescreened until at least 4 weeks after the date of their previous screen failure and at least 2 weeks after resolution of the infection.
4. Patients with history of basal cell or squamous epithelial skin cancer or melanoma
5. Presence of significant uncontrolled neuropsychiatric disorder, are clinically judged by the investigator to be at risk for suicide.
6. Have any serious concomitant illness that is anticipated to require the use of systemic corticosteroids or otherwise interfere with study participation or require active frequent monitoring (e.g., unstable chronic asthma).
7. Current alcohol, drug, or chemical abuse.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Mean variation in percentage of the Vitiligo Area Scoring Index (VASI) score between baseline and week 36.

Secondary endpoints 15

1. The safety and tolerability of anifrolumab and phototherapy will be assessed based on clinical and biological exams.
2. Mean variation in percentage of the Vitiligo Area Scoring Index (VASI) score between baseline, week 12, 24 and 48.
3. Mean variation in percentage of Face Vitiligo Area Scoring Index (F-VASI) score between baseline , week 12, 24, 36 and 48
4. Mean variation in percentage of Vitiligo European Task Force (VETF) score between baseline , week 12, 24, 36 and 48
5. Mean variation in percentage of Vitiligo Extent Score (VES) score between baseline , week 12, 24, 36 and 48
6. Evolution of the activity of vitiligo will be assessed by measuring the variation in percentage of the Vitiligo Signs of Activity Score (VSAS) between baseline , week 12, 24, 36 and 48
7. Variation of the Dermatology Life Quality Index (DLQI) between baseline, week 12, 24, 36 and 48
8. Variation of the Score of the Skindex 29 between inclusion, week 12, 24, 36 and 48
9. Variation of the Vitiligo Impact Scale (VIPs) between inclusion, week 12, 24, 36 and 48 weeks
10. Evolution of vitiligo noticeability scale (VNS) score between inclusion, week 12, 24, 36 and 48
11. Evolution of Physician's Global Impression of Change- Vitiligo (PhGIC-V) between inclusion, week 12, 24, 36 and 48
12. Evolution of Patient's Global Impression of Change-Vitiligo (PaGIC-V) between inclusion, week 12, 24, 36 and 48
13. Evolution of Total – Physician Global Vitiligo Assessment (T-PhGVA) between inclusion, week 12, 24, 36 and 48
14. Evolution of Total – Patient Global Vitiligo Assessment (T-PaGVA) between inclusion, week 12, 24, 36 and 48
15. Blood inflammatory markers will be measured at inclusion, week 12, 24, 36 weeks using multiplex ELISA on patients’ serum Skin inflammatory markers will be measured at inclusion, 12 and 36 weeks using immunofluorescence on skin biopsies, and transcriptomic analysis on skin biopsies.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire De Bordeaux

Sponsor organisation

Centre Hospitalier Universitaire De Bordeaux

Address

1 Rue Jean Burguet

City

Bordeaux

Postcode

33000

Country

France

Scientific contact point

Organisation

Centre Hospitalier Universitaire De Bordeaux

Contact name

Coordinating investigator

Public contact point

Organisation

Centre Hospitalier Universitaire De Bordeaux

Contact name

Coordinating investigator

Locations

1 EU/EEA country · 5 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications