A Phase 3 Study Investigating the Efficacy, Safety, and Tolerability of Ritlecitinib in Participants with Nonsegmental Vitiligo

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Pfizer Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17], Diseases [C] - Immune System Diseases [C20]

Trial duration

26 Jun 2023 → 6 Feb 2026

Decision date (initial)

2023-04-05

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

External identifiers

EU CT number

2022-501668-16-00

ClinicalTrials.gov

NCT05583526

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To compare the efficacy of ritlecitinib 50 mg QD versus placebo in participants with nonsegmental vitiligo; To evaluate the safety and tolerability of ritlecitinib over time in participants with nonsegmental vitiligo.

Secondary objectives 6

1. To compare the efficacy of ritlecitinib 50 mg QD versus placebo on F-VASI75 at Weeks 24 and 36 in participants with nonsegmental vitiligo
2. To compare the efficacy of ritlecitinib 50 mg QD versus placebo on T-VASI50 at Weeks 24, 36, and 52 in participants with nonsegmental vitiligo
3. To compare the efficacy of ritlecitinib 50 mg QD versus placebo on % CFB in F-VASI at Weeks 24, 36, and 52
4. To compare the efficacy of ritlecitinib 50 mg QD versus placebo on % CFB in T-VASI at Weeks 24, 36, and 52
5. To compare the efficacy of ritlecitinib 50 mg QD versus placebo on PGIS-F at Weeks 24, 36, and 52 in participants with nonsegmental vitiligo
6. To compare the efficacy of ritlecitinib 50 mg QD versus placebo on PGIS-V at Weeks 24, 36, and 52 in participants with nonsegmental vitiligo

Conditions and MedDRA coding

NON SEGMENTAL VITILIGO

Study design 1 period

Regulatory references

Scientific advice from competent authorities

Medicines And Healthcare Products Regulatory Agency, Pharmaceuticals And Medical Devices Agency, Food And Drug Administration, European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002451-PIP03-22

Plan to share IPD

Yes

IPD plan description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Participants ≥18 years of age at screening. Adolescents (12 to <18 years of age) are also eligible for this study, but only if approved by the local IRB/EC and regulatory health authority. Where these approvals have not been granted, only participants ≥18 years of age will be enrolled.
2. Eligible participants must have at both Screening and Baseline: A clinical diagnosis of nonsegmental vitiligo for at least 3 months; and BSA involvement 4%-60% inclusive, excluding involvements at palms of the hands, soles of the feet, or dorsal aspect of the feet; and BSA ≥0.5% involvement on the face (face is defined as including the area on the forehead to the original hairline, on the cheek vertically to the jawline, and laterally from the corner of the mouth to the tragus. The face will not include scalp, ears, neck, or surface area of the lips, but will include the nose and the eyelids); and F-VASI ≥0.5 & T-VASI ≥3; and Either active or stable disease nonsegmental vitiligo at both Screening and Baseline visits. All participants who do not have the features of active vitiligo (defined below) are required to have stable disease.
3. Participants must agree to stop all other treatments for vitiligo from Screening through the final follow-up visit.

Exclusion criteria 3

1. Medical conditions pertaining to vitiligo and other diseases/conditions affecting the skin.
2. History of severe allergic or anaphylactoid reaction to any kinase inhibitor.
3. Adolescent participants 12 to <18 years of age without one of the following: Documented evidence from a health professional of having received varicella vaccination (2 doses); or Evidence of prior exposure to VZV based on serological testing (ie, a positive VZV IgG Ab result) at screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Response based on F-VASI75 (defined as at least 75% improvement in F-VASI from BL) at Week 52; • Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events (AEs) leading to discontinuation. • Incidence of clinically significant laboratory abnormalities

Secondary endpoints 6

1. Response based on F-VASI75 at Weeks 24 and 36
2. Response based on T-VASI50 at Weeks 24, 36, and 52
3. % CFB in F-VASI at Weeks 24, 36, and 52.
4. % CFB in T-VASI at Weeks 24, 36, and 52.
5. Response based on improvement in PGIS-Fb at Weeks 24, 36, and 52
6. Response based on improvement in PGIS-Vc at Weeks 24, 36, and 52

Investigational products

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

Test 1

Placebo 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Pfizer Inc.

Sponsor organisation

Pfizer Inc.

Address

66 Hudson Boulevard East

City

New York

Postcode

10001-2189

Country

United States

Scientific contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Public contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Third parties 1

Locations

5 EU/EEA countries · 20 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 273 files

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

Application history

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