RITLECITINIB TOSILATE for Non-segmental vitiligo

Inclusion criteria

• {"criterion_text":"- Participants must be ≥18 years of age at Screening."}
• {"criterion_text":"- Eligible participants must have at both Screening and BL: •\tA clinical diagnosis of nonsegmental vitiligo for at least 3 months; and •\tBody surface area (BSA) involvement between 4%-60% inclusive, excluding involvements at palms of the hands, soles of the feet, or dorsal aspect of the feet; and •\tBSA ≥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 •\tF-VASI ≥0.5 and T-VASI ≥3; and •\tEither active or stable nonsegmental vitiligo at both Screening and BL visits. All participants who do not have the features of active vitiligo (defined below) will be classified as having stable disease."}

Exclusion criteria

• {"criterion_text":"- Medical conditions pertaining to vitiligo and other diseases/conditions affecting the skin"}
• {"criterion_text":"- History of severe allergic or anaphylactoid reaction to any kinase inhibitor or a known allergy/hypersensitivity to any component (including excipients) of the study intervention."}

Primary endpoints

• {"endpoint_text":"- Part Ia: Response based on F-VASI75 (defined as at least 75% improvement in Facial Vitiligo Area Scoring Index [F-VASI] from BL) at Week 52a","definition_or_measurement_approach":"Defined as at least 75% improvement in F-VASI from baseline; measured at Week 52."}
• {"endpoint_text":"- Part Ia: Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events (AEs) leading to discontinuation","definition_or_measurement_approach":"Safety endpoint based on incidence counts of TEAEs, SAEs, and AEs leading to discontinuation during Part Ia."}
• {"endpoint_text":"- Part Ia: Incidence of clinically significant laboratory abnormalities","definition_or_measurement_approach":"Clinically significant laboratory abnormalities as reported during Part Ia (laboratory safety monitoring)."}
• {"endpoint_text":"- Part Ib: Incidence of TEAEs, SAEs, and AEs leading to discontinuation","definition_or_measurement_approach":"Safety endpoint based on incidence counts of TEAEs, SAEs, and AEs leading to discontinuation during Part Ib (double-blind extension)."}
• {"endpoint_text":"- Part Ib: Incidence of clinically significant laboratory abnormalities","definition_or_measurement_approach":"Clinically significant laboratory abnormalities as reported during Part Ib."}
• {"endpoint_text":"- Part II: Incidence of TEAEs, SAEs, and AEs leading to discontinuation","definition_or_measurement_approach":"Safety endpoint based on incidence counts of TEAEs, SAEs, and AEs leading to discontinuation during Part II (open-label cohort)."}
• {"endpoint_text":"- Part II: Incidence of clinically significant laboratory abnormalities.","definition_or_measurement_approach":"Clinically significant laboratory abnormalities as reported during Part II."}
• {"endpoint_text":"- Part Ia: Response based on T-VASI50 at all time points in the SoA except for those included as primary and key secondary endpointa","definition_or_measurement_approach":"Response based on achieving T-VASI50 (50% improvement in Total Vitiligo Area Scoring Index) at schedule timepoints, excluding those included as primary/key secondary endpoints."}

Secondary endpoints

• {"endpoint_text":"- Part Ia: Response based on T-VASI50 at Weeks 24, 36, and 52a","definition_or_measurement_approach":"T-VASI50 at Weeks 24, 36, and 52."}
• {"endpoint_text":"- Part Ia: Response based on PGIC-F (defined as at least “moderately better” reported change in severity of vitiligo on the face from baseline [BL]) at Weeks 36 and 52a","definition_or_measurement_approach":"Patient Global Impression of Change – Face (PGIC-F); at least 'moderately better' at Weeks 36 and 52."}
• {"endpoint_text":"- Part Ia: Response based on PGIC-V (defined as at least “moderately better” reported change in severity of total body vitiligo from BL) at Weeks 36 and 52a","definition_or_measurement_approach":"Patient Global Impression of Change – Overall Vitiligo (PGIC-V); at least 'moderately better' at Weeks 36 and 52."}
• {"endpoint_text":"- Part Ia: Response based on improvement in PGIS-Fb at Weeks 36 and 52a","definition_or_measurement_approach":"Patient Global Impression of Severity - Face (PGIS-Fb) improvement at Weeks 36 and 52."}
• {"endpoint_text":"- Part Ia: Response based on improvement in PGIS-Vc at Weeks 36 and 52a","definition_or_measurement_approach":"Patient Global Impression of Severity - Vitiligo (PGIS-Vc) improvement at Weeks 36 and 52."}
• {"endpoint_text":"- Part Ia: Response based on F-VASI75 at Weeks 24 and 36a","definition_or_measurement_approach":"F-VASI75 (≥75% improvement in facial VASI) at Weeks 24 and 36."}
• {"endpoint_text":"- Part Ia: Response based on stabilization of disease at all time points after Week 8 in the Schedule of Activities (SoA)","definition_or_measurement_approach":"Stabilization of disease status at SoA timepoints after Week 8."}
• {"endpoint_text":"- Part Ia: Response based on F-VASI75 at all time points in the SoA except for those included as primary and key secondary endpointsa","definition_or_measurement_approach":"F-VASI75 at schedule timepoints excluding those included as primary/key secondary endpoints."}
• {"endpoint_text":"- Part Ia: Response based on T-VASI75 at all time points in the SoA except for that included as a primary endpointa","definition_or_measurement_approach":"T-VASI75 at schedule timepoints excluding the primary endpoint timepoint."}
• {"endpoint_text":"- Part Ia: Response based on sustained improvement in TVASI (defined as maintenance of ≥T-VASI50 from Week 36 to Week 52)","definition_or_measurement_approach":"Sustained maintenance of ≥T-VASI50 from Week 36 to Week 52."}
• {"endpoint_text":"- Part Ia: Response based on sustained improvement in FVASI (defined as maintenance of ≥F-VASI75 from Week 36 to 52)","definition_or_measurement_approach":"Sustained maintenance of ≥F-VASI75 from Week 36 to Week 52."}
• {"endpoint_text":"- Part Ia: Time to rescue medication use","definition_or_measurement_approach":"Time to first use of rescue medication."}
• {"endpoint_text":"- Part Ia: Percent change from baseline (CFB) in F-VASI at all time points in the SoA","definition_or_measurement_approach":"Percent change from baseline in F-VASI at scheduled timepoints."}
• {"endpoint_text":"- Part Ia: Percent CFB in T-VASI at all time points in the SoA","definition_or_measurement_approach":"Percent change from baseline in T-VASI at scheduled timepoints."}
• {"endpoint_text":"- Part Ia: Response based on T-VASI90 (defined as at least 90% improvement in T-VASI from BL) at all time points in the SoA","definition_or_measurement_approach":"T-VASI90 (≥90% improvement) at schedule timepoints."}
• {"endpoint_text":"- Part Ia: Response based on T-VASI100 (defined as 100% improvement in T-VASI from BL) at all time points in the SoA","definition_or_measurement_approach":"T-VASI100 (100% improvement) at schedule timepoints."}
• {"endpoint_text":"- Part Ia: Response based on F-VASI50 (defined as at least 50% improvement in F-VASI from BL) at all time points in the SoA","definition_or_measurement_approach":"F-VASI50 (≥50% improvement) at schedule timepoints."}
• {"endpoint_text":"- Part Ia: Response based on F-VASI75 at all time points in the SoA unless included as primary or secondary endpoints","definition_or_measurement_approach":"F-VASI75 at schedule timepoints unless already evaluated as primary/secondary."}
• {"endpoint_text":"- Part Ia: Response based on F-VASI90 (defined as at least 90% improvement in F-VASI from BL) at all time points in the SoAa","definition_or_measurement_approach":"F-VASI90 at schedule timepoints."}
• {"endpoint_text":"- Part Ia: Response based on F-VASI100 (defined as 100% improvement in F-VASI from BL) at all time points in the SoA","definition_or_measurement_approach":"F-VASI100 at schedule timepoints."}
• {"endpoint_text":"- Part Ia: Response based on improvement in PGIS-Fe at Week 36 unless included as key secondary endpoint","definition_or_measurement_approach":"Improvement in PGIS-Fe measured at Week 36."}
• {"endpoint_text":"- Part Ia: Response based on improvement in PGIS-Vf at Week 36 unless included as key secondary endpoint","definition_or_measurement_approach":"Improvement in PGIS-Vf measured at Week 36."}
• {"endpoint_text":"- Part Ia: Response based on PGIC-F (defined as at least “moderately better” reported change in facial vitiligo from BL) at Weeks 36 and 52 unless included as key secondary endpoint","definition_or_measurement_approach":"PGIC-F at Weeks 36 and 52; at least 'moderately better'."}
• {"endpoint_text":"- Part Ia: Response based on PGIC-V (defined as at least “moderately better” reported change in total body vitiligo from BL) at Weeks 36 and 52 unless included as key secondary endpoint","definition_or_measurement_approach":"PGIC-V at Weeks 36 and 52; at least 'moderately better'."}
• {"endpoint_text":"- Part Ia: CFB in Dermatology Life Quality Index (DLQI) at Week 52","definition_or_measurement_approach":"Change from baseline (CFB) in DLQI at Week 52."}
• {"endpoint_text":"- Part Ia: CFB in the Hospital Anxiety and Depression Scale (HADS) depression subscale at Week 52","definition_or_measurement_approach":"CFB in HADS depression subscale at Week 52."}
• {"endpoint_text":"- Part Ia: CFB in the HADS anxiety subscale at Week 52","definition_or_measurement_approach":"CFB in HADS anxiety subscale at Week 52."}
• {"endpoint_text":"- Part Ia: Response based on a ‘normal’ subscale score indicative of an absence of depression at Week 52 (in participants with BL HADS subscale scores indicative of depression)","definition_or_measurement_approach":"Proportion achieving a 'normal' HADS depression subscale score at Week 52 among participants with baseline scores indicative of depression."}
• {"endpoint_text":"- Part Ia: Response based on a ‘normal’ subscale score indicative of an absence of anxiety at Week 52 (in participants with BL HADS subscale scores indicative of anxiety)","definition_or_measurement_approach":"Proportion achieving a 'normal' HADS anxiety subscale score at Week 52 among participants with baseline scores indicative of anxiety."}
• {"endpoint_text":"- Part Ia: Response based on T-VASI50 at all time points in the SoA except for those included as primary and key secondary endpointa","definition_or_measurement_approach":"T-VASI50 at schedule timepoints excluding primary/key secondary endpoints."}
• {"endpoint_text":"- Part Ib: Response based on T-VASI75 (relative to BL) at all time points in the SoA","definition_or_measurement_approach":"T-VASI75 relative to baseline at schedule timepoints in Part Ib."}
• {"endpoint_text":"- Part Ib: Response based on F-VASI75 (relative to BL) at all time points in the SoA","definition_or_measurement_approach":"F-VASI75 relative to baseline at schedule timepoints in Part Ib."}
• {"endpoint_text":"- Part Ib: Response based on T-VASI50 (relative to BL) at all time points in the SoA","definition_or_measurement_approach":"T-VASI50 relative to baseline at schedule timepoints in Part Ib."}
• {"endpoint_text":"- Part Ib: Response based on stabilization of disease at all time points after Week 60","definition_or_measurement_approach":"Disease stabilization status evaluated at timepoints after Week 60 in Part Ib."}
• {"endpoint_text":"- Part Ib: Response based on F-VASI50 (relative to BL) at all time points in the SoA","definition_or_measurement_approach":"F-VASI50 relative to baseline at schedule timepoints in Part Ib."}
• {"endpoint_text":"- Part Ib: Response based on F-VASI90 (relative to BL) at all time points in the SoA","definition_or_measurement_approach":"F-VASI90 relative to baseline at schedule timepoints in Part Ib."}
• {"endpoint_text":"- Part Ib: Response based on T-VASI90 (relative to BL) at all time points in the SoA","definition_or_measurement_approach":"T-VASI90 relative to baseline at schedule timepoints in Part Ib."}
• {"endpoint_text":"- Part Ib: Response based on T-VASI100 (relative to BL) at all time points in the SoA","definition_or_measurement_approach":"T-VASI100 relative to baseline at schedule timepoints in Part Ib."}
• {"endpoint_text":"- Part Ib: Response based on improvement in PGIS-Fc at Week 104","definition_or_measurement_approach":"Improvement in PGIS-F (country-specific PGIS-Fc) at Week 104."}
• {"endpoint_text":"- Part Ib: Response based on improvement in PGIS-Vd at Week 104","definition_or_measurement_approach":"Improvement in PGIS-V at Week 104."}
• {"endpoint_text":"- Part Ib: Response based on scoring at least “moderately better” in facial vitiligo on PGIC-F at Week 104","definition_or_measurement_approach":"PGIC-F scoring 'moderately better' at Week 104."}
• {"endpoint_text":"- Part Ib: Response based on scoring at least “moderately better” in total body vitiligo on PGIC-V at Week 104","definition_or_measurement_approach":"PGIC-V scoring 'moderately better' at Week 104."}
• {"endpoint_text":"- Part II: Response based on T-VASI75 at all time points in the SoA","definition_or_measurement_approach":"T-VASI75 at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on F-VASI75 at all time points in the SoA","definition_or_measurement_approach":"F-VASI75 at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on T-VASI50 at all time points in the SoA","definition_or_measurement_approach":"T-VASI50 at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on stabilization of disease at all time points after Week 8 in the SoA","definition_or_measurement_approach":"Disease stabilization assessed at schedule timepoints after Week 8 in Part II."}
• {"endpoint_text":"- Part II: Response based on sustained improvement in T- VASI","definition_or_measurement_approach":"Sustained improvement in T-VASI (maintenance criteria defined in protocol) in Part II."}
• {"endpoint_text":"- Part II: Response based on sustained improvement in F- VASI","definition_or_measurement_approach":"Sustained improvement in F-VASI in Part II."}
• {"endpoint_text":"- Part II: Time to rescue medication use","definition_or_measurement_approach":"Time to first use of rescue medication in Part II."}
• {"endpoint_text":"- Part II: Percent CFB in F-VASI at all time points in the SoA","definition_or_measurement_approach":"Percent change from baseline in F-VASI at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Percent CFB in T-VASI at all time points in the SoA","definition_or_measurement_approach":"Percent change from baseline in T-VASI at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on T-VASI90 at all time points in the SoA","definition_or_measurement_approach":"T-VASI90 at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on T-VASI100 at all time points in the SoA","definition_or_measurement_approach":"T-VASI100 at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on F-VASI50 at all time points in the SoA","definition_or_measurement_approach":"F-VASI50 at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on F-VASI90 at all time points in the SoA","definition_or_measurement_approach":"F-VASI90 at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on F-VASI100 at all time points in the SoA","definition_or_measurement_approach":"F-VASI100 at schedule timepoints in Part II."}
• {"endpoint_text":"- Part II: Response based on PGIC-F (defined as at least “moderately better” reported change in facial vitiligo from BL) at Weeks 36 and 52","definition_or_measurement_approach":"PGIC-F at Weeks 36 and 52 in Part II."}
• {"endpoint_text":"- Part II: Response based on PGIC-V (defined as at least “moderately better” reported change in total body vitiligo from BL) at Weeks 36 and 52","definition_or_measurement_approach":"PGIC-V at Weeks 36 and 52 in Part II."}
• {"endpoint_text":"- Part II: Response based on improvement in PGIS-Fb at Weeks 36 and 52","definition_or_measurement_approach":"PGIS-Fb improvement at Weeks 36 and 52 in Part II."}
• {"endpoint_text":"- Part II: Response based on improvement in PGIS-Vc at Weeks 36 and 52","definition_or_measurement_approach":"PGIS-Vc improvement at Weeks 36 and 52 in Part II."}
• {"endpoint_text":"- Part II: CFB in DLQI at Week 52","definition_or_measurement_approach":"CFB in DLQI at Week 52 in Part II."}
• {"endpoint_text":"- Part II: CFB in the HADS depression subscale at Week 52","definition_or_measurement_approach":"CFB in HADS depression subscale at Week 52 in Part II."}
• {"endpoint_text":"- Part II: CFB in the HADS anxiety subscale at Week 52","definition_or_measurement_approach":"CFB in HADS anxiety subscale at Week 52 in Part II."}
• {"endpoint_text":"- Part II: Response based on a ‘normal’ subscale score indicative of an absence of depression at Week 52 (in participants with BL HADS subscale scores indicative of depression)","definition_or_measurement_approach":"Proportion achieving 'normal' HADS depression subscale at Week 52 among those with baseline depression."}
• {"endpoint_text":"- Part II: Response based on a ‘normal’ subscale score indicative of an absence of anxiety at Week 52 (in participants with BL HADS subscale scores indicative of anxiety)","definition_or_measurement_approach":"Proportion achieving 'normal' HADS anxiety subscale at Week 52 among those with baseline anxiety."}

Methods

• Online/social media outreach — materials titled 'Online SocialMediaOutreach' and country-specific online advertising materials (digital adverts, ClinLife/Online outreach).
• ClinLife platform / ClinLife notes — use of ClinLife platform and related EC notes for recruitment.
• HCP referral / Doctor referral letters — HCP referral letters and referral fact cards for clinician-driven recruitment.
• Study brochures, posters and patient invite letters — study brochures, posters, patient invite letters provided as recruitment materials.
• Scout/ScoutPass and reloadable card incentives — 'ScoutPass Reloadable' materials and patient-facing landing pages described in recruitment documents.
• Digital patient-facing landing pages and eConsent/electronic materials — eConsent submission letters, IQVIA patient-facing landing pages and eConsent guidance included.

Germany

Earliest CTIS Part Ii Submission Date

04-03-2024

Latest Decision Or Authorization Date

11-10-2024

Processing Time Days

221

Number Of Participants

60

Belgium

Earliest CTIS Part Ii Submission Date

05-03-2024

Latest Decision Or Authorization Date

11-10-2024

Processing Time Days

220

Number Of Participants

16

Italy

Earliest CTIS Part Ii Submission Date

19-01-2024

Latest Decision Or Authorization Date

16-01-2025

Processing Time Days

363

Number Of Participants

30

Poland

Earliest CTIS Part Ii Submission Date

29-02-2024

Latest Decision Or Authorization Date

30-07-2025

Processing Time Days

517

Number Of Participants

97

Hungary

Earliest CTIS Part Ii Submission Date

02-02-2024

Latest Decision Or Authorization Date

24-07-2025

Processing Time Days

538

Number Of Participants

38

Bulgaria

Earliest CTIS Part Ii Submission Date

05-03-2024

Latest Decision Or Authorization Date

24-11-2025

Processing Time Days

629

Number Of Participants

41

Slovakia

Earliest CTIS Part Ii Submission Date

19-03-2024

Latest Decision Or Authorization Date

18-02-2026

Processing Time Days

701

Number Of Participants

49

Spain

Earliest CTIS Part Ii Submission Date

02-02-2024

Latest Decision Or Authorization Date

04-02-2026

Processing Time Days

733

Number Of Participants

24

Primary sponsor

Full Name

Pfizer Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

CRS Clinical Research Services Management GmbH