Study of Efficacy, Safety and Tolerability of Crisaborole and PF-07038124 With and Without NBUVB in Vitiligo

NCT05298033 | Completed Phase 2 FDA Drug FDA Device

• 1 other identifier: 21-4837
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase 2A clinical trial designed to test the pro-melanogenic and anti-inflammatory role of phosphodiesterase-4 inhibitors (PDE4i), alone and in combination with active narrow band UVB (NBUVB), in vitiligo lesions. This is a double-blind, randomized controlled trial (RCT) with six study arms. The goal is for 64 participants to be recruited and complete the study.

Conditions

Vitiligo

Keywords

Vitiligo; PDE-4 inhibitor; Phototherapy; Repigmentation

Outcome Measures

Primary Outcomes (1)

• Proportion of participants achieving at 50% or greater improvement from baseline in facial Vitiligo Area Scoring Index (F)-VASI

  Assessment of facial repigmentation via changes in depigmented areas

  6 months (week 24)

Secondary Outcomes (4)

• Proportion of participants achieving 90% or greater improvement from baseline in F-VASI

  6 months (week 24)

• Proportion of participants achieving 50% or greater improvement from baseline in total (T)-VASI

  6 months (week 24)

• Proportion of participants achieving a Vitiligo Noticeability Scale (VNS) rating of "a lot less noticeable" or "no longer noticeable"

  6 months (week 24)

• Percentage change from baseline in facial segment of Body Surface Area affected (F-BSA)

  6 months (24 weeks)

Other Outcomes (3)

• Quantification of pigment via Fontana-Masson staining

  Baseline (day 0, pre-treatment) and 3 months (12 weeks)

• Assessment of melanocyte population expansion via immunohistochemistry (IHC) studies

  Baseline (day 0, pre-treatment) and 3 months (12 weeks)

• Assessment of percent change from baseline in serum key inflammatory chemokines

  Baseline (pre-treatment) and at 3 months (12 weeks) and 6 months (24 weeks)

Study Arms (6)

Active NBUVB plus Crisaborole 2% topical ointment

EXPERIMENTAL

12 participants will receive 6 months of treatment with crisaborole 2% topical along with 6 months of treatment with active NBUVB

Drug: Crisaborole 2 % Topical Ointment; Device: NBUVB phototherapy

Active NBUVB plus PF-07038124 0.01% topical ointment

EXPERIMENTAL

12 participants will receive 6 months of treatment with active NBUVB, to be combined with 3 months of treatment with PF-07038124 0.01% topical ointment followed by 3 months of vehicle ointment

Drug: PF-07038124 0.01% topical ointment; Device: NBUVB phototherapy; Drug: Vehicle

Active NBUVB plus vehicle ointment

ACTIVE COMPARATOR

8 participants will receive 6 months of treatment with active NBUVB combined with 6 months of vehicle ointment application

Device: NBUVB phototherapy; Drug: Vehicle

Sham phototherapy plus crisaborole 2% topical ointment

EXPERIMENTAL

12 participants will receive 6 months of treatment with crisaborole 2% topical ointment combined with 6 months of sham phototherapy

Drug: Crisaborole 2 % Topical Ointment; Device: Sham phototherapy

Sham phototherapy plus PF-07038124 0.01% topical ointment

EXPERIMENTAL

12 participants will receive 3 months of treatment with PF-07038124 0.01% topical ointment, followed by 3 months of vehicle ointment, along with 6 months of sham phototherapy

Drug: PF-07038124 0.01% topical ointment; Device: Sham phototherapy; Drug: Vehicle

Sham phototherapy plus vehicle ointment

PLACEBO COMPARATOR

8 participants will receive 6 months of sham phototherapy combined with 6 months of vehicle ointment application

Device: Sham phototherapy; Drug: Vehicle

Interventions

Crisaborole 2 % Topical Ointment DRUG

Twice daily crisaborole 2% topical ointment

Also known as: Eucrisa

Active NBUVB plus Crisaborole 2% topical ointment; Sham phototherapy plus crisaborole 2% topical ointment

PF-07038124 0.01% topical ointment DRUG

Twice daily PF-07038124 0.01% topical ointment

Active NBUVB plus PF-07038124 0.01% topical ointment; Sham phototherapy plus PF-07038124 0.01% topical ointment

NBUVB phototherapy DEVICE

Home narrow band UVB phototherapy exposure sessions 3 times per week

Also known as: Narrow band UVB

Active NBUVB plus Crisaborole 2% topical ointment; Active NBUVB plus PF-07038124 0.01% topical ointment; Active NBUVB plus vehicle ointment

Sham phototherapy DEVICE

Non-NBUVB visible light radiation exposure sessions 3 times per week

Sham phototherapy plus PF-07038124 0.01% topical ointment; Sham phototherapy plus crisaborole 2% topical ointment; Sham phototherapy plus vehicle ointment

Vehicle DRUG

Twice daily vehicle ointment

Active NBUVB plus PF-07038124 0.01% topical ointment; Active NBUVB plus vehicle ointment; Sham phototherapy plus PF-07038124 0.01% topical ointment; Sham phototherapy plus vehicle ointment

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Active or stable non-segmental vitiligo at Screening and Baseline visits:
• A clinical diagnosis of non-segmental vitiligo (vitiligo vulgaris or acrofacial vitiligo) for at least 3 months, AND
• Body Surface Area affected (BSA) involvement 3%-90%, excluding involvement of scalp, palms of the hands, soles of the feet, AND
• BSA \>= 0.25% involvement of the facial area, AND
• Minimum Facial Vitiligo Area Scoring Index (F-VASI) \>=0.25% and Total VASI \>=3%
• Must agree that the treatment area will involve 3%-25% BSA
• If receiving concomitant medication for any reason other than vitiligo, must be on a stable regimen (no new drug or dosage changes within 7 days of baseline visit) and willing to remain on stable regimen for duration of the study
• Must agree to stop all other treatments for vitiligo from screening through 1 week after discontinuation of study drug treatment
• Must be capable of giving signed informed consent and comply with the requirements and restrictions as listed in the informed consent document and protocol
• Must agree to avoid exposure to the sun as much as possible and not to use tanning booths, sun lamps, or other ultraviolet light sources other than requested by the study team during the study

You may not qualify if:

• Pregnant or breastfeeding females
• Females of childbearing potential who are unwilling or unable to use contraception for the duration of the study and for at least 28 days after the last dose of study intervention
• Other types of vitiligo that do not meet criteria for active or stable or non-segmental vitiligo, including segmental, mucosal, focal, and mixed vitiligo, and those with vitiligo universalis
• Active forms of other hypo- or depigmentation, as detailed in the protocol
• Active forms of inflammatory skin disease(s) associated with hypo- or depigmentation at the time of screening or baseline that, in the opinion of the investigator, would interfere with evaluation of vitiligo or response to treatment
• Leukotrichia in more than 33% of the facial area affected with vitiligo lesions OR leukotrichia in more than 33% of the total BSA affected with vitiligo lesions
• History of transplantation procedure for vitiligo at any point
• History of any skin bleaching treatment for vitiligo or other dermatoses at any point
• Active acute or chronic skin infection requiring treatment with systemic antibiotics, antivirals, antiparasitics, antiprotozoals, or antifungals within 4 weeks prior to first drug application (baseline), OR superficial skin infections within 2 weeks prior to first drug application (NOTE: MAY BE RESCREENED AFTER INFECTION RESOLUTION)
• Known history of severe allergic or anaphylactoid reaction to any PDE4 inhibitors or lidocaine
• Documented lack of response to prior PDE4 inhibitor therapy
• Presence of other severe, progressive, or uncontrolled diseases, including but not limited to renal, hepatic, cardiac, pulmonary, endocrine, immunological/rheumatological, hematological, gastrointestinal, metabolic, neurologic, psychiatric, immunodeficiency (including HIV positive serology), OR significant laboratory abnormalities that would increase risk associated with study participation or interfere with interpretation of study results, or in the opinion of the investigator, the participant is inappropriate for entry into the study, or unwilling/unable to comply with protocol-specified assessments and lifestyle considerations
• Any malignancies or history of skin malignancies, excluding adequately treated or excised non-metastatic basal cell or squamous cell skin cancer, or cervical carcinoma in situ
• Significant trauma or major surgery 1 month prior to screening or considered in imminent need of surgery during the study
• Alcohol or substance abuse within 6 months of screening that in the opinion of the investigator would preclude participation or adherence in the study

Sponsors & Collaborators

• University of Colorado, Denver: lead
• Pfizer: collaborator

Study Sites (1)

University of Colorado Anschutz - Clinical and Translational Research Centers

Aurora, Colorado, 80045, United States

Location

MeSH Terms

Conditions

Vitiligo

Interventions

crisaborole; Ointments

Condition Hierarchy (Ancestors)

Hypopigmentation; Pigmentation Disorders; Skin Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Study Officials

• Stanca Birlea, MD

  University of Colorado, Denver

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double-blind
• Purpose: TREATMENT
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 17, 2022
• First Posted: March 28, 2022
• Study Start: September 7, 2022
• Primary Completion: April 30, 2024
• Study Completion: April 30, 2024
• Last Updated: June 12, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)