Ritlecitinib (PF-06651600) in Participants With Chronic Spontaneous Urticaria

NCT06795373 | Withdrawn Phase 2 FDA Drug

• 1 other identifier: STUDY-24-01277
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research study is to see if a drug called ritlecitinib is safe and effective for treating chronic spontaneous urticaria (CSU). CSU is hives and itching lasting over six weeks. Ritlecitinib is approved by the Food and Drug Administration (FDA) to treat another condition, but it is not approved for treating CSU. Participation is expected to last 20 weeks and include 7 clinic visits. This study will involve physical examinations, blood tests, looking at and taking pictures of participant's skin and hives, optional skin biopsies, and hearing tests. Eligible participants for this study will take ritlecitinib for 12 weeks and complete a daily diary about their skin and hives. The main risks of being in this study are side effects from ritlecitinib. Less than 1 in 10 people taking ritlecitinib experience diarrhea, acne, hives, rash, inflammation of hair follicles, dizziness, and increased blood levels of creatine phosphokinase (a muscle protein). Participants could also experience a rare but serious side effect, such as shingles, unusual infection, cancer, or blood clot. Benefits of participating in this study include a potential improvement in participant's condition and quality of life. Participating in this study may also help researchers develop new ways of helping future patients.

Conditions

Chronic Spontaneous Urticaria; CSU

Keywords

Chronic Spontaneous Urticaria; CSU; Ritlecitinib

Outcome Measures

Primary Outcomes (1)

• Change in Urticaria Activity Score (UAS7)

  The UAS7 is calculated by adding up the daily UAS scores over a 7-day period. The score ranges from 0 to 42, with higher scores indicating more active disease. Score Ranges: 0: Urticaria-free; 1-6: Well-controlled urticaria; 7-15: Mild urticaria; 16-27: Moderate urticaria; 28-42: Severe urticaria.

  Baseline to Week 12

Secondary Outcomes (9)

• Change in Urticaria Activity Score (UAS7)

  Baseline to Week 2, 4, 8, and 16

• Change in Angioedema Activity Score (AAS7)

  Baseline to Week 2, 4, 8, and 16

• Percentage change of Health-Related Quality of Life (CU-Q2oL)

  Baseline to Week 12

• Change in Urticaria Control Test (UCT)

  Baseline to Week 12

• Percentage change in Urticaria Activity Score (UAS7)

  Baseline to Week 2, 4, 8, 12, and 16

Study Arms (1)

persistent, progressive, or recurrent chronic spontaneous urticaria (CSU).

EXPERIMENTAL

Participants with persistent, progressive, or recurrent chronic spontaneous urticaria (CSU).

Drug: Ritlecitinib

Interventions

Ritlecitinib DRUG

Once daily dose of 100mg of ritlecitinib taken orally for 12 weeks

Also known as: PF-06651600

persistent, progressive, or recurrent chronic spontaneous urticaria (CSU).

Eligibility Criteria

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

You may qualify if:

• Participants are eligible to be included in the study only if all the following criteria apply:
• Participant has been informed about study procedures and medications and has provided informed consent prior to initiation of any study-specific activities/procedures
• Participant is able to communicate with the investigator, and understands and complies with the requirements of the study
• Age ≥ 18 to ≤ 65 years of age at screening
• Participant has a negative Tuberculin purified protein derivative (PPD) or QuantiFERON TBGold test (QFT) at screening or within the last 12 months.
• Chronic spontaneous urticaria diagnosis ≥ 3 months at the time of screening visit 1
• Diagnosis of CSU inadequately controlled by second-generation H1-antihistamines (sgAH) at enrollment, as defined by the following:
• The presence of itch and hives for ≥ 6 consecutive weeks at any time prior to screening visit 2 despite current use of an approved dose of H1-antihistamine
• Urticaria Activity Score over 7 days (UAS7) (range 0-42) ≥ 16 and Hive Severity Score over 7 days (HSS7) (range 0-21) ≥ 8 during the 7 days prior to enrollment
• Participant must have been on or failed a sgAH at approved or increased doses (up to 2 or 4x the approved dose) for treatment of CSU prior to the Baseline visit and must have documented current use on the day of screening visit ○ If participants are currently on a sgAH, they must continue the same dose throughout the trial

You may not qualify if:

• Disease Related
• Urticaria is solely due to inducible urticaria
• Active dermatologic diseases (or conditions) other than chronic urticaria, with urticaria wheals or angioedema symptoms such as urticarial vasculitis, erythema multiforme, cutaneous mastocytosis (urticaria pigmentosa) and hereditary or acquired angioedema (eg, due to C1 inhibitor deficiency)
• Any other active skin disease associated with chronic itching that might influence, in the investigator's opinion, the study evaluations and results (eg, atopic dermatitis, dermatitis herpetiformis, senile pruritus, etc.)
• Other Medical Conditions
• History of, or a concurrent, clinically significant illness, medical condition or laboratory abnormality that, in the investigator's opinion, could affect the conduct of the study
• Active immunosuppression by previous (5 x half-lives or 12 weeks, whichever is longer) or current systemic cytotoxic therapies
• Uncontrolled current illness, including, but not limited to the following: Ongoing or active infections requiring intravenous antimicrobials; symptomatic congestive heart failure defined as NYHA class III or IV; unstable angina pectoris within 6 months of study enrollment; history of myocardial infarction, stroke or intracranial hemorrhage within 6 months prior to enrollment; moderate to severe hepatic impairment (Child-Pugh class B or C); psychiatric illness or social situations that would limit compliance with study requirements
• Previous or current cancer, except curatively treated basal or squamous cell carcinoma of the skin, and curatively treated malignant melanoma stage 0-1A with a low risk of recurrence/metastasis as per assessment of the investigator, cervical carcinoma in situ, treated basal cell carcinoma, superficial bladder tumors (Ta, Tis and T1)
• Known HIV infection
• Infected with Hepatitis B or Hepatitis C viruses
• Participants with history of either untreated or inadequately treated latent or active TB infections/currently being treated for active TB
• Recent (within 21 days before visit 1) major surgery
• Participants who have history of a single episode of disseminated HZ or disseminated HS or recurrent (\> 1 episode of) localized dermatomal HZ should be excluded
• Any gastrointestinal or metabolic condition that could interfere with the absorption of the oral medication

Sponsors & Collaborators

• Ahuva D Cices: lead

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

MeSH Terms

Conditions

Chronic Urticaria

Interventions

PF-06651600

Condition Hierarchy (Ancestors)

Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Ahuva Cices, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Model Details: This is a prospective phase IIA study to investigate efficacy, safety, and tolerability of ritlecitinib treatment in participants with persistent, progressive, or recurrent chronic spontaneous urticaria (CSU). It is designed as an open label, single-arm, single-center pilot study. A total of up to 20 participants with a physician-based diagnosis of CSU and the other characteristics mentioned in the inclusion criteria will be included.

Study Record Dates

• First Submitted: January 22, 2025
• First Posted: January 28, 2025
• Study Start: December 31, 2025
• Primary Completion: March 31, 2026
• Study Completion: March 31, 2026
• Last Updated: December 9, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)