BARZOLVOLIMAB for Chronic spontaneous urticaria

Inclusion criteria

• {"criterion_text":"- 1. Read, understood, and provided written informed consent, and Health Insurance Portability and Accountability Act (HIPAA) authorization if applicable, after the nature of the study has been fully explained. Participants must be able to provide informed consent themselves."}
• {"criterion_text":"- 2. Male or female, ≥ 18 years of age at the time of signing the informed consent."}
• {"criterion_text":"- 3. CSU ≥ 6 months prior to Screening. Note: the investigator should establish the presence of CSU for the noted duration based on all available supporting documentation, including written medical records and communication with the participants’ previous healthcare providers, if applicable."}
• {"criterion_text":"- CSU refractory to a stable dose and regimen containing a second-generation H1AH as defined by all of the following: • Recurrent pruritic wheals with or without angioedema for ≥ 6 weeks at any time prior to Screening (Visit 1) despite treatment with a H1AH (hives consistent with CSU should be documented and confirmed by the investigator prior to randomization) • Participants must have been on a stable dose and regimen containing a secondgeneration H1 antihistamine (H1AH) at approved or increased (up to 4x approved) dose as background therapy for the treatment of CSU for ≥ 4 weeks prior to randomization and which is expected to remain stable throughout the study. • UAS7 (range: 0 to 42) ≥ 16 and ISS7 (range: 0 to 21) ≥ 8 during the 7-day period (Day -7 to Day -1) immediately prior to randomization."}
• {"criterion_text":"- 5. Willing and able to comply with all study requirements and procedures, including the completion of a daily symptom diary during screening and throughout the study."}

Exclusion criteria

• {"criterion_text":"- 1. Diseases with possible symptoms of urticaria or angioedema such as urticarial vasculitis, erythema multiforme, cutaneous mastocytosis (urticaria pigmentosa), autoimmune syndromes with urticarial lesions (e.g., Schnitzler Syndrome) and hereditary or acquired angioedema (e.g., due to C1 inhibitor deficiency)."}
• {"criterion_text":"- 2. Chronic urticaria whose predominant manifestation is due to CIndU including symptomatic dermographism [urticaria factitia], cold-, heat-, solar-, pressure-, delayed pressure-, aquagenic-, cholinergic-, or contact urticaria Note: CIndU is not excluded per se"}
• {"criterion_text":"- 3. Any other active pruritic skin diseases that would confound CSU assessments (e.g., atopic dermatitis, psoriasis, bullous pemphigoid, dermatitis herpetiformis, prurigo nodularis, chronic pruritus of unknown origin) based on the investigator's clinical judgment."}
• {"criterion_text":"- 4. Prior receipt of barzolvolimab or other anti-KIT therapy."}

Primary endpoints

• {"endpoint_text":"- Mean change from baseline in UAS7 at Week 12","definition_or_measurement_approach":"Mean change from baseline in weekly urticaria activity score (UAS7) measured at Week 12."}

Secondary endpoints

• {"endpoint_text":"- 1. Mean change from baseline in ISS7 and HSS7 at Week 12","definition_or_measurement_approach":"Mean change from baseline in weekly itch severity score (ISS7) and weekly hive severity score (HSS7) at Week 12."}
• {"endpoint_text":"- 2. Proportion of participants with UAS7 = 0 at Week 12","definition_or_measurement_approach":"Proportion of participants achieving UAS7 = 0 at Week 12."}
• {"endpoint_text":"- 3. Proportion of participants with UAS7 ≤ 6 at Week 12","definition_or_measurement_approach":"Proportion of participants with UAS7 ≤ 6 at Week 12."}
• {"endpoint_text":"- 4. Proportion of participants with AAS7 = 0 at Week 12 in participants who have AAS7 > 0 at baseline","definition_or_measurement_approach":"Proportion of participants with weekly angioedema activity score (AAS7) = 0 at Week 12 among those with AAS7 > 0 at baseline."}
• {"endpoint_text":"- 5. Mean change from baseline in UAS7 in participants refractory to omalizumab treatment at Week 12","definition_or_measurement_approach":"Mean change from baseline in UAS7 at Week 12 in the subgroup refractory to omalizumab treatment."}
• {"endpoint_text":"- 6. Proportion of participants with UAS7 = 0 in participants refractory to omalizumab treatment at Week 12","definition_or_measurement_approach":"Proportion of omalizumab-refractory participants achieving UAS7 = 0 at Week 12."}

Methods

• Social media campaigns and social media fact sheets (materials labeled SOCIAL MEDIA, SM_FactSheet) — digital outreach targeting potential patients with chronic spontaneous urticaria; country-specific social media materials available (e.g., BG, HR, ES, PL, DE, NL, IT, HU, LT, SVK).
• Landing pages, mobile landing pages and PPC/keyword search (documents labeled PP_URLs, PP_KEYWORD_SEARCH, PP_LandingPage, PP_MOBILE LandingPage) — online recruitment channels/paid search targeting patients seeking urticaria information.
• Doctor-to-patient letters and GP/doctor letters (documents labeled Doctor_to_Patient_Letter, Doctor-to-Patient-Letter, Doctor-to-Doctor-Letter, GP letter) — site-based referral via treating physicians.
• Site flyers, posters, recruitment brochures, flipcharts and appointment reminder cards (documents labeled Recruitment_Brochure, Flipchart, Appt Reminder Card) — on-site recruitment materials distributed at trial sites.
• Prescreening tools and patient referral questionnaires (documents labeled prescreening tool questions, Patient Referral Questionnaire) — pre-screen via site or online prescreen forms.
• Email communications (documents labeled Email-Communication, Scout-Email-Comm) — direct email outreach to potential participants or registrants.
• Patient concierge and reimbursement/participant support services (documents referencing Scout Clinical, Patient concierge, Reimbursement-ICF, Patient reimbursement) — participant support and reimbursement to facilitate site visits.
• Self-capture app and at-home photography tools (documents in LT: TrialMax/App materials, Subject-At-Home-Photography-QRG, App-Subject_Facing_Screens) — digital remote capture of symptoms/photos and app-based data collection.

Hungary

Earliest CTIS Part Ii Submission Date

13-11-2024

Latest Decision Or Authorization Date

22-04-2026

Processing Time Days

525

Number Of Participants

20

Croatia

Earliest CTIS Part Ii Submission Date

19-11-2024

Latest Decision Or Authorization Date

21-04-2026

Processing Time Days

518

Number Of Participants

20

Bulgaria

Earliest CTIS Part Ii Submission Date

01-11-2024

Latest Decision Or Authorization Date

22-04-2026

Processing Time Days

537

Number Of Participants

75

Slovakia

Earliest CTIS Part Ii Submission Date

08-11-2024

Latest Decision Or Authorization Date

17-04-2026

Processing Time Days

525

Number Of Participants

24

Italy

Earliest CTIS Part Ii Submission Date

12-11-2024

Latest Decision Or Authorization Date

21-04-2026

Processing Time Days

525

Number Of Participants

30

Lithuania

Earliest CTIS Part Ii Submission Date

01-11-2024

Latest Decision Or Authorization Date

20-04-2026

Processing Time Days

535

Number Of Participants

34

Netherlands

Earliest CTIS Part Ii Submission Date

01-11-2024

Latest Decision Or Authorization Date

21-04-2026

Processing Time Days

536

Number Of Participants

20

Germany

Earliest CTIS Part Ii Submission Date

01-11-2024

Latest Decision Or Authorization Date

21-04-2026

Processing Time Days

536

Number Of Participants

60

Spain

Earliest CTIS Part Ii Submission Date

01-11-2024

Latest Decision Or Authorization Date

21-04-2026

Processing Time Days

536

Number Of Participants

45

Poland

Earliest CTIS Part Ii Submission Date

20-11-2024

Latest Decision Or Authorization Date

21-04-2026

Processing Time Days

517

Number Of Participants

200

Primary sponsor

Full Name

Celldex Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

PPD Development LP

Responsibilities

sponsorDuties codes: 1, 5

Third parties

• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 1, 5","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Inato","duties_or_roles":"Site selection (code 15); other duties (code 2)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Canfield Scientific Inc.","duties_or_roles":"Photo collection services","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"thyroid peroxidase (and code 4)","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"electronic data capture/support (code 7)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Quest Diagnostics Inc.","duties_or_roles":"Anti-IgE IgG (and code 4)","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"biopsy storage and shipment (and code 4)","organisation_type":"Pharmaceutical company"}
• {"country":"Croatia","full_name":"Optimapharm d.o.o.","duties_or_roles":"operational support (codes 1 and 12)","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Intuvigilance Limited","duties_or_roles":"pharmacovigilance/safety (code 8)","organisation_type":"Industry"}
• {"country":"Germany","full_name":"Catalent Germany Schorndorf GmbH","duties_or_roles":"manufacturing/packaging duties (code 14)","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient concierge","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"National Jewish Health","duties_or_roles":"IgE receptor Antibody (and code 4)","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"PPD Development LP (second entry)","duties_or_roles":"ADA/PK (and code 4)","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Signant Health Global Solutions Limited","duties_or_roles":"eCOA","organisation_type":"Pharmaceutical company"}