24 Weeks Double-blind Randomized Placebo-controlled Trial to Evaluate Efficacy, PK, Safety of LOU064 in Adolescents (12 - <18) With CSU and Inadequate Response to H1-antihistamine Followed by Optional 3 Years Open-label Extension and an Optional 3 Years Safety Long-term Treatment-free Follow-up

Launched by NOVARTIS PHARMACEUTICALS · Dec 23, 2022

Keywords

ClinConnect Summary

This clinical trial is investigating a new treatment called remibrutinib for adolescents aged 12 to less than 18 years who have chronic spontaneous urticaria (CSU), which means they experience itchy hives for a long time and haven’t found relief with standard antihistamines. The study will last for 24 weeks, where some participants will receive remibrutinib while others will receive a placebo (a non-active treatment) without knowing which one they are getting. The goal is to see how effective and safe remibrutinib is compared to the placebo. After the initial 24 weeks, there’s an option for participants to continue in an open-label extension study for up to three years to gather more information about the long-term effects of the treatment.

To be eligible for this trial, participants must be experiencing CSU for at least six months and have been using antihistamines without sufficient relief. They should be between 12 and 18 years old and meet specific health criteria to ensure their safety during the study. Participants can expect regular check-ins with doctors, and they will be monitored for any side effects throughout the trial. It’s important to note that certain health conditions or previous treatments may exclude someone from participating. This trial aims to provide a better understanding of how remibrutinib can help young people with persistent hives.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • Male and female adolescent participants aged \>= 12 to \< 18 years of age at the time of signing the informed consent
• • CSU duration for \>= 6 months prior to screening (defined as the onset of CSU determined by the investigator based on all available supporting documentation)
• * Diagnosis of CSU inadequately controlled by second-generation H1-AH at the time of randomization defined as:
• • The presence of itch and hives for ≥ 6 consecutive weeks prior to screening despite the use of second-generation H1-AH during this time period according to local treatment guidelines
• • UAS7 score (range 0 - 42) \>= 16, ISS7 score (range 0 - 21) \>= 6 and HSS7 score (range 0 - 21) \>= 6 during the 7 days prior to randomization (Day 1)
• • Documentation of hives within three months before randomization (either at screening and/or at randomization; or documented in the participants' medical history)
• Key Exclusion criteria:
• • Previous use of remibrutinib or other BTK inhibitors
• • Significant bleeding risk or coagulation disorders
• • History of gastrointestinal bleeding
• • Requirement for anti-platelet medication, except for acetylsalicylic acid up to 100 mg/d or clopidogrel up to 75 mg/d. The use of dual anti-platelet therapy (e.g., acetylsalicylic acid + clopidogrel) is prohibited
• • History or current hepatic disease
• • Evidence of clinically significant cardiovascular, neurological, psychiatric, pulmonary, renal, hepatic, endocrine, metabolic, hematological disorders, gastrointestinal disease or immunodeficiency that, in the investigator's opinion, would compromise the safety of the participant, interfere with the interpretation of the study results or otherwise preclude participation or protocol adherence of the participant
• • History of hypersensitivity to any of the study drugs or its excipients or to drugs of similar chemical classes
• • Participants having a clearly defined predominant or sole trigger of their chronic urticaria (chronic inducible urticaria) including urticaria factitia (symptomatic dermographism), cold-, heat-, solar-, pressure-, delayed pressure-, aquagenic-, cholinergic-, or contact-urticaria
• • Other diseases with symptoms of urticaria or angioedema, including but not limited to urticaria vasculitis, urticaria pigmentosa, erythema multiforme, mastocytosis, hereditary angioedema, or drug-induced urticaria
• • Any other skin disease associated with chronic itching that might influence in the investigator's opinion the study evaluations and results, e.g., atopic dermatitis, bullous pemphigoid, dermatitis herpetiformis, senile pruritus or psoriasis
• • Other protocol-defined inclusion/exclusion criteria may apply.