Study of Efficacy, Safety and Tolerability of Remibrutinib in Adult Participants With an Allergy to Peanuts

A One Month, Investigator and Participant Blinded Study to Investigate the Efficacy and Safety of Remibrutinib (LOU064) at Multiple Dose Levels in Adult Participants With Peanut Allergy

A study to evaluate the safety, efficacy and tolerability of remibrutinib at three doses versus placebo in adult participants who have a confirmed allergy to peanuts. The efficacy was measured by the ability of participants to tolerate increasing doses of peanut protein during an oral food challenge after 1 month of study treatment.

Study Overview

• Status: Completed
• Conditions: Allergy, Peanut
• Intervention / Treatment: Drug: placebo; Drug: LOU064

Detailed Description

This was a randomized, participant- and investigator-blinded, placebo-controlled study to assess the safety and clinical efficacy of oral LOU064 versus placebo across five treatment arms in participants with a medically confirmed diagnosis of IgE-mediated peanut allergy for one-month treatment period (up to 5 weeks). Participants had oral food challenges at the beginning of the study and at the end of the treatment period to assess their symptoms from increasing doses of peanut allergen.

• Study Type: Interventional
• Enrollment (Actual): 76
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35209
      • AllerVie Clinical Research
  • Arkansas
    • Little Rock, Arkansas, United States, 72202
      • Arkansas Children's Hospital
  • California
    • Los Angeles, California, United States, 90025
      • California Allergy and Asthma Medical Group
    • Walnut Creek, California, United States, 94598
      • Allergy and Asthma Clin Res Inc
  • Colorado
    • Colorado Springs, Colorado, United States, 80907
      • Asthma and Allergy Associates P C
    • Denver, Colorado, United States, 80230
      • Colorado Allergy and Asthma Ctr PC
  • District of Columbia
    • Washington D.C., District of Columbia, United States, 20010
      • Childrens National Hospital
  • Idaho
    • Boise, Idaho, United States, 83706
      • Treasure Valley Medical Research
  • Illinois
    • Normal, Illinois, United States, 61761
      • Midwest Allergy Sinus Asthma SC
    • River Forest, Illinois, United States, 60305
      • Asthma and Allergy Center of Chicago S C
  • Kentucky
    • Lexington, Kentucky, United States, 40509
      • Bluegrass Allergy Research
    • Lexington, Kentucky, United States, 40509
      • Bluegrass Allergy Research .
    • Louisville, Kentucky, United States, 40217
      • Family Allergy and Asthma
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins Hospital
    • Chevy Chase, Maryland, United States, 20815
      • Institute for Asthma and Allergy PC
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • University of Michigan Clinical Trials Office
  • New Jersey
    • Berlin, New Jersey, United States, 08009
      • CenExel HRI
  • Ohio
    • Columbus, Ohio, United States, 43213
      • CR Services Acquisition US Main center
    • Columbus, Ohio, United States, 43213
      • CR Services Acquisition US
  • Oklahoma
    • Tulsa, Oklahoma, United States, 74136
      • Vital Prospects Clinical Research Institute
  • Texas
    • El Paso, Texas, United States, 79924
      • Western Sky Medical Research
  • Utah
    • Sandy City, Utah, United States, 84093
      • Allergy Associates of Utah
  • Washington
    • Seattle, Washington, United States, 98115
      • Seattle Allergy and Asthma Rsch

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 51 years (Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Medical History of allergy to peanuts
• Positive peanut IgE >= 0.35 kUA/L
• Positive Skin Prick test for peanut allergen during screening for study
• Positive Oral Food Challenge to peanut during screening for study
• Willingness to comply with study schedule and procedures and avoid other allergens during study period

Exclusion Criteria:

• History of severe or life-threatening hypersensitivity event leading to ICU admission or intubation within 60 days of screening
• Uncontrolled asthma
• Bleeding risk or coagulation disorder(s)
• Use of anticoagulants or anti-platelets (aspirin or clopidogrel may be permitted)
• History of splenectomy
• Any significant disease that would put the safety of the patient at risk. This includes, but is not limited to: history of cancer, significant cardiac disease/history, hematology disorders, history of GI bleeding, active infectious process, liver disease, renal disease, immunologic disease (stable diabetes and thyroid disease may be permitted), alcohol or drug abuse, etc.

Other protocol-defined inclusion/exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: LOU064 10 mg; LOU064 10 mg was administered orally twice per day, on Days 1 through 28.	Drug: LOU064; oral tablets; Other Names: remibrutinib
Experimental: LOU064 25 mg; LOU064 25 mg was administered orally twice per day, on Days 1 through 28.	Drug: LOU064; oral tablets; Other Names: remibrutinib
Experimental: LOU064 100 mg; LOU064 100 mg was administered orally twice per day, on Days 1 through 28.	Drug: LOU064; oral tablets; Other Names: remibrutinib
Experimental: Placebo + LOU064 25 mg; Placebo was administered orally twice per day, on Days 1 through 21 followed by LOU064 25 mg twice per day, on Days 22 through 28.	Drug: placebo; oral tablets; Drug: LOU064; oral tablets; Other Names: remibrutinib
Placebo Comparator: Placebo; Placebo was administered orally twice per day, on Days 1 through 28.	Drug: placebo; oral tablets

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Tolerated a Single Dose of >= 600 mg (1044 mg Cumulative Tolerated Dose) of Peanut Protein Without Dose-limiting Symptoms	Responder rate was defined as the percentage of participants tolerating a single dose of >= 600 mg (1044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC). The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms.	Week 4

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Who Tolerated a Single Dose of >= 1000 mg (2044 mg Cumulative Tolerated Dose) of Peanut Protein Without Dose-limiting Symptoms	Responder rate was defined as the percentage of participants tolerating a single dose of >= 1000 mg (2044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC). The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms.	Week 4
Percentage of Participants Who Tolerated a Single Dose of 3000 mg (5044 mg Cumulative Tolerated Dose) of Peanut Protein Without Dose-limiting Symptoms	Responder rate was defined as the percentage of participants tolerating a single dose of >= 3000 mg (5044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC). The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms.	Week 4
Percentage of Participants Who Tolerated a Single Dose of >= 600 mg (1044 mg Cumulative Tolerated Dose) of Peanut Protein Without Dose-limiting Symptoms - Placebo+LOU064 25 mg and Placebo	Responder rate was defined as the percentage of participants tolerating a single dose of >= 600 mg (1044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC). The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms.	Week 4
Number of Participants Presenting Maximum Severity of Symptoms Occurring at Any Challenge Dose of Peanut Protein up to and Including 3000mg During the DBPCFC	Maximum severity of symptoms occurring at any challenge dose of peanut protein up to and including 3000 mg during the DBPCFC conducted at one month, will be categorized as 4 levels: None, Mild, Moderate, Severe.	4 weeks
Change From Baseline of Peanut-specific IgE (Including Peanut Components)	IgE is a soluble biomarker that provide LOU064 response to treatment and disease severity biomarkers.	Baseline, Day 25 pre-dose and Day 31 (End of Study)
Change From Baseline of Peanut-specific IgG4 (Including Peanut Components)	IgG4 is a soluble biomarker that provide LOU064 response to treatment and disease severity biomarkers.	Baseline, Day 25 pre-dose and Day 31 (End of Study)
Change From Screening in Allergen-specific Skin Prick Test (SPT) Mean Wheal Diameters	An allergen specific skin prick test (SPT) is a commonly used diagnostic tool. In this study a titration SPT using peanut allergen provided additional information on the impact of Bruton's tyrosine kinase (BTK) suppression on skin mast cells. Skin reactions were recorded after 15 minutes of applying allergen to the pricked location. The size of the wheel and flare (the longest diameter and the midpoint orthogonal diameter) at each site were recorded.	Baseline, Day 26
Maximum Observed Blood Concentration (Cmax) of LOU064	Cmax is the maximum (peak) observed blood concentration of LOU064 after dose administration. Pharmacokinetic parameters were calculated using a non-compartmental method (WinNonLin Version 8.3.4 or higher). The LOU064 concentration was determined by a validated Liquid chromatography-mass spectrometry (LC-MS/MS) method with a lower limit of quantification (LLOQ) of 0.1 ng/mL.	Day 8 and Day 25: pre-dose, 0.5, 1, 2, 3, 4 hours.
Area Under Blood Concentration-time Curve (AUClast) of LOU064	AUClast is the area under the blood concentration-time curve from time zero to the time of last quantifiable concentration (tlast) of LOU064. Pharmacokinetic parameters were calculated using a non-compartmental method (WinNonLin Version 8.3.4 or higher). The LOU064 concentration was determined by a validated Liquid chromatography-mass spectrometry (LC-MS/MS) method with a lower limit of quantification (LLOQ) of 0.1 ng/mL.	Day 8 and Day 25: pre-dose, 0.5, 1, 2, 3, 4 hours.
Area Under Plasma Concentration-time Curve (AUCtau) of LOU064	AUCtau is the area under the plasma concentration-time curve. Estimation of AUCtau values required extrapolation of the concentration-time profile from the last measured time-point at 4 h to the end of dosing interval at 12 h post-dose. In some cases, this extrapolation was not possible. For this reason, the number of participants with measurable AUCtau values was less when compared to those with AUClast. Pharmacokinetic parameters were calculated using a non-compartmental method (WinNonLin Version 8.3.4 or higher). The LOU064 concentration was determined by a validated Liquid chromatography-mass spectrometry (LC-MS/MS) method with a lower limit of quantification (LLOQ) of 0.1 ng/mL.	Day 8 and Day 25: pre-dose, 0.5, 1, 2, 3, 4 hours.
Time to Reach Maximum Observed Blood Concentration (Tmax) of LOU064	Tmax is the time to reach maximum (peak) of LOU064 blood concentration after single-dose administration (time). Pharmacokinetic parameters were calculated using a non-compartmental method (WinNonLin Version 8.3.4 or higher). The LOU064 concentration was determined by a validated Liquid chromatography-mass spectrometry (LC-MS/MS) method with a lower limit of quantification (LLOQ) of 0.1 ng/mL.	Day 8 and Day 25: pre-dose, 0.5, 1, 2, 3, 4 hours.
Number of Participants Presenting Maximum Severity of Symptoms Occurring at Any Challenge Dose of Peanut Protein up to and Including 1000mg During the DBPCFC	Responder rate was defined as the percentage of participants tolerating a single dose of >= 3000 mg (5044 mg cumulative tolerated dose) of peanut protein without dose-limiting symptoms during the double blind placebo controlled food challenge (DBPCFC). The cumulative tolerated dose is the sum of the tolerated doses, not including the reactive dose. Dose-limiting symptoms indicate a true allergic reaction occurring during administration of a single dose of peanut protein at the DBPCFC that should preclude the administration of any further doses in the view of the investigator. Symptoms that require administration of any rescue medication were considered dose-limiting symptoms.	4 weeks

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): October 12, 2022
• Primary Completion (Actual): March 11, 2025
• Study Completion (Actual): March 11, 2025

Study Registration Dates

• First Submitted: May 31, 2022
• First Submitted That Met QC Criteria: June 20, 2022
• First Posted (Actual): June 27, 2022

Study Record Updates

• Last Update Posted (Actual): June 12, 2026
• Last Update Submitted That Met QC Criteria: May 18, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: BTKi; Peanut Allergy; Food Allergy; IgE; Oral food challenge; Remibrutinib; Hypersensitivity, Peanut; Groundnut Hypersensitivity
• Additional Relevant MeSH Terms: Nut and Peanut Hypersensitivity; Immune System Diseases; Hypersensitivity, Immediate; Hypersensitivity; Food Hypersensitivity; Peanut Hypersensitivity; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Protein Kinase Inhibitors; remibrutinib
• Other Study ID Numbers: CLOU064I12201; 2021-006950-30 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No