LOU064 (open-label) for Chronic Spontaneous Urticaria

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Novartis Investigative Site, San Juan, Puerto Rico
Chronic Spontaneous Urticaria+2 More
LOU064 (open-label) - Drug
Eligibility
18+
All Sexes
Eligible conditions
Select

Study Summary

A Phase 3 Study of Efficacy and Safety of Remibrutinib in the Treatment of CSU in Adults Inadequately Controlled by H1 Antihistamines

See full description

Eligible Conditions

  • Chronic Spontaneous Urticaria

Treatment Effectiveness

Effectiveness Estimate

2 of 3
This is better than 85% of similar trials

Study Objectives

This trial is evaluating whether LOU064 (open-label) will improve 2 primary outcomes and 11 secondary outcomes in patients with Chronic Spontaneous Urticaria. Measurement will happen over the course of 2 weeks.

12 weeks
Absolute change in ISS7 and absolute change in HSS7 (Scenario 2 with ISS7 and HSS7 as co-primary efficacy endpoints)
Achievement of DLQI = 0 - 1
Change from baseline in UAS7 (Scenario 1 with UAS7 as primary efficacy endpoint)
Change from baseline in UAS7 (only in scenario 2)
Complete absence of hives and itch (UAS7 = 0)
Disease activity control (UAS7 ≤ 6)
Number of weeks without angioedema (AAS = 0)
Reduction of weekly HSS score (only in scenario 1)
Reduction of weekly ISS score (only in scenario 1)
Sustained disease activity control (UAS7 ≤6)
2 weeks
Early onset of disease control (UAS7 ≤ 6 at week 2)
56 weeks
Number of participants with Adverse Events
Safety and tolerability of remibrutinib

Trial Safety

Safety Estimate

3 of 3
This is better than 85% of similar trials

Trial Design

2 Treatment Groups

Arm 1: LOU064 (blinded)
1 of 2
Arm 2: LOU064 placebo (blinded)
1 of 2
Experimental Treatment
Non-Treatment Group

This trial requires 450 total participants across 2 different treatment groups

This trial involves 2 different treatments. LOU064 (open-label) is the primary treatment being studied. Participants will all receive the same treatment. Some patients will receive a placebo treatment. The treatments being tested are in Phase 3 and have had some early promising results.

Arm 1: LOU064 (blinded)LOU064 (blinded) taken orally for 24 weeks, followed by LOU064 (open-label) taken orally open label for 28 weeks. Randomized in a 2:1 ratio (arm 1:arm 2).
Arm 2: LOU064 placebo (blinded)LOU064 placebo (blinded) taken orally for 24 weeks, followed by LOU064 (open-label) taken orally for 28 weeks. Randomized in a 2:1 ratio (arm 1:arm 2).

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 56 weeks
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 56 weeks for reporting.

Closest Location

Novartis Investigative Site - Waco, TX

Eligibility Criteria

This trial is for patients born any sex aged 18 and older. There are 8 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Signed informed consent must be obtained prior to participation in the study.
Male and female adult participants ≥18 years of age.
CSU duration for ≥ 6 months prior to screening (defined as the onset of CSU determined by the investigator based on all available supporting documentation).
The presence of itch and hives for ≥6 consecutive weeks prior to screening despite the use of second generation H1-antihistamines during this time period
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).
Willing and able to complete an Urticaria Patient Daily Diary (UPDD) for the duration of the study and adhere to the study protocol.
Participants must not have had more than one missing UPDD entry (either morning or evening) in the 7 days prior to randomization (Day 1).

Patient Q&A Section

How many people get chronic spontaneous urticaria a year in the United States?

"around 3 million people in the United States will develop CU at some point during their lives. Some of these patients develop CU later in their life. Those with late onset CU are more likely to develop CU due to a lower urticarial disease activity at presentation." - Anonymous Online Contributor

Unverified Answer

Can chronic spontaneous urticaria be cured?

"The prognosis of chronic spontaneous urticaria is variable, depending on the number of symptoms, disease duration and response to treatment. In cases with few symptoms and in cases with a prolonged spontaneous onset, the clinical course is somewhat better. The long term prognosis is still unpredictable." - Anonymous Online Contributor

Unverified Answer

What is chronic spontaneous urticaria?

"CSU patients are predominantly women, with a mean age of about 45 years, and more than 75% of them have symptoms of anxiety/depression. We also observed low prevalence of CSU related to the use of drugs, but with a slight increase during the symptomatic period." - Anonymous Online Contributor

Unverified Answer

What are common treatments for chronic spontaneous urticaria?

"Spontaneous urticaria is a very common condition that is characterized by a variable onset and severity of symptoms. Most patients have at least a subset of the symptoms. Despite the recent introduction of several effective and generally well tolerated treatments, many are left with less than control of the symptoms. These patients' distress often exceeds that of those with other skin diseases like atopic dermatitis. Current and emerging treatments are discussed, as well as the current management guidelines." - Anonymous Online Contributor

Unverified Answer

What causes chronic spontaneous urticaria?

"Chronic spontaneous urticaria usually has an autoimmune origin, due to some kind of immune system malfunction. It is a good strategy to manage the disease by giving drugs that suppress the immune response and diminish symptoms." - Anonymous Online Contributor

Unverified Answer

What are the signs of chronic spontaneous urticaria?

"The skin-prick test, serum histamine and tryptase levels are useful tools in the diagnosis of chronic spontaneous urticaria. The skin-prick test positivity might be linked to the production of histamine. There is also a trend for increased tryptase levels." - Anonymous Online Contributor

Unverified Answer

Is lou064 (open-label) typically used in combination with any other treatments?

"The combination of lou064 plus low-dose topical anticholinergics appears to be safe and effective in patients with chronic urticaria, and it achieves improvements in patients as a stand-alone treatment or in combination with other medications." - Anonymous Online Contributor

Unverified Answer

What are the latest developments in lou064 (open-label) for therapeutic use?

"The safety and tolerability in both Phase 1 clinical trials has shown lou064 has a similar profile to other urushiols and it was generally well tolerated. Due to the potential toxicity from long-term treatment, clinicians and their partners now have a greater awareness of these new therapies (e.g. lou064) and to monitor patients for long term side effects. While the use of this medication requires the cooperation of their physician, a review of lou064's safety and efficacy profile can help determine if this is the therapeutic treatment for them. The clinical trial is a collaboration between the Faculty of Medicine at Monash University, Monash University and Monash University South Australia." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving lou064 (open-label)?

"Lou064 is well tolerated. The safety and efficacy profile of lou064 in LS are similar to those observed in a pivotal trial of 5-mino-adamantane administered 4.5 minutes after loratadine inhalation, which shows that lou064 could safely be administered in LS. ClinicalTrials.gov ID: NCT01558905." - Anonymous Online Contributor

Unverified Answer

Does chronic spontaneous urticaria run in families?

"CSU is heritable, and the disease phenotype in siblings is correlated. CSU is caused by common, familial determinants as well as rare contributing variants, highlighting the importance of considering CSU as a genetic disorder that can, and should, be treated." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating chronic spontaneous urticaria?

"The recent clinical trials with dapsone or azathioprine seem to be encouraging when compared to other treatments. Results from a recent paper suggest a need to study these drugs more closely in randomized-controlled trials, preferably including patients with mild or moderate-to-severe disease." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of chronic spontaneous urticaria?

"In most patients, the predominant cause is idiopathic. However, it remains unclear why many individuals develop CSU when seemingly matched by history, autoantibodies, and autoantigen." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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