Clinical trial NCT04074590: Study of Efficacy, Safety, and Tolerability of LYS006, in Patients With Mild to Moderate Ulcerative Colitis
Official title: A Randomized, Multi-center, Subject and Investigator-blinded, Placebo-controlled, Parallel-group Study to Assess the Efficacy Safety and Tolerability of LYS006 in Patients With Mild to Moderate Ulcerative Colitis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Colitis, Ulcerative
Keywords: ulcerative colitis, efficacy, safety
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2019-08-30
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This is a randomized, placebo-controlled, subject and investigator blinded, multicenter, non-confirmatory, parallel group, proof of concept study in patients with mild to moderate ulcerative colitis. This study consists of a screening period of up to 4 weeks, and a 8 week treatment period followed by a 30 day post treatment period safety follow up. The maximum study duration for each such including the 4 week screening period is 16 weeks.

At the beginning of the treatment period, subjects will be randomized to one of the two following treatment groups in 2:1 ratio:

* LYS006
* matching placebo
Primary purpose: TREATMENT

Brief summary: The purpose of the study was to assess preliminary efficacy, safety, and tolerability of LYS006 in adult patients with mild to moderate ulcerative colitis and to determine if LYS006 has an adequate clinical profile for further development in this indication.

Detailed description: This was a randomized, placebo-controlled, subject and investigator blinded, multicenter, non-confirmatory, parallel group, proof of concept study in patients with mild to moderate ulcerative colitis. This study consisted of a screening period of up to 4 weeks, and a 8 week treatment period followed by a 30 day post treatment period safety follow up. The maximum study duration for each participant including the 4 week screening period was 16 weeks.

At the beginning of the treatment period, subjects were randomized to one of the two following treatment groups in 2:1 ratio

* LYS006
* matching placebo

Eligibility criteria: Key Inclusion Criteria:

* Male and female subjects 18-75 years of age with an established diagnosis of ulcerative colitis at least 3 months prior to screening are eligible for the study.
* Patients must have active disease with a full Mayo Score between 5 and 10 (inclusive) with an endoscopy score of 2 or 3; rectal bleeding and stool frequency scores 1 to 3 and a physician's global assessment of 1 or 2.
* Patients must have responded inadequately to conventional therapy with oral 5-ASA prior to screening.

Key Exclusion Criteria:

* Has previous diagnosis with Crohn's disease, indeterminate colitis, microscopic colitis or acute diverticulitis based on medical history.
* History of toxic megacolon, abdominal abscess, symptomatic colonic stricture, or stoma; history or is at risk of colectomy.
* Treatment with biologics within 3 months or 5 half-lives (whichever is longer) prior to screening endoscopy, non-biologics advanced therapies within 4 weeks prior to screening endoscopy, systemic immunosuppressant or immunomodulator within 6 week, topical treatment with 5-ASA or steroids within 2 weeks prior to screening endoscopy

Primary outcomes:
measure: Clinical Remission Rate at the End of the Study Treatment
timeFrame: Week 8
Secondary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Adverse events were reported from first dose of study treatment until end of study treatment plus 30 days post treatment, up to a maximum duration of approximately 86 days.
Other outcomes:
Locations:
city: Sofia
country: Bulgaria
facility: Novartis Investigative Site
city: Praha 19
country: Czechia
facility: Novartis Investigative Site
state: Czech Republic
city: Zlin
country: Czechia
facility: Novartis Investigative Site
state: Czech Republic
city: Berlin
country: Germany
facility: Novartis Investigative Site
city: Nowy Targ
country: Poland
facility: Novartis Investigative Site
state: Malopolskie
city: Warszawa
country: Poland
facility: Novartis Investigative Site
state: Mazowieckie
city: Poznan
country: Poland
facility: Novartis Investigative Site
city: Novosibirsk
country: Russian Federation
facility: Novartis Investigative Site
city: Kosice
country: Slovakia
facility: Novartis Investigative Site
Study officials:
References: