Study of Efficacy, Safety, and Tolerability of LYS006, in Patients With Mild to Moderate Ulcerative Colitis

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

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.

Study Overview

• Status: Terminated
• Conditions: Colitis, Ulcerative
• Intervention / Treatment: Drug: LYS006; Drug: Placebo

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

• Study Type: Interventional
• Enrollment (Actual): 23
• Phase: Phase 2

Contacts and Locations

Study Locations

• Bulgaria
  • Sofia, Bulgaria, 1612
    • Novartis Investigative Site
• Czechia
  • Czech Republic
    • Praha 19, Czech Republic, Czechia, 19000
      • Novartis Investigative Site
    • Zlin, Czech Republic, Czechia, 762 75
      • Novartis Investigative Site
• Germany
  • Berlin, Germany, 10629
    • Novartis Investigative Site
• Poland
  • Poznan, Poland, 60 529
    • Novartis Investigative Site
  • Malopolskie
    • Nowy Targ, Malopolskie, Poland, 34-400
      • Novartis Investigative Site
  • Mazowieckie
    • Warszawa, Mazowieckie, Poland, 00-728
      • Novartis Investigative Site
• Russian Federation
  • Novosibirsk, Russian Federation, 630007
    • Novartis Investigative Site
• Slovakia
  • Kosice, Slovakia, 04013
    • Novartis Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; Placebo comparator	Drug: Placebo; capsule for oral use
Experimental: LYS006; Experimental drug	Drug: LYS006; capsule for oral use

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Clinical Remission Rate at the End of the Study Treatment	The Mayo score is an instrument designed to measure activity of ulcerative colitis. The Mayo score comprises of four sub scores: stool frequency, rectal bleeding, endoscopic findings and the Physician's Global Assessment (PhGA). Each sub score is graded from 0 to 3 with higher scores indicating more severe disease. The full Mayo score is the sum of four sub scores, ranging from 0 to 12. Clinical remission is defined as a full Mayo score of 2 points or lower, with no individual subscore exceeding one point. The clinical remission rate is expressed as percentage of participants. The binary endpoint of clinical remission rate (Yes/No) at the EoT visit was modelled with binomial distribution and analyzed via the Bayesian approach with baseline total Mayo score and treatment group as explanatory variables, to compare the remission rates between the LYS006 and placebo groups.	Week 8

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)	Number of participants with treatment emergent AEs, AEs led to study treatment discontinuation, SAEs and SAEs led to study treatment discontinuation.	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.

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): February 3, 2020
• Primary Completion (Actual): November 7, 2022
• Study Completion (Actual): November 7, 2022

Study Registration Dates

• First Submitted: August 29, 2019
• First Submitted That Met QC Criteria: August 29, 2019
• First Posted (Actual): August 30, 2019

Study Record Updates

• Last Update Posted (Estimated): November 9, 2023
• Last Update Submitted That Met QC Criteria: October 18, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: ulcerative colitis, efficacy, safety
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Gastrointestinal Diseases; Gastroenteritis; Colonic Diseases; Intestinal Diseases; Inflammatory Bowel Diseases; Ulcer; Colitis; Colitis, Ulcerative
• Other Study ID Numbers: CLYS006X2202; 2019-003113-34 (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