Trial Outcomes & Findings for Study of Efficacy, Safety, and Tolerability of LYS006, in Patients With Mild to Moderate Ulcerative Colitis (NCT NCT04074590)

Last Updated: 2024-06-20

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

23 participants

Primary outcome timeframe

Week 8

Results posted on

2024-06-20

Participant Flow

Participants took part in 9 investigative sites in 6 countries.

After signing informed consent, screening evaluations took place from Day -28 to Day -1. During that period all safety and other assessments were performed to evaluate eligibility. Eligible patients returned for the Baseline visit on Day 1. Eligibility was confirmed prior to randomization and required baseline assessments were completed prior to dosing on Day 1.

Participant milestones

Participant milestones
Measure	LYS006 20mg LYS006 20mg oral dose, twice daily	Placebo Placebo oral dose, twice daily
Overall Study STARTED	16	7
Overall Study COMPLETED	12	7
Overall Study NOT COMPLETED	4	0

Reasons for withdrawal

Reasons for withdrawal
Measure	LYS006 20mg LYS006 20mg oral dose, twice daily	Placebo Placebo oral dose, twice daily
Overall Study Adverse Event	1	0
Overall Study Physician Decision	1	0
Overall Study Withdrawal by Subject	2	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	LYS006 20mg n=16 Participants LYS006 20mg oral dose, twice daily	Placebo n=7 Participants Placebo oral dose, twice daily	Total n=23 Participants Total of all reporting groups
Age, Continuous	38.3 years STANDARD_DEVIATION 12.20 • n=5 Participants	45.7 years STANDARD_DEVIATION 12.37 • n=7 Participants	40.6 years STANDARD_DEVIATION 12.46 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants	5 Participants n=7 Participants	13 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	2 Participants n=7 Participants	10 Participants n=5 Participants
Race/Ethnicity, Customized White	16 Participants n=5 Participants	7 Participants n=7 Participants	23 Participants n=5 Participants

PRIMARY outcome

Timeframe: Week 8

Population: The PD analysis set included all participants who received any study drug and had no protocol deviations with relevant impact on PD data.

Outcome measures

Outcome measures
Measure	LYS006 20mg n=14 Participants LYS006 20mg oral dose, twice daily	Placebo n=7 Participants Placebo oral dose, twice daily
Clinical Remission Rate at the End of the Study Treatment	8.95 Percentage of participants Interval 0.87 to 23.31	12.24 Percentage of participants Interval 5.67 to 24.12

SECONDARY outcome

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.

Population: The safety analysis set included all participants that received any study drug.

Number of participants with treatment emergent AEs, AEs led to study treatment discontinuation, SAEs and SAEs led to study treatment discontinuation.

Outcome measures

Outcome measures
Measure	LYS006 20mg n=16 Participants LYS006 20mg oral dose, twice daily	Placebo n=7 Participants Placebo oral dose, twice daily
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) At least one SAE	0 Participants	0 Participants
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) AE leading to discontinuation	2 Participants	0 Participants
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) SAE leading to discontinuation	0 Participants	0 Participants
Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) At least one AE	7 Participants	5 Participants

Adverse Events

LYS006 20 mg

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Total

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	LYS006 20 mg n=16 participants at risk LYS006 20 mg oral dose, twice daily	Placebo n=7 participants at risk Placebo oral dose, twice daily	Total n=23 participants at risk Total
Cardiac disorders Atrioventricular block first degree	0.00% 0/16 • 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	14.3% 1/7 • 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	4.3% 1/23 • 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
Gastrointestinal disorders Colitis ulcerative	18.8% 3/16 • 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	14.3% 1/7 • 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	17.4% 4/23 • 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
Gastrointestinal disorders Vomiting	6.2% 1/16 • 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	0.00% 0/7 • 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	4.3% 1/23 • 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
General disorders Pyrexia	6.2% 1/16 • 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	0.00% 0/7 • 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	4.3% 1/23 • 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
Hepatobiliary disorders Drug-induced liver injury	0.00% 0/16 • 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	14.3% 1/7 • 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	4.3% 1/23 • 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
Infections and infestations Nasopharyngitis	0.00% 0/16 • 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	14.3% 1/7 • 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	4.3% 1/23 • 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
Infections and infestations Upper respiratory tract infection	6.2% 1/16 • 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	0.00% 0/7 • 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	4.3% 1/23 • 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
Investigations Faecal calprotectin increased	0.00% 0/16 • 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	14.3% 1/7 • 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	4.3% 1/23 • 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
Investigations Urine protein/creatinine ratio increased	0.00% 0/16 • 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	14.3% 1/7 • 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	4.3% 1/23 • 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
Musculoskeletal and connective tissue disorders Pain in extremity	6.2% 1/16 • 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	0.00% 0/7 • 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	4.3% 1/23 • 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
Nervous system disorders Headache	6.2% 1/16 • 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	0.00% 0/7 • 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	4.3% 1/23 • 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

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: + 1 862 778 8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER