E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute mild to moderate ulcerative colitis |
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E.1.1.1 | Medical condition in easily understood language |
Sensitive, calm to medium inflammatory bowel disease (IBD) that causes frequent inflammation of the digestive tract
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10066678 |
E.1.2 | Term | Acute ulcerative colitis |
E.1.2 | System Organ Class | 100000004856 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of this study is to evaluate the safety and tolerability of orally administered LX1606 after 8 weeks in a cohort of subjects with acute, mild to moderate ulcerative colitis. |
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E.2.2 | Secondary objectives of the trial |
• Examine the relationship between reductions in 5-HIAA levels and clinical improvement in ulcerative colitis
• Evaluate differences between each LX1606 dose group and placebo for the following measures at Week 8:
- Proportion of subjects achieving clinical response
- Proportion of subjects achieving clinical remission
- Change from baseline in the total modified Mayo score
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subjects must meet all of the following criteria to be considered eligible to participate in the study:
1. Diagnosis of ulcerative colitis of at least 6 months duration
2. Disease extends at least 15cm proximally from the anal verge (defined as the transitional zone between the perianal skin and the surface of the anal canal) documented within the past 3 years.
Flare occurs on a background of 5-ASA/mesalamine therapy; subject is willing to remain on stable dose for the duration of the blinded trial period. 4. Age >18 years and <70 years at the time of Screening.
5. Ability and willingness to provide written informed consent prior to participation in any study-related activities and to participate in and comply with the study requirements. |
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E.4 | Principal exclusion criteria |
Subjects presenting with any of the following will not be included in the trial
1. Subject has had any prior terminal ileum or colonic surgery, except appendectomy or hemorrhoid surgery.
2. Presence of indeterminate colitis, microscopic colitis, ischemic colitis, infectious colitis, or clinical findings suggestive of Crohn’s disease.
3. Subjects displaying clinical signs of fulminant colitis or toxic megacolon.
4. Subjects with a history of Dysplasia associated lesion or mass (DALM).
5. Subjects who have had surgery for UC or in the opinion of the Investigator, are likely to require surgery for UC during the study period.
6. Subjects with a history of primary sclerosing cholangitis.
7. Any physical finding or laboratory abnormality the investigator deems clinically significant that would pose a safety issue for the subject or interfere with interpretation of the data.
8. Major surgery within 60 days prior to Screening.
9. Administration of any investigational agent within 30 days of Screening or any therapeutic protein or antibody within 90 days of Screening. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Assessment of safety and tolerability of LX1606 after 8 weeks in a cohort of subjects with acute, mild to moderate ulcerative colitis. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Throughout the duration of the study |
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E.5.2 | Secondary end point(s) |
- Examination of the relationship between reductions in 5-HIAA levels and clinical improvement in ulcerative colitis
- Evaluatation of differences between each LX1606 dose group and placebo for the following measures at Week 8:
Proportion of subjects achieving clinical response
Proportion of subjects achieving clinical remission
Change from baseline in the total modified 1 Mayo score
1 Endoscopy evaluation is based on modified criteria:
0 = Normal or inactive disease
1 = Mild disease (erythema, decreased vascular pattern, no friability
2 = Moderate disease (marked erythema, absent vascular pattern, friability, erosions)
3 = Severe disease (spontaneous bleeding, ulceration) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 18 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
Lithuania |
Poland |
Slovakia |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |