A Study to Evaluate Safety and Efficacy of Telotristat Etiprate (LX1606) in Participants With Acute, Mild to Moderate Ulcerative Colitis

Phase 2

Completed

Conditions: Ulcerative Colitis

Interventions: Drug: Telotristat Etiprate
Drug: Placebo

Registration Number: NCT01456052

Lead Sponsor: Lexicon Pharmaceuticals

Brief Summary: This is a multicenter, placebo-controlled, parallel-group, pilot study to evaluate safety and preliminary effectiveness of two blinded dose levels of telotristat etiprate (LX1606) in participants with acute, mild to moderate ulcerative colitis on 5-aminosalicylic acid/mesalamine therapy.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 59

Inclusion Criteria

Diagnosis of ulcerative colitis of at least 6 months duration
Disease extends at least 15 cm proximally from the anal verge, documented within the past 3 years
Flare occurs on 5-aminosalicylic acid (5-ASA)/mesalamine therapy and subject is willing to remain on a stable dose for the duration of the study
Age ≥18 and <70 years of age
Able and willing to provide written informed consent

Exclusion Criteria

Prior terminal ileum or colonic surgery, except appendectomy or hemorrhoid surgery
Presence of indeterminate colitis, microscopic colitis, ischemic colitis, infectious colitis, or clinical findings suggestive of Crohn's disease
Clinical signs of fulminant colitis or toxic megacolon
History of dysplasia associated lesion or mass (DALM)
Subjects who have had surgery for ulcerative colitis, or in the opinion of the investigator, are likely to require surgery for ulcerative colitis during the study
History of primary sclerosing cholangitis
Any physical or laboratory abnormality deemed by the investigator as clinically significant
Major surgery within 60 days of Screening
Use of any investigational agent within 30 days of Screening or any therapeutic protein or antibody within 90 days of Screening

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
High Dose Telotristat Etiprate	Telotristat Etiprate	500 mg telotristat etiprate (LX1606) administered orally three times daily (TID).
Low Dose Telotristat Etiprate	Telotristat Etiprate	500 mg telotristat etiprate (LX1606) administered orally once daily (QD).
Placebo	Placebo	Matching placebo administered orally.

Primary Outcome Measures

Name	Time	Method
Number of Participants Experiencing a Treatment Emergent Adverse Event	8 weeks

Secondary Outcome Measures

Name	Time	Method
Number of Participants Achieving Clinical Response	Baseline to 8 weeks	Clinical response is defined as a decrease in the total modified Mayo score from baseline of ≥3 or a ≥30% decrease in the total modified Mayo score from baseline, along with a decrease in the rectal bleeding score ≥1 or an absolute rectal bleeding score ≤1 at Week 8. A modified Mayo score was used to evaluate disease activity using 4 components, including stool frequency, rectal bleeding, endoscopy, and physician assessment. Components = Stool frequency score 0-3 (normal- \>4 stools/day more than normal), rectal bleeding score 0-3 (none-passing blood alone), mucosal appearance at endoscopy 0-3 (normal-severe disease), physician rating of disease activity 0-3 (normal-severe). The total Modified Mayo score ranges from 0 to 12, with higher scores indicating greater disease severity.
Number of Participants Achieving Clinical Remission	Baseline to 8 weeks	Clinical remission is defined as a total modified Mayo score ≤2 with no individual score \>1 at Week 8. A modified Mayo score was used to evaluate disease activity using 4 components, including stool frequency, rectal bleeding, endoscopy, and physician assessment. Components = Stool frequency score 0-3 (normal- \>4 stools/day more than normal), rectal bleeding score 0-3 (none-passing blood alone), mucosal appearance at endoscopy 0-3 (normal-severe disease), physician rating of disease activity 0-3 (normal-severe). The total Modified Mayo score ranges from 0 to 12, with higher scores indicating greater disease severity.
Change From Baseline in Total Modified Mayo Score	Baseline to 8 weeks	A modified Mayo score was used to evaluate disease activity using 4 components, including stool frequency, rectal bleeding, endoscopy, and physician assessment. Components = Stool frequency score 0-3 (normal- \>4 stools/day more than normal), rectal bleeding score 0-3 (none-passing blood alone), mucosal appearance at endoscopy 0-3 (normal-severe disease), physician rating of disease activity 0-3 (normal-severe). The total Modified Mayo score ranges from 0 to 12, with higher scores indicating greater disease severity.