A Phase 2 Study of Interferon Beta-1a (Avonex®) in Ulcerative Colitis

Phase 2

Completed

Conditions: Active Ulcerative Colitis

Interventions: Drug: Placebo
Drug: BG9418 (Interferon beta-1a)

Registration Number: NCT00616434

Lead Sponsor: Biogen

Brief Summary: The primary objective of the study is to evaluate the clinical activity of interferon beta-1a in participants with moderate to severe ulcerative colitis (UC). Secondary objectives of this study are to determine (i) the safety and tolerability of interferon beta-1a in participants with moderate to severe UC, and (ii) the percentage of participants, with a decrease in the Simple Clinical Colitis Activity Index (SCCAI) score of ≥3 points at Week 8.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 123

Inclusion Criteria

Established diagnosis of ulcerative colitis (UC) for ≥6 months
- 20 cm active disease at Screening endoscopy
Must have active UC with a Mayo Score/Disease Activity Index (DAI) of 6 to 13 points and moderate to severe disease on endoscopy (Mayo endoscopic score of at least 2) despite prior or concomitant treatment
Colonoscopy within past 5 years for extent of disease and to exclude polyps
For subjects with UC for more than 10 years, colonoscopy with appropriate biopsies within 1 year prior to Screening to exclude dysplasia and neoplasia.
Must be willing and able to practice effective birth control during the study and for 1 month after the last dose of study treatment.

Key

Exclusion Criteria

Diagnosis of indeterminate colitis or Crohn's disease
Need for imminent surgery
Diagnosis of primary sclerosing cholangitis or toxic megacolon
Hemoglobin ≤9 g/dL
White blood cell count < 3500 cells/mm^3
Lymphocyte count <1000 cells/µL
Platelet count <100,000 cells/µL
Female subjects who are pregnant or who wish to become pregnant during the study, or who are lactating
Known symptomatic colonic stricture
Stool cultures positive for enteric infection
History of malignant disease
History of major abdominal surgery (e.g., gastrectomy) within past 5 years
History of small bowel or colonic obstruction or resection
History of drug or alcohol abuse (as defined by the Investigator) within 2 years prior to Screening
Use of anti-diarrheal agents during the screening period
Previous participation in this study
Previous treatment with interferon beta or other interferon products

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo IM injection twice weekly for 12 weeks
Interferon beta-1a	BG9418 (Interferon beta-1a)	Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With a Clinical Response	Baseline and Week 8	Clinical response is defined as a decrease from baseline in the total Mayo score of at least 3 points and at least 30%, accompanied by a decrease in the subscore for rectal bleeding of at least 1 point or an absolute subscore of 1 or less. Baseline was defined as the score collected during the screening period. The Mayo Score/Disease Activity Index (DAI) measures disease activity through assessment of 4 items: stool frequency, rectal bleeding, endoscopy findings, and Physician Global Assessment (PGA). Each item of the score is assessed on a 4-point scale, 0, 1, 2, or 3, with a higher score representing greater severity. In this study, the endoscopy subscore was expanded to a 5-point scale to increase sensitivity in this important dimension of the disease (0=normal/inactive disease, 4=deep ulceration). The Total Mayo Score can therefore range from 0 to13 points.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs)	Up to 16 weeks	An AE is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE, can therefore, be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. All AE's were analyzed based on the principle of treatment emergence. An AE was regarded as treatment-emergent if it was not present prior to receiving the first injection but subsequently appeared, or if it was present prior to receiving the first injection and subsequently worsened in severity.
Percentage of Participants With a Decrease on Simple Clinical Colitis Activity Index (SCCAI) of ≥3 Points at Week 8	Baseline and Week 8	The SCCAI measures disease activity as defined by both participants and examiners and includes the following 13 items: general well-being, abdominal pain, bowel frequency, stool consistency, bleeding, anorexia, nausea or vomiting, abdominal tenderness, extra-intestinal complications (eye, mouth, joint, skin), temperature, sigmoidoscopic assessment, nocturnal bowel movements, and urgency of defecation. Scores range from 0 to 19 points, and scores \<2.5 have been shown to correlate with Patient-Defined Remission, and a decrease of \>1.5 points from Baseline correlates with Patient-Defined Significant Improvement. Baseline is defined as the mean of the screening and visit 1 scores.