Trial Outcomes & Findings for A Phase 2 Study of Interferon Beta-1a (Avonex®) in Ulcerative Colitis (NCT NCT00616434)

Last Updated: 2014-08-13

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

123 participants

Primary outcome timeframe

Baseline and Week 8

Results posted on

2014-08-13

Participant Flow

Participant milestones

Participant milestones
Measure	Interferon Beta-1a Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks	Placebo Placebo IM injection twice weekly for 12 weeks
Overall Study STARTED	62	61
Overall Study COMPLETED	56	53
Overall Study NOT COMPLETED	6	8

Reasons for withdrawal

Reasons for withdrawal
Measure	Interferon Beta-1a Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks	Placebo Placebo IM injection twice weekly for 12 weeks
Overall Study Adverse Event	5	2
Overall Study Physician Decision	0	4
Overall Study Withdrawal by Subject	1	1
Overall Study Protocol deviation	0	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Interferon Beta-1a n=62 Participants Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks	Placebo n=61 Participants Placebo IM injection twice weekly for 12 weeks	Total n=123 Participants Total of all reporting groups
Age, Continuous	41.1 years n=93 Participants	41.0 years n=4 Participants	41.0 years n=27 Participants
Sex: Female, Male Female	19 Participants n=93 Participants	26 Participants n=4 Participants	45 Participants n=27 Participants
Sex: Female, Male Male	43 Participants n=93 Participants	35 Participants n=4 Participants	78 Participants n=27 Participants

PRIMARY outcome

Timeframe: Baseline and Week 8

Population: Intent-to-treat (ITT): Defined as all randomized participants who received at least one dose of study treatment for whom a baseline measure was available.

Outcome measures

Outcome measures
Measure	Interferon Beta-1a n=62 Participants Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks	Placebo n=61 Participants Placebo IM injection twice weekly for 12 weeks
Percentage of Participants With a Clinical Response	53 percentage of participants	44 percentage of participants

SECONDARY outcome

Timeframe: Up to 16 weeks

Population: Safety Population; participants who were randomized and received at least one dose of study treatment.

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.

Outcome measures

Outcome measures
Measure	Interferon Beta-1a n=62 Participants Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks	Placebo n=61 Participants Placebo IM injection twice weekly for 12 weeks
Number of Participants With Adverse Events (AEs)	52 participants	35 participants

SECONDARY outcome

Timeframe: Baseline and Week 8

Population: Intent-to-treat (ITT): Defined as all randomized participants who received at least one dose of study treatment for whom a baseline SCCAI ≥ 3 was available.

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.

Outcome measures

Outcome measures
Measure	Interferon Beta-1a n=61 Participants Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks	Placebo n=61 Participants Placebo IM injection twice weekly for 12 weeks
Percentage of Participants With a Decrease on Simple Clinical Colitis Activity Index (SCCAI) of ≥3 Points at Week 8	64 percentage of participants	46 percentage of participants

Adverse Events

Interferon Beta-1a

Serious events: 1 serious events

Other events: 44 other events

Deaths: 0 deaths

Placebo

Serious events: 3 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Interferon Beta-1a n=62 participants at risk Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks	Placebo n=61 participants at risk Placebo IM injection twice weekly for 12 weeks
Gastrointestinal disorders Colitis Ulcerative	1.6% 1/62 • Up to 16 weeks	3.3% 2/61 • Up to 16 weeks
Injury, poisoning and procedural complications Tibia Fracture	0.00% 0/62 • Up to 16 weeks	1.6% 1/61 • Up to 16 weeks

Other adverse events

Other adverse events
Measure	Interferon Beta-1a n=62 participants at risk Interferon beta-1a 30 µg intramuscular (IM) injection twice weekly for 12 weeks	Placebo n=61 participants at risk Placebo IM injection twice weekly for 12 weeks
Infections and infestations Upper Respiratory Tract Infection	4.8% 3/62 • Up to 16 weeks	0.00% 0/61 • Up to 16 weeks
Blood and lymphatic system disorders Anaemia	1.6% 1/62 • Up to 16 weeks	9.8% 6/61 • Up to 16 weeks
Nervous system disorders Headache	17.7% 11/62 • Up to 16 weeks	4.9% 3/61 • Up to 16 weeks
Vascular disorders Hypertension	4.8% 3/62 • Up to 16 weeks	0.00% 0/61 • Up to 16 weeks
Gastrointestinal disorders Colitis Ulcerative	8.1% 5/62 • Up to 16 weeks	6.6% 4/61 • Up to 16 weeks
Musculoskeletal and connective tissue disorders Myalgia	6.5% 4/62 • Up to 16 weeks	0.00% 0/61 • Up to 16 weeks
General disorders Influenza Like Illness	40.3% 25/62 • Up to 16 weeks	9.8% 6/61 • Up to 16 weeks
General disorders Pyrexia	12.9% 8/62 • Up to 16 weeks	3.3% 2/61 • Up to 16 weeks
General disorders Malaise	4.8% 3/62 • Up to 16 weeks	1.6% 1/61 • Up to 16 weeks

Additional Information

Biogen Idec Study Medical Director

Biogen Idec

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Our agreement is subject to confidentiality but generally the PI can publish, for noncommercial purposes only, results and methods of the trial, but no other Sponsor Confidential Information. PI must give Sponsor no less than 60 days to review any manuscript for a proposed publication and must delay publication for up to an additional 90 days thereafter if Sponsor needs to file any patent application to protect any of Sponsor's intellectual property contained in the proposed publication.
Publication restrictions are in place

Restriction type: OTHER