Trial Outcomes & Findings for (CB-01-02/06) Oral Budesonide-Multi-Matrix System (MMX) 9mg Extended Release Tablets (NCT NCT01100112)

Results Overview

The primary efficacy endpoint is clinical remission at 8 weeks, defined as a Ulcerative Colitis Disease Activity Index score of \< or = 1 with a score of 0 for both rectal bleeding and stool frequency, and \> or = 1 point reduction from baseline in endoscopy score, without any sign of mucosal friability (a score of 0 for mucosal appearance). The UCDAI has 4 components. Each component is scored on scale of 0 to 3 (total maximum \[worst\] score = 12). Definitions of component scores are as follows: stool frequency: 0 = normal frequency, 1 = 1 - 2 stools per day greater than normal frequency, 2 = 3 - 4 stools per day greater than normal frequency, and 3 = \> 4 stools per day greater than normal frequency; rectal bleeding: 0 = none, 1 = streaks of blood, 2 = obvious blood, 3 = mostly blood; physician's rating of disease activity: 0 = normal, 1 = mild, 2 = moderate, 3 = severe; mucosal appearance: 0 = normal, 1 = mild friability, 2 = moderate friability, 3 = exudation, spontaneous bleeding.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

61 participants

Primary outcome timeframe

At the end of the 8 week treatment period

Results posted on

2019-11-29

Participant Flow

Recruited from February 2010 to July 2010

Patient had to have failed to achieve clinical remission in parent study CB-01-02/01. One of the 61 enrolled patients did not receive study drug. Therefore, 60 patients were evaluable for safety and efficacy analyses.

Participant milestones

Participant milestones
Measure	Budesonide Budesonide-multi-matrix system (MMX) 9 mg tablet Budesonide : One Budesonide-MMX 9 mg tablet will be taken in the morning after breakfast for 8 weeks.
Overall Study STARTED	60
Overall Study COMPLETED	52
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

(CB-01-02/06) Oral Budesonide-Multi-Matrix System (MMX) 9mg Extended Release Tablets

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Budesonide n=60 Participants Budesonide-MMX 9 mg tablet Budesonide : One Budesonide-MMX 9 mg tablet will be taken in the morning after breakfast for 8 weeks.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	60 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	40.3 years STANDARD_DEVIATION 11.41 • n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants
Sex: Female, Male Male	41 Participants n=5 Participants
Region of Enrollment India	60 participants n=5 Participants

PRIMARY outcome

Timeframe: At the end of the 8 week treatment period

Population: All patients who received at least 1 dose of study drug.

The primary efficacy endpoint is clinical remission at 8 weeks, defined as a Ulcerative Colitis Disease Activity Index score of \< or = 1 with a score of 0 for both rectal bleeding and stool frequency, and \> or = 1 point reduction from baseline in endoscopy score, without any sign of mucosal friability (a score of 0 for mucosal appearance). The UCDAI has 4 components. Each component is scored on scale of 0 to 3 (total maximum \[worst\] score = 12). Definitions of component scores are as follows: stool frequency: 0 = normal frequency, 1 = 1 - 2 stools per day greater than normal frequency, 2 = 3 - 4 stools per day greater than normal frequency, and 3 = \> 4 stools per day greater than normal frequency; rectal bleeding: 0 = none, 1 = streaks of blood, 2 = obvious blood, 3 = mostly blood; physician's rating of disease activity: 0 = normal, 1 = mild, 2 = moderate, 3 = severe; mucosal appearance: 0 = normal, 1 = mild friability, 2 = moderate friability, 3 = exudation, spontaneous bleeding.

Outcome measures

Outcome measures
Measure	Budesonide n=60 Participants Budesonide-MMX 9 mg tablet Budesonide : One Budesonide-MMX 9 mg tablet will be taken in the morning after breakfast for 8 weeks.
The Percentage of Patients Achieving Clinical Remission	25 percentage of patients

SECONDARY outcome

Timeframe: After 8 weeks treatment period

Population: All patients who received at least 1 dose of study drug.

The secondary efficacy endpoint is clinical improvement, defined as a drop in the Ulcerative Colitis Disease Activity Index score of \> or = 3 points from baseline.

Outcome measures

Outcome measures
Measure	Budesonide n=60 Participants Budesonide-MMX 9 mg tablet Budesonide : One Budesonide-MMX 9 mg tablet will be taken in the morning after breakfast for 8 weeks.
The Secondary Efficacy Endpoint is Clinical Improvement	26.7 percentage of patients

SECONDARY outcome

Timeframe: Throughout the 8 week treatment period

Population: All patients who received at least 1 dose of study drug.

Safety will be assessed by evaluating SAEs and AEs. The outcome measure data are the numbers of patients who experienced SAEs or other nonserious AEs.

Outcome measures

Outcome measures
Measure	Budesonide n=60 Participants Budesonide-MMX 9 mg tablet Budesonide : One Budesonide-MMX 9 mg tablet will be taken in the morning after breakfast for 8 weeks.
Safety Evaluations: the Numbers of Patients Who Experience Serious Adverse Events (SAEs) or Other Nonserious Adverse Events (AEs) During the Course of the Study. Serious Adverse Events	2 participants
Safety Evaluations: the Numbers of Patients Who Experience Serious Adverse Events (SAEs) or Other Nonserious Adverse Events (AEs) During the Course of the Study. Other non-serious adverse events	29 participants

SECONDARY outcome

Timeframe: 8 weeks

Population: All patients who received at least one dose of study drug.

Greater or equal to a 1 point improvement in the mucosal appearance subscore of the ulcerative colitis disease activity index (UCDAI), from baseline to week 8. The UCDAI mucosal appearance subscore is graded as follows: 0 = normal, 1 = mild friability, 2 = moderate friability, 3 = exudation, spontaneous bleeding.

Outcome measures

Outcome measures
Measure	Budesonide n=60 Participants Budesonide-MMX 9 mg tablet Budesonide : One Budesonide-MMX 9 mg tablet will be taken in the morning after breakfast for 8 weeks.
Endoscopic Improvement	40 percentage of patients

Adverse Events

Budesonide

Serious events: 2 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Budesonide n=60 participants at risk Budesonide-MMX 9 mg tablet Budesonide : One Budesonide-MMX 9 mg tablet will be taken in the morning after breakfast for 8 weeks.
Gastrointestinal disorders Colitis ulcerative	1.7% 1/60 • Number of events 1 • 56 day ± 2 day (study duration) + 30 day safety followup period.
Reproductive system and breast disorders Endometrial hyperplasia	1.7% 1/60 • Number of events 1 • 56 day ± 2 day (study duration) + 30 day safety followup period.

Other adverse events

Other adverse events
Measure	Budesonide n=60 participants at risk Budesonide-MMX 9 mg tablet Budesonide : One Budesonide-MMX 9 mg tablet will be taken in the morning after breakfast for 8 weeks.
Endocrine disorders Cushingoid	5.0% 3/60 • Number of events 3 • 56 day ± 2 day (study duration) + 30 day safety followup period.
Gastrointestinal disorders Diarrhoea	3.3% 2/60 • Number of events 2 • 56 day ± 2 day (study duration) + 30 day safety followup period.
General disorders Asthenia	3.3% 2/60 • Number of events 2 • 56 day ± 2 day (study duration) + 30 day safety followup period.
General disorders Pyrexia	3.3% 2/60 • Number of events 2 • 56 day ± 2 day (study duration) + 30 day safety followup period.
Infections and infestations Urinary tract infection	11.7% 7/60 • Number of events 7 • 56 day ± 2 day (study duration) + 30 day safety followup period.
Investigations Blood cortisol decreased	15.0% 9/60 • Number of events 9 • 56 day ± 2 day (study duration) + 30 day safety followup period.
Investigations Blood glucose decreased	3.3% 2/60 • Number of events 2 • 56 day ± 2 day (study duration) + 30 day safety followup period.
Investigations Blood potassium increased	3.3% 2/60 • Number of events 2 • 56 day ± 2 day (study duration) + 30 day safety followup period.

Additional Information

Michael Huang, MD, Senior Medical Director, Clinical Research

Santarus, Inc.

Phone: 8583145700

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place