Trial Outcomes & Findings for Efficacy, Pharmacodynamics, Safety and Tolerability of Oral BL-7040 in Patients With Moderately Active Ulcerative Colitis (NCT NCT01506362)

Results Overview

Mayo score assesses stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment. It is assessed according to the following parameters: Stool frequency (subscore 0-3) 0: Normal number of stools for patient 1. 1 to 2 stools per day more than normal 2. 3 to 4 stools more than normal 3. \> or = to 5 stools more than normal Rectal bleeding (subscore 0-3) 0: No blood seen 1. Streaks of blood with stool less than half the time 2. Obvious blood with stool most of the time 3. Blood alone passes Endoscopic findings (subscore 0-3) 0: Normal or inactive disease 1 Mild Disease (erythema, decreased vascular pattern, mild friability) 2: Moderate Disease (marked erythema, lack of vascular pattern, friability erosions) 3: Severe Disease (spontaneous bleeding, ulceration) Physician's Global Assessment (subscore 0-3) 0: Normal 1. Mild disease 2. Moderate disease 3. Severe disease

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

22 participants

Primary outcome timeframe

From Baseline to day 34 (end of treatment period)

Results posted on

2014-07-15

Participant Flow

Participant milestones

Participant milestones
Measure	A Synthetic Oligonucleotide for Treatment of IBD. BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. Study duration can last up to 8 weeks, including up to 9 days in the screening period, up to 5 weeks of treatment with BL-7040, and up to 2 weeks for follow up. BL-7040 12 mg QD for 19-21 days followed by BL-7040 40 mg QD for 14 days.
Overall Study STARTED	22
Overall Study COMPLETED	16
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Efficacy, Pharmacodynamics, Safety and Tolerability of Oral BL-7040 in Patients With Moderately Active Ulcerative Colitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	A Synthetic Oligonucleotide for Treatment of IBD. n=22 Participants BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. BL-7040: BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. Study duration can last up to 8 weeks, including up to 9 days in the screening period, up to 5 weeks of treatment with BL-7040, and up to 2 weeks for follow up. BL-7040 12 mg QD for 19-21 days followed by BL-7040 40 mg QD for 14 days.
Age, Continuous	33.9 years STANDARD_DEVIATION 13.9 • n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	21 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants
Region of Enrollment Israel	22 participants n=5 Participants

PRIMARY outcome

Timeframe: From Baseline to day 34 (end of treatment period)

Mayo score assesses stool frequency, rectal bleeding, endoscopic findings, and physician's global assessment. It is assessed according to the following parameters: Stool frequency (subscore 0-3) 0: Normal number of stools for patient 1. 1 to 2 stools per day more than normal 2. 3 to 4 stools more than normal 3. \> or = to 5 stools more than normal Rectal bleeding (subscore 0-3) 0: No blood seen 1. Streaks of blood with stool less than half the time 2. Obvious blood with stool most of the time 3. Blood alone passes Endoscopic findings (subscore 0-3) 0: Normal or inactive disease 1 Mild Disease (erythema, decreased vascular pattern, mild friability) 2: Moderate Disease (marked erythema, lack of vascular pattern, friability erosions) 3: Severe Disease (spontaneous bleeding, ulceration) Physician's Global Assessment (subscore 0-3) 0: Normal 1. Mild disease 2. Moderate disease 3. Severe disease

Outcome measures

Outcome measures
Measure	A Synthetic Oligonucleotide for Treatment of IBD. n=22 Participants BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. BL-7040: BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. Study duration can last up to 8 weeks, including up to 9 days in the screening period, up to 5 weeks of treatment with BL-7040, and up to 2 weeks for follow up. BL-7040 12 mg QD for 19-21 days followed by BL-7040 40 mg QD for 14 days.
Proportion of Patients Who After Study Completion Achieve Clinical Response Defined by at Least a 3point Decrease & 30% Reduction From Baseline in Mayo Score Plus ≥ 1 Point Decrease in Rectal Bleeding Sub-score or Absolute Rectal Bleeding Subscore of ≤ 1	8 participants

SECONDARY outcome

Timeframe: 5 weeks following first administration

Outcome measures

Outcome data not reported

Adverse Events

A Synthetic Oligonucleotide for Treatment of IBD.

Serious events: 1 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	A Synthetic Oligonucleotide for Treatment of IBD. n=22 participants at risk BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. BL-7040: BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. Study duration can last up to 8 weeks, including up to 9 days in the screening period, up to 5 weeks of treatment with BL-7040, and up to 2 weeks for follow up. BL-7040 12 mg QD for 19-21 days followed by BL-7040 40 mg QD for 14 days.
Blood and lymphatic system disorders hemoglobin decreased	4.5% 1/22 • Number of events 1

Other adverse events

Other adverse events
Measure	A Synthetic Oligonucleotide for Treatment of IBD. n=22 participants at risk BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. BL-7040: BL-7040 is an orally available new chemical entity for the treatment of IBD. BL-7040 is a synthetic oligonucleotide with dual activity on both the nervous and immune systems. Study duration can last up to 8 weeks, including up to 9 days in the screening period, up to 5 weeks of treatment with BL-7040, and up to 2 weeks for follow up. BL-7040 12 mg QD for 19-21 days followed by BL-7040 40 mg QD for 14 days.
Gastrointestinal disorders Exacerbation of ulcerative colitis	18.2% 4/22 • Number of events 4
General disorders Influenza-like illness	13.6% 3/22 • Number of events 6
Gastrointestinal disorders Dry mouth	9.1% 2/22 • Number of events 2
General disorders Fatigue	9.1% 2/22 • Number of events 2
Nervous system disorders Headache	9.1% 2/22 • Number of events 2

Additional Information

Dr. Arnon Aharon

BioLineRx LTD

Phone: 972-2-548-9100

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place