Trial Outcomes & Findings for An Efficacy and Safety Study of Mongersen (GED-0301) in Subjects With Active Ulcerative Colitis (NCT NCT02601300)

Results Overview

Clinical remission was defined as a modified Mayo score of ≤ 2, with no individual subscore \> 1, at Week 8. The MMS was based on a modification of the total Mayo score (TMS) which included the stool frequency, rectal bleeding, and endoscopic subscores of the TMS and excluded the Physician's Global Assessment (PGA) subscore, since this was a global measure that is subjective in nature. The MMS ranges from 0 to 9 points with higher scores indicating greater disease severity. The endoscopy subscores was centrally reviewed. Two-sided confidence intervals for the within-group percentage were based on the Wilson score method.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

41 participants

Primary outcome timeframe

Baseline to Week 8

Results posted on

2018-10-17

Participant Flow

Participants were enrolled at 21 study centers within the United States, Bulgaria, Poland, Slovakia and Canada.

Participants were 18 years of age and older with active ulcerative colitis for 3 months prior to screening, had a modified Mayo score (MMS) ≥ 4 to ≤ 9 absolute rectal bleeding (RBS) subscore ≥ 1 at screening, a mayo endoscopic subscore ≥ 2 at screening and must have had a therapeutic failure or been intolerant to other therapies.

Participant milestones

Participant milestones
Measure	Mongersen (Weeks 0-52) Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Induction Phase STARTED	41
Induction Phase COMPLETED	38
Induction Phase NOT COMPLETED	3
Extension Phase STARTED	35
Extension Phase Treated With IP	34
Extension Phase COMPLETED	18
Extension Phase NOT COMPLETED	17

Reasons for withdrawal

Reasons for withdrawal
Measure	Mongersen (Weeks 0-52) Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Induction Phase Lack of Efficacy	2
Induction Phase Protocol Violation	1
Extension Phase Withdrawal by Subject	3
Extension Phase Adverse Event	4
Extension Phase Lack of Efficacy	10

Baseline Characteristics

An Efficacy and Safety Study of Mongersen (GED-0301) in Subjects With Active Ulcerative Colitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Age, Continuous	42.0 years STANDARD_DEVIATION 11.91 • n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	28 Participants n=5 Participants
Race/Ethnicity, Customized Asian	1 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	2 Participants n=5 Participants
Race/Ethnicity, Customized White	37 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants
Duration of Ulcerative Colitis	9.91 years STANDARD_DEVIATION 9.107 • n=5 Participants
Baseline Modified Mayo Score (MMS)	6.5 units on a scale STANDARD_DEVIATION 1.50 • n=5 Participants
Baseline Mayo Endoscopic Subscore	2.6 units on a scale STANDARD_DEVIATION 0.50 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline to Week 8

Population: The intent to treat population included all participants who received at least one dose of IP. The primary approach to handing missing data was non-responder imputation (NRI) method, where participants who had insufficient data for response determination at Week 8 were considered non-responders for clinical remission.

Clinical remission was defined as a modified Mayo score of ≤ 2, with no individual subscore \> 1, at Week 8. The MMS was based on a modification of the total Mayo score (TMS) which included the stool frequency, rectal bleeding, and endoscopic subscores of the TMS and excluded the Physician's Global Assessment (PGA) subscore, since this was a global measure that is subjective in nature. The MMS ranges from 0 to 9 points with higher scores indicating greater disease severity. The endoscopy subscores was centrally reviewed. Two-sided confidence intervals for the within-group percentage were based on the Wilson score method.

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Percentage of Participants Who Achieved Clinical Remission in the Modified Mayo Score (MMS) at Week 8	17.1 percentage of participants Interval 8.5 to 31.3

SECONDARY outcome

Timeframe: Baseline to Week 8

Population: The intent to treat population included all participants who received at least one dose of IP. The primary approach to handling missing data was NRI method, where participants who had insufficient data for response determination at Week 8 were considered non-responders for the MMS response.

A MMS was used to evaluate disease activity using 3 components: stool frequency, rectal bleeding and endoscopy; the MMS ranges from 0-9 with higher scores indicating greater disease severity. Stool frequency subscore was defined as 0-3: 0 = Normal number of stools for patient 1. = 1-2 stools per day more than normal 2. = 3-4 stools more than normal 3. = 5 or more stools more than normal Rectal bleeding (subscore 0-3) was defined as: 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 subscore: Findings were defined as: 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)

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Percentage of Participants Who Achieved a Modified Mayo Score of ≤ 2, With Rectal Bleeding Subscore (RBS) of 0 and Stool Frequency Subscore (SFS) and Mayo Endoscopic Subscore ≤ 1 at Week 8	14.6 percentage of participants Interval 6.9 to 28.4

SECONDARY outcome

Timeframe: Baseline to Week 8

Population: The intent to treat population included all participants who received at least one dose of IP. The primary approach to handling missing data was NRI method, where participants who had insufficient data for response determination at Week 8 were considered non-responders for Mayo Endoscopic Subscore.

A Mayo endoscopic subscore of ≤ 1 was assessed and evaluated in participants who achieved a Mayo endoscopic subscore at Week 8. The endoscopy subscore findings are defined as: 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) The endoscopy scores were centrally reviewed. Two-sided 95% CIs for the within-group percentage were based on the Wilson score method.

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Percentage of Participants Who Achieved a Mayo Endoscopic Subscore of ≤ 1 at Week 8	19.5 percentage of participants Interval 10.2 to 34.0

SECONDARY outcome

Timeframe: Baseline to Week 8

Population: The intent to treat population included all participants who received at least one dose of IP. Only participants with sufficient data for response determination in each segment were included in the analysis.

A Mayo endoscopic subscore by individual segment (rectum, sigmoid, descending colon, transverse colon, ascending colon/cecum) of ≤ 1 was evaluated at week 8. The endoscopy subscore findings are defined as: 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) The endoscopy scores were centrally reviewed. Two-sided 95% CIs for the within-group percentage were based on the Wilson score method.

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Percentage of Participants Who Achieved a Mayo Endoscopic Subscore of ≤ 1 by Individual Segment at Week 8 Rectum	27.0 percentage of participants Interval 15.4 to 43.0
Percentage of Participants Who Achieved a Mayo Endoscopic Subscore of ≤ 1 by Individual Segment at Week 8 Sigmoid	27.0 percentage of participants Interval 15.4 to 43.0
Percentage of Participants Who Achieved a Mayo Endoscopic Subscore of ≤ 1 by Individual Segment at Week 8 Transverse Colon	60.7 percentage of participants Interval 42.4 to 76.4
Percentage of Participants Who Achieved a Mayo Endoscopic Subscore of ≤ 1 by Individual Segment at Week 8 Ascending Colon/Cecum	80.0 percentage of participants Interval 60.9 to 91.1
Percentage of Participants Who Achieved a Mayo Endoscopic Subscore of ≤ 1 by Individual Segment at Week 8 Descending Colon	54.3 percentage of participants Interval 38.2 to 69.5

SECONDARY outcome

Timeframe: Baseline to Week 8

Population: The intent to treat population included all participants who received at least one dose of IP. The primary approach to handling missing data was NRI method, where participants who had insufficient data for response determination at Week 8 were considered non-responders for MMS.

Clinical response in the MMS was defined as a decrease from baseline in the MMS of at least 2 points and at least 25%, along with a reduction in the RBS of at least 1 point or an absolute RBS ≤ 1. The MMS was based on the stool frequency, rectal bleeding, and endoscopic subscores of the TMS and excluded the PGA subscore. The MMS ranges from 0 to 9 points with higher scores indicating greater disease severity. The RBS was defined as: 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 The daily bleeding score represents the most severe bleeding score represents the most severe bleeding of the day.

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Percentage of Participants Who Achieved a Clinical Response in the Modified Mayo Score at Week 8	36.6 percentage of participants Interval 23.6 to 51.9

SECONDARY outcome

Timeframe: Baseline and Week 8

Population: The intent to treat population included all participants who received at least one dose of IP. The primary approach to handling missing data was NRI method, where participants who had insufficient data for response determination at Week 8 were considered non-responders for Mayo endoscopic response.

Endoscopic response was defined as a decrease from baseline of at least 1 point in the Mayo endoscopic subscore. The Mayo endoscopy subscore findings are defined as: 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) The endoscopy subscores were centrally reviewed. Two-sided 95% CIs for the within-group percentage were based on the Wilson score method.

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Percentage of Participants Who Achieved a Mayo Endoscopic Response at Week 8	31.7 percentage of participants Interval 19.6 to 47.0

SECONDARY outcome

Timeframe: Baseline to Week 8

Population: The intent to treat population included all participants who received at least one dose of IP. The primary approach to handling missing data was NRI method, where participants who had insufficient data for response determination at Week 8 were considered non-responders for Total Mayo score.

Clinical remission in total Mayo score was defined as a total Mayo score of ≤ 2, with no individual subscore \>1. The TMS is an instrument designed to measure disease activity of ulcerative colitis. The TMS ranges from 0 to 12 points. It consists of 4 subscores, each graded from 0 to 3 with higher scores indicating more severe disease. * Stool frequency subscore (SFS) * Rectal bleeding subscore (RBS) * Endoscopic subscore * Physician's Global Assessment (PGA)

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Percentage of Participants Who Achieved a Clinical Remission in the Total Mayo Score (TMS) at Week 8	9.8 percentage of participants Interval 3.9 to 22.5

SECONDARY outcome

Timeframe: Baseline to Week 8

Population: The intent to treat population included all participants who received at least one dose of IP. The primary approach to handling missing data was NRI method, where participants who had insufficient data for response determination at Week 8 were considered non-responders for Total Mayo score.

Clinical response in the TMS was defined as a decrease from baseline in the TMS of ≥ 3 points and ≥ 30%, along with a reduction in the RBS of ≥ 1 point or an absolute RBS of ≤ 1 at Week 8. The TMS is an instrument designed to measure disease activity of ulcerative colitis. The TMS ranges from 0 to 12 points. It consists of 4 subscores, each graded from 0 to 3 with higher scores indicating more severe disease. * Stool frequency subscore * Rectal bleeding subscore * Endoscopic subscore * Physician's Global Assessment

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Percentage of Participants Who Achieved a Clinical Response in the Total Mayo Score at Week 8	36.6 percentage of participants Interval 23.6 to 51.6

SECONDARY outcome

Timeframe: From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; maximum duration of treatment was 56 weeks

Population: Safety population included all participants who were enrolled and received at least 1 dose of IP.

A TEAE was defined as any adverse event (AE) occurring or worsening on or after the first treatment of mongersen and up to 28 days after the last mongersen dose or the last follow-up date, whichever occurred earlier. A serious AE = any AE which results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. The severity of AEs was assessed by the investigator and based on the following scale: Mild = asymptomatic or mild symptoms; clinical or diagnostic observations only; Moderate = Symptoms cause moderate discomfort; Severe (could be non-serious or serious) = symptoms causing severe discomfort/pain.

Outcome measures

Outcome measures
Measure	Mongersen (Weeks 0-52) n=41 Participants Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase, followed by 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase, weeks 8 through 52). Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) Any Serious IP-Related TEAE	0 Participants
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) Any TEAE	30 Participants
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) Any IP-Related TEAE	6 Participants
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) Any Serious TEAE (SAE)	5 Participants
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) Any TEAE Leading to IP Withdrawal	4 Participants
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) Any TEAE Leading to IP Interruption	1 Participants
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) Any Severe TEAE	5 Participants
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) Any TEAE Leading to Death	0 Participants

Adverse Events

Mongersen (Induction Phase: Weeks 0-8)

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Mongersen (Extension Phase: Weeks 8-52)

Serious events: 4 serious events

Other events: 14 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Mongersen (Induction Phase: Weeks 0-8) n=41 participants at risk Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase.	Mongersen (Extension Phase: Weeks 8-52) n=34 participants at risk Participants received oral mongersen 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase. Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Blood and lymphatic system disorders Anaemia	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	2.9% 1/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Gastrointestinal disorders Colitis	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	2.9% 1/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Gastrointestinal disorders Colitis ulcerative	2.4% 1/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	2.9% 1/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Hepatobiliary disorders Cholecystitis acute	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	2.9% 1/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks

Other adverse events

Other adverse events
Measure	Mongersen (Induction Phase: Weeks 0-8) n=41 participants at risk Participants received oral mongersen 160 mg tablets daily for 8 weeks during the induction phase.	Mongersen (Extension Phase: Weeks 8-52) n=34 participants at risk Participants received oral mongersen 160 mg tablets daily on an alternating dosing schedule for 4 weeks, followed by a 4 week break, (4 weeks on investigational product (IP), followed by 4 weeks off) for an additional 44 weeks during the extension phase. Participants who did not achieve at least a 20% decrease in a partial Mayo score (PMS) from baseline at Week 12 were discontinued from the study.
Blood and lymphatic system disorders Anaemia	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	5.9% 2/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Gastrointestinal disorders Colitis ulcerative	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	11.8% 4/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Gastrointestinal disorders Diarrhoea	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	5.9% 2/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Gastrointestinal disorders Nausea	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	5.9% 2/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Gastrointestinal disorders Vomiting	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	5.9% 2/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Infections and infestations Sinusitis	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	5.9% 2/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Infections and infestations Viral upper respiratory tract infection	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	8.8% 3/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/41 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks	5.9% 2/34 • From the first day of mongersen until 28 days after the last dose of IP or at follow-up visit, whichever occurred earlier; the maximum duration of treatment was 56 weeks

Additional Information

Anne McClain, Senior Manager, Clinical Trial Disclosure

Celgene Corporation

Phone: 888-260-1599

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Results from a center cannot be submitted for publication before results of multicenter study are published unless it is more than 1 year since study completion. Then, Investigator can publish if manuscript is submitted to Celgene 60 days prior to submission. If Celgene decides publication would hinder drug development, Investigator must delay submission for up to 90 days. Investigator must delete confidential information before submission and defer publication to permit patent applications.
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