Brief Summary
The purpose of study is to test the effects of an experimental medication GED-0301 (mongersen) in patients who have active Crohn's disease. The study will test GED-0301 compared to placebo for 52 weeks. The study treatment is blinded which means that patients and the study doctor will not know which treatment has been assigned. Patients in this study will be allowed treatment with stable doses of oral aminosalicylates, oral corticosteroids, immunosupressants and antibiotics for the treatment of Crohn's disease.
After 12 weeks in the study until the end of the study, patients who do not have an improvement in their Crohns disease symptoms will have the option to enter a long term active treatment study. Participants who discontinued the study anytime or completed the study at Week 52 were then observed for an additional 4 weeks.
After 12 weeks in the study until the end of the study, patients who do not have an improvement in their Crohns disease symptoms will have the option to enter a long term active treatment study. Participants who discontinued the study anytime or completed the study at Week 52 were then observed for an additional 4 weeks.
Brief Title
Efficacy and Safety Study of Mongersen (GED-0301) for the Treatment of Subjects With Active Crohn's Disease
Categories
Completion Date
Completion Date Type
Actual
Conditions
Crohn Disease
Eligibility Criteria
Inclusion Criteria:
Subjects must satisfy the following criteria to be enrolled in the study:
* Male or female ≥ 18 years
* Active Crohn's disease (CD) disease as determined by the Crohn's Disease Activity Index (CDAI) score and the Simple Endoscopic Score for Crohn's Disease (SES-CD)
* Must meet a determined average minimum number of daily stools or rating of abdominal pain over a 7 day period
* Subject must have failed or experienced intolerance to at least one of the following: budesonide; systemic corticosteroids; immunosuppressants (eg, azathiopurine, 6-mercaptopurine, or methotrexate); or biologics for the treatment of CD.
Exclusion Criteria:
The presence of any of the following will exclude a subject from enrollment:
* Diagnosis of ulcerative colitis (UC), indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis
* Local manifestations of Crohn's Disease (CD) such as symptomatic/severe strictures, abscesses, short bowel syndrome; or other disease complications for which surgery might be indicated or could confound the evaluation of efficacy
* Intestinal resection within 6 months or any intra-abdominal surgery within 3 months prior to the Screening Visit
* Ileostomy or a colostomy
* Subject has a history of any clinically significant medical condition that, in the investigator's opinion, would prevent the subject from participating in the study.
Subjects must satisfy the following criteria to be enrolled in the study:
* Male or female ≥ 18 years
* Active Crohn's disease (CD) disease as determined by the Crohn's Disease Activity Index (CDAI) score and the Simple Endoscopic Score for Crohn's Disease (SES-CD)
* Must meet a determined average minimum number of daily stools or rating of abdominal pain over a 7 day period
* Subject must have failed or experienced intolerance to at least one of the following: budesonide; systemic corticosteroids; immunosuppressants (eg, azathiopurine, 6-mercaptopurine, or methotrexate); or biologics for the treatment of CD.
Exclusion Criteria:
The presence of any of the following will exclude a subject from enrollment:
* Diagnosis of ulcerative colitis (UC), indeterminate colitis, ischemic colitis, microscopic colitis, radiation colitis or diverticular disease-associated colitis
* Local manifestations of Crohn's Disease (CD) such as symptomatic/severe strictures, abscesses, short bowel syndrome; or other disease complications for which surgery might be indicated or could confound the evaluation of efficacy
* Intestinal resection within 6 months or any intra-abdominal surgery within 3 months prior to the Screening Visit
* Ileostomy or a colostomy
* Subject has a history of any clinically significant medical condition that, in the investigator's opinion, would prevent the subject from participating in the study.
Inclusion Criteria
Inclusion Criteria:
Subjects must satisfy the following criteria to be enrolled in the study:
* Male or female ≥ 18 years
* Active Crohn's disease (CD) disease as determined by the Crohn's Disease Activity Index (CDAI) score and the Simple Endoscopic Score for Crohn's Disease (SES-CD)
* Must meet a determined average minimum number of daily stools or rating of abdominal pain over a 7 day period
* Subject must have failed or experienced intolerance to at least one of the following: budesonide; systemic corticosteroids; immunosuppressants (eg, azathiopurine, 6-mercaptopurine, or methotrexate); or biologics for the treatment of CD.
Subjects must satisfy the following criteria to be enrolled in the study:
* Male or female ≥ 18 years
* Active Crohn's disease (CD) disease as determined by the Crohn's Disease Activity Index (CDAI) score and the Simple Endoscopic Score for Crohn's Disease (SES-CD)
* Must meet a determined average minimum number of daily stools or rating of abdominal pain over a 7 day period
* Subject must have failed or experienced intolerance to at least one of the following: budesonide; systemic corticosteroids; immunosuppressants (eg, azathiopurine, 6-mercaptopurine, or methotrexate); or biologics for the treatment of CD.
Gender
All
Gender Based
false
Keywords
Crohn's Disease
GED-0301
Mongersen
IBD
Safety
Efficacy
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT02596893
Org Class
Industry
Org Full Name
Celgene
Org Study Id
GED-0301-CD-002
Overall Status
Terminated
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Investigate the Efficacy and Safety of Mongersen (GED-0301) for the Treatment of Subjects With Active Crohn's Disease
Primary Outcomes
Outcome Description
Clinical remission is defined as a Crohn's Disease Activity Index (CDAI) score \< 150. The Crohn's Disease Activity Index is used to quantify the signs and symptoms of Crohn's disease and the affect on patient's quality of life. It consists of 8 variables which include patient reported outcomes over a 7 day period and physician assessments which are scored numerically and weighted. Scores range from 0 to 600, with the most severe disease defined \>450.
Outcome Measure
The Percentage of Participants Who Achieved a Clinical Remission at Week 12
Outcome Time Frame
Week 12
Secondary Outcomes
Outcome Description
Clinical remission is defined as a CDAI score \< 150 and is used to quantify the signs and symptoms of Crohn's disease and the effect on patient's quality of life. It consists of 8 variables which include patient reported outcomes over a 7 day period and physician assessments which are scored numerically and weighted. Scores range from 0 to 600, with the most severe disease defined \>450.
Outcome Time Frame
Week 52
Outcome Measure
Percentage of Participants Who Achieved Clinical Remission at Week 52
Outcome Description
An endoscopic response-50 is defined as a reduction of at least 50% compared with baseline in simple endoscopic score for Crohn's Disease (SES-CD). The SES-CD assesses the size of mucosal ulcers, the extent of ulcerated surface, the extent of affected surface, and the presence and type of narrowings. Scores range from 0 to 60 with higher scores reflecting more severe disease. The SES-CD calculations include:
* Ulcers scored as:
0: no
1. aphthous (0.1-0.5 cm)
2. large (0.5-2 cm)
3. very large (\>2 cm)
* Surface involved disease 0: 0%
1. \<50%
2. 50-75%
3. \>75%
Surface involved by ulcerations:
0: 0%
1. \<10%
2. 10-30%
3. \>30% - Narrowings:
0: No
1. Single, can be passed
2. Multiple, can be passed
3. Cannot be passed Grand Total = SES-CD score
* Ulcers scored as:
0: no
1. aphthous (0.1-0.5 cm)
2. large (0.5-2 cm)
3. very large (\>2 cm)
* Surface involved disease 0: 0%
1. \<50%
2. 50-75%
3. \>75%
Surface involved by ulcerations:
0: 0%
1. \<10%
2. 10-30%
3. \>30% - Narrowings:
0: No
1. Single, can be passed
2. Multiple, can be passed
3. Cannot be passed Grand Total = SES-CD score
Outcome Time Frame
Week 52
Outcome Measure
Percentage of Participants With Endoscopic Response-50 Centrally Read at Week 52
Outcome Description
A clinical response is defined as a CDAI score decrease from baseline ≥ 100 points. The CDAI is used to quantify the signs and symptoms of Crohn's disease and the effect on patient's quality of life. It consists of 8 variables which include patient reported outcomes over a 7 day period and physician assessments which are scored numerically and weighted. Scores range from 0 to 600, with the most severe disease defined \>450.
Outcome Time Frame
Week 12
Outcome Measure
The Percentage of Participants Who Achieved a Clinical Response at Week 12
Outcome Description
A clinical response is defined as a decrease from baseline in CDAI ≥ 100 points. The Crohn's Disease Activity Index is used to quantify the signs and symptoms of Crohn's disease and the effect on patient's quality of life. It consists of 8 variables which include patient reported outcomes over a 7 day period and physician assessments which are scored numerically and weighted. Scores range from 0 to 600, with the most severe disease defined \>450.
Outcome Time Frame
Week 4
Outcome Measure
The Percentage of Participants Who Achieved a Clinical Response at Week 4
Outcome Description
A clinical remission is a CDAI score \< 150. The Crohn's Disease Activity Index is used to quantify the signs and symptoms of Crohn's disease and the effect on patient's quality of life. It consists of 8 variables which include patient reported outcomes over a 7 day period and physician assessments which are scored numerically and weighted. Scores range from 0 to 600, with the most severe disease defined \>450.
Outcome Time Frame
Week 4
Outcome Measure
The Percentage of Participants Who Achieved a Clinical Remission at Week 4
Outcome Description
The percentage of participants who were receiving oral corticosteroids for Crohn's disease, at baseline and achieved a clinical remission (CDAI score \<150) at Week 52 without corticosteroids. The Crohn's Disease Activity Index is used to quantify the signs and symptoms of Crohn's disease and the effect on patient's quality of life. It consists of 8 variables which include patient reported outcomes over a 7 day period and physician assessments which are scored numerically and weighted. Scores range from 0 to 600, with the most severe disease defined \>450.
Outcome Time Frame
Week 52
Outcome Measure
The Percentage of Participants Who Achieved a Corticosteroid-Free Clinical Remission at Week 52
Outcome Description
For participants who achieved a sustained clinical remission at both week 12 and 52, the clinical remission is a CDAI score \< 150. The Crohn's Disease Activity Index is used to quantify the signs and symptoms of Crohn's disease and the effect on patient's quality of life. It consists of 8 variables which include patient reported outcomes over a 7 day period and physician assessments which are scored numerically and weighted. Scores range from 0 to 600, with the most severe disease defined \>450.
Outcome Time Frame
Weeks 12 and 52
Outcome Measure
Percentage of Participants Who Achieved a Sustained Clinical Remission at Both Week 12 and 52
Outcome Description
An endoscopic response-25 is defined as a reduction of at least 25% compared with baseline in simple endoscopic score for Crohn's disease (SES-CD). The SES-CD assesses the size of mucosal ulcers, the extent of ulcerated surface, the extent of affected surface, and the presence and type of narrowings. Scores range from 0 to 60 with higher scores reflecting more severe disease. The SES-CD calculations include:
* Ulcers scored as:
0: no
1. aphthous (0.1-0.5 cm)
2. large (0.5-2 cm)
3. very large (\>2 cm)
* Surface involved disease 0: 0%
1. \<50%
2. 50-75%
3. \>75%
Surface involved by ulcerations:
0: 0%
1. \<10%
2. 10-30%
3. \>30% - Narrowings:
0: No
1. Single, can be passed
2. Multiple, can be passed
3. Cannot be passed Grand Total = SES-CD score
* Ulcers scored as:
0: no
1. aphthous (0.1-0.5 cm)
2. large (0.5-2 cm)
3. very large (\>2 cm)
* Surface involved disease 0: 0%
1. \<50%
2. 50-75%
3. \>75%
Surface involved by ulcerations:
0: 0%
1. \<10%
2. 10-30%
3. \>30% - Narrowings:
0: No
1. Single, can be passed
2. Multiple, can be passed
3. Cannot be passed Grand Total = SES-CD score
Outcome Time Frame
Week 0, Week 12
Outcome Measure
Percentage of Participants With Endoscopic Response-25 Centrally Read at Week 12
Outcome Description
Endoscopic remission is defined as a simple endoscopic score for Crohn's disease (SES-CD) of ≤2 at the specified timeframe. The SES-CD assesses the size of mucosal ulcers, the extent of ulcerated surface, the extent of affected surface, and the presence and type of narrowings. Scores range from 0 to 60 with higher scores reflecting more severe disease. The SES-CD calculations include:
* Ulcers scored as:
0: no
1. aphthous (0.1-0.5 cm)
2. large (0.5-2 cm)
3. very large (\>2 cm)
* Surface involved disease 0: 0%
1. \<50%
2. 50-75%
3. \>75%
Surface involved by ulcerations:
0: 0%
1. \<10%
2. 10-30%
3. \>30% - Narrowings:
0: No
1. Single, can be passed
2. Multiple, can be passed
3. Cannot be passed Grand Total = SES-CD score
* Ulcers scored as:
0: no
1. aphthous (0.1-0.5 cm)
2. large (0.5-2 cm)
3. very large (\>2 cm)
* Surface involved disease 0: 0%
1. \<50%
2. 50-75%
3. \>75%
Surface involved by ulcerations:
0: 0%
1. \<10%
2. 10-30%
3. \>30% - Narrowings:
0: No
1. Single, can be passed
2. Multiple, can be passed
3. Cannot be passed Grand Total = SES-CD score
Outcome Time Frame
Week 52
Outcome Measure
Percentage of Participants With Endoscopic Remission Centrally Read at Week 52
Outcome Description
A TEAE was defined as any adverse event (AE) occurring or worsening on or after the first treatment of GED-0301 and up to 28 days after the last GED-0301 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.
Outcome Time Frame
From the first day of GED-0301 until 28 days after the last dose of investigational product (IP); maximum treatment duration was 52.6 weeks
Outcome Measure
The Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE) From Week 0 to Week 52
Outcome Description
A TEAE was defined as any AE occurring or worsening on or after the first dose of GED-0301 and up to 28 days after the last GED-0301 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 Time Frame
From the first day of GED-0301 until 28 days after the last dose of IP; maximum treatment duration was 52.6 weeks
Outcome Measure
The Number of Participants Who Discontinued IP Due to an Treatment Emergent Adverse Events
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Dana Lukin
Investigator Email
dlukin@montefiore.org
Investigator Phone
917-769-8594
Categories Mesh Debug
Digestive System --- CROHN DISEASE
Digestive System --- INFLAMMATORY BOWEL DISEASES
Digestive System --- GASTROENTERITIS
Gastrointestinal (GI) Cancers --- GASTROINTESTINAL DISEASES
Digestive System --- GASTROINTESTINAL DISEASES
Gastrointestinal (GI) Cancers --- DIGESTIVE SYSTEM DISEASES
Digestive System --- DIGESTIVE SYSTEM DISEASES
Liver --- DIGESTIVE SYSTEM DISEASES
Gastrointestinal (GI) Cancers --- INTESTINAL DISEASES
Digestive System --- INTESTINAL DISEASES
MeSH Terms
CROHN DISEASE
INFLAMMATORY BOWEL DISEASES
GASTROENTERITIS
GASTROINTESTINAL DISEASES
DIGESTIVE SYSTEM DISEASES
INTESTINAL DISEASES
GED0301
COUNTERFEIT DRUGS
SUBSTANDARD DRUGS
PHARMACEUTICAL PREPARATIONS