Trial Outcomes & Findings for A Placebo-controlled Trial With rFXIII Administered to Subjects With Mild to Moderate Active Ulcerative Colitis (NCT NCT01706159)
NCT ID: NCT01706159
Last Updated: 2014-10-02
Results Overview
The primary endpoint was the binary variable ("responder" vs. "non-responder") where "responders" were the subjects with endoscopic remission (endoscopic mucosal healing) at Week 8, defined as a modified Baron score of 0. Subjects with a modified Baron score ≥1 were designated as "non-responders".
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
20 participants
Primary outcome timeframe
At week 8
Results posted on
2014-10-02
Participant Flow
The trial was conducted at 12 sites in 6 countries as follows: Bulgaria (4 sites), Denmark (1 site), Hungary (1 site), Poland (4 sites), Russian Federation (1 site) and Ukraine (1 site).
No pre-assignments were given for this trial.
Participant milestones
| Measure |
rFXIII
Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.
|
Placebo
Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.
|
|---|---|---|
|
Overall Study
STARTED
|
14
|
6
|
|
Overall Study
Exposed
|
13
|
5
|
|
Overall Study
Completers (With Baron Score at Week 8)
|
6
|
3
|
|
Overall Study
COMPLETED
|
6
|
4
|
|
Overall Study
NOT COMPLETED
|
8
|
2
|
Reasons for withdrawal
| Measure |
rFXIII
Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.
|
Placebo
Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.
|
|---|---|---|
|
Overall Study
Withdrawal Criteria
|
2
|
1
|
|
Overall Study
Unclassified
|
6
|
1
|
Baseline Characteristics
A Placebo-controlled Trial With rFXIII Administered to Subjects With Mild to Moderate Active Ulcerative Colitis
Baseline characteristics by cohort
| Measure |
rFXIII
n=13 Participants
Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.
|
Placebo
n=5 Participants
Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.
|
Total
n=18 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
36.9 years
STANDARD_DEVIATION 10.6 • n=5 Participants
|
45.0 years
STANDARD_DEVIATION 15.4 • n=7 Participants
|
39.2 years
STANDARD_DEVIATION 12.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Whites
|
13 participants
n=5 Participants
|
5 participants
n=7 Participants
|
18 participants
n=5 Participants
|
|
Body Mass Index
|
24.5 kg/m^2
STANDARD_DEVIATION 3.2 • n=5 Participants
|
25.6 kg/m^2
STANDARD_DEVIATION 5.8 • n=7 Participants
|
24.8 kg/m^2
STANDARD_DEVIATION 3.9 • n=5 Participants
|
PRIMARY outcome
Timeframe: At week 8Population: Full analysis set (FAS) included all randomised and treated subjects.
The primary endpoint was the binary variable ("responder" vs. "non-responder") where "responders" were the subjects with endoscopic remission (endoscopic mucosal healing) at Week 8, defined as a modified Baron score of 0. Subjects with a modified Baron score ≥1 were designated as "non-responders".
Outcome measures
| Measure |
rFXIII
n=13 Participants
Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.
|
Placebo
n=5 Participants
Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.
|
|---|---|---|
|
Endoscopic Remission Defined as a Modified Baron Score of 0
|
1 Subjects
|
1 Subjects
|
SECONDARY outcome
Timeframe: At Week 8Population: Full analysis set (FAS) included all randomised and treated subjects. Two subjects in the rFXIII group had no UC-DAI score at any visit, including baseline and they were excluded from the analysis.
Analysis of responders defined by a clinical component of: ulcerative colitis disease activity index (UC-DAI) score of less than or equal to 1 with 0 for rectal bleeding and 0 for stool frequency and an endoscopic component of: no mucosal friability (modified Baron score less than or equal to 1).
Outcome measures
| Measure |
rFXIII
n=11 Participants
Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.
|
Placebo
n=5 Participants
Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.
|
|---|---|---|
|
Remission (Clinical and Endoscopic)
|
0 Subjects
|
0 Subjects
|
SECONDARY outcome
Timeframe: Week 0 to 10Population: Safety analysis set included all randomised and treated subjects.
Number of adverse events reported from the first trial-related activity, after the subject was exposed to the trial drug, until the end of the post-treatment follow-up period.
Outcome measures
| Measure |
rFXIII
n=13 Participants
Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.
|
Placebo
n=5 Participants
Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.
|
|---|---|---|
|
Number of Adverse Events (AEs)
Adverse events
|
7 events
|
4 events
|
|
Number of Adverse Events (AEs)
Serious adverse events
|
1 events
|
0 events
|
|
Number of Adverse Events (AEs)
Severe adverse events
|
1 events
|
0 events
|
|
Number of Adverse Events (AEs)
Moderate adverse events
|
0 events
|
2 events
|
|
Number of Adverse Events (AEs)
Mild adverse events
|
6 events
|
2 events
|
|
Number of Adverse Events (AEs)
Fatal adverse events
|
0 events
|
0 events
|
SECONDARY outcome
Timeframe: Samples were collected before and up to 72 hours after the first dose of rFXIII.Population: It was not possible to obtain credible single dose pharmakokinetics (PK) for rFXIII in this trial due to a small number of subjects with required PK measurements and a spurious behaviour of individual PK profiles.
The volume of plasma cleared of the drug per unit time.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Samples were collected before and up to 72 hours after the first dose of rFXIII.Population: It was not possible to obtain credible single dose PK for rFXIII in this trial due to a small number of subjects with required PK measurements and a spurious behaviour of individual PK profiles.
The peak plasma concentration of the drug after dose administration.
Outcome measures
Outcome data not reported
Adverse Events
rFXIII
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
rFXIII
n=13 participants at risk
Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.
|
Placebo
n=5 participants at risk
Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.
|
|---|---|---|
|
Gastrointestinal disorders
Colitis ulcerative
|
7.7%
1/13 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
0.00%
0/5 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
Other adverse events
| Measure |
rFXIII
n=13 participants at risk
Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.
|
Placebo
n=5 participants at risk
Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
0.00%
0/13 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
20.0%
1/5 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
Gastrointestinal disorders
Colitis ulcerative
|
0.00%
0/13 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
20.0%
1/5 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
General disorders
Asthenia
|
7.7%
1/13 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
0.00%
0/5 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
General disorders
Fatigue
|
0.00%
0/13 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
20.0%
1/5 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
General disorders
Oedema peripheral
|
7.7%
1/13 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
0.00%
0/5 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
Infections and infestations
Bronchitis
|
0.00%
0/13 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
20.0%
1/5 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
Infections and infestations
Urinary tract infection
|
7.7%
1/13 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
0.00%
0/5 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
Investigations
Blood creatinine increased
|
7.7%
1/13 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
0.00%
0/5 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
7.7%
1/13 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
0.00%
0/5 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
7.7%
1/13 • Number of events 1 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
0.00%
0/5 • Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).
Safety analysis set was identical to the FAS which included all randomised and treated subjects.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Novo Nordisk maintains the right to be informed of any Investigator plans for publication and to review any scientific paper, presentation, communication or other information concerning the investigation described in this protocol. Novo Nordisk reserves the right to prior review of such publications and to ask for delay of publication of individual site results until after the primary manuscript is accepted for publication.
- Publication restrictions are in place
Restriction type: OTHER