Trial Outcomes & Findings for An Explorative Trial to Evaluate the Pharmacodynamic Effect of SPD557 on Reflux Parameters in Refractory GERD Patients (NCT NCT01370863)
NCT ID: NCT01370863
Last Updated: 2021-06-10
Results Overview
This is used to characterize gastric reflux events. The measurements were made over a 24-hour period at baseline and again at week 4.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
67 participants
Primary outcome timeframe
Baseline and 4 weeks
Results posted on
2021-06-10
Participant Flow
Participant milestones
| Measure |
SPD557
0.5 mg tablet administered 3 times daily (t.i.d.) for 4 weeks in addition to stable proton pump inhibitor (PPI) treatment
|
Placebo
Matching placebo tablet administered three times daily (t.i.d.) for 4 weeks in addition to stable PPI treatment
|
|---|---|---|
|
Overall Study
STARTED
|
34
|
33
|
|
Overall Study
COMPLETED
|
32
|
30
|
|
Overall Study
NOT COMPLETED
|
2
|
3
|
Reasons for withdrawal
| Measure |
SPD557
0.5 mg tablet administered 3 times daily (t.i.d.) for 4 weeks in addition to stable proton pump inhibitor (PPI) treatment
|
Placebo
Matching placebo tablet administered three times daily (t.i.d.) for 4 weeks in addition to stable PPI treatment
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
1
|
|
Overall Study
Did not want second pH/MII monitoring
|
1
|
1
|
|
Overall Study
Not meeting inclusion/exclusion criteria
|
0
|
1
|
Baseline Characteristics
An Explorative Trial to Evaluate the Pharmacodynamic Effect of SPD557 on Reflux Parameters in Refractory GERD Patients
Baseline characteristics by cohort
| Measure |
SPD557
n=34 Participants
0.5 mg tablet t.i.d. for 4 weeks in addition to stable proton pump inhibitor (PPI) treatment
|
Placebo
n=31 Participants
Matching placebo tablet t.i.d. for 4 weeks in addition to stable PPI treatment
|
Total
n=65 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
43.8 years
STANDARD_DEVIATION 16.04 • n=5 Participants
|
45.8 years
STANDARD_DEVIATION 14.50 • n=7 Participants
|
44.8 years
STANDARD_DEVIATION 15.24 • n=5 Participants
|
|
Age, Customized
Between 18 and 70 years inclusive
|
34 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
65 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
|
Region of Enrollment
France
|
13 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
8 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Region of Enrollment
Switzerland
|
4 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and 4 weeksPopulation: Pharmacodynamics Population (PD) defined as all randomized subjects with at least 1 administration of the investigational product and with both a baseline and post-baseline PD assessment.
This is used to characterize gastric reflux events. The measurements were made over a 24-hour period at baseline and again at week 4.
Outcome measures
| Measure |
SPD557
n=31 Participants
0.5 mg tablet t.i.d. for 4 weeks in addition to stable proton pump inhibitor (PPI) treatment
|
Placebo
n=29 Participants
Matching placebo tablet t.i.d. for 4 weeks in addition to stable PPI treatment
|
|---|---|---|
|
Change From Baseline in the Number of Liquid-containing Reflux Events (pH/MII Monitoring) at 4 Weeks
|
-17.9 Number of Reflux Events
Standard Deviation 59.88
|
-13.7 Number of Reflux Events
Standard Deviation 37.58
|
SECONDARY outcome
Timeframe: Baseline and 4 weeksPopulation: Pharmacodynamics Population (PD) defined as all randomized subjects with at least 1 administration of the investigational product and with both a baseline and post-baseline PD assessment.
Outcome measures
| Measure |
SPD557
n=31 Participants
0.5 mg tablet t.i.d. for 4 weeks in addition to stable proton pump inhibitor (PPI) treatment
|
Placebo
n=29 Participants
Matching placebo tablet t.i.d. for 4 weeks in addition to stable PPI treatment
|
|---|---|---|
|
Change From Baseline in the Number of Days With Heartburn and/or Regurgitation at 4 Weeks
|
-0.90 Number of days
Standard Deviation 1.434
|
-0.88 Number of days
Standard Deviation 1.610
|
SECONDARY outcome
Timeframe: Baseline and 4 weeksPopulation: Pharmacodynamics Population (PD) defined as all randomized subjects with at least 1 administration of the investigational product and with both a baseline and post-baseline PD assessment.
The PAGI-SYM contains 20 items and the scores range from 0 (no symptoms)-5 (very severe symptoms) for each item with a total score of 0-100. Higher scores indicate more severe gastrointestinal symptoms.
Outcome measures
| Measure |
SPD557
n=31 Participants
0.5 mg tablet t.i.d. for 4 weeks in addition to stable proton pump inhibitor (PPI) treatment
|
Placebo
n=29 Participants
Matching placebo tablet t.i.d. for 4 weeks in addition to stable PPI treatment
|
|---|---|---|
|
Change From Baseline in the Patient Assessment of Upper Gastrointestinal Symptom Severity Index (PAGI-SYM) Questionnaire at 4 Weeks
|
-0.41 Units on a scale
Standard Deviation 0.943
|
-0.37 Units on a scale
Standard Deviation 0.760
|
Adverse Events
SPD557
Serious events: 0 serious events
Other events: 25 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
SPD557
n=34 participants at risk
0.5 mg tablet t.i.d. for 4 weeks in addition to stable proton pump inhibitor (PPI) treatment
|
Placebo
n=31 participants at risk
Matching placebo tablet t.i.d. for 4 weeks in addition to stable PPI treatment
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
38.2%
13/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
6.5%
2/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Gastrointestinal disorders
Abdominal pain
|
8.8%
3/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
6.5%
2/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Gastrointestinal disorders
Nausea
|
8.8%
3/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
6.5%
2/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.9%
2/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
0.00%
0/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Gastrointestinal disorders
Dyspepsia
|
5.9%
2/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
3.2%
1/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
6.5%
2/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Nervous system disorders
Headache
|
35.3%
12/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
12.9%
4/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Nervous system disorders
Dizziness
|
5.9%
2/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
6.5%
2/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Infections and infestations
Influenza
|
2.9%
1/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
6.5%
2/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Infections and infestations
Nasopharyngitis
|
8.8%
3/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
3.2%
1/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
6.5%
2/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
|
General disorders
Edema peripheral
|
0.00%
0/34
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
6.5%
2/31
The Safety Population was defined as all subjects randomized into the study with at least 1 administration of the investigational product. Two subjects never received investigational product (n = 65).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER