Trial Outcomes & Findings for Gastrointestinal and Health-related Quality of Life Outcomes in Patients With Autoimmune Diseases Treated With Mycophenolate (NCT NCT00351377)

NCT ID: NCT00351377

Last Updated: 2011-04-21

Results Overview

Changes in GI symptom severity was measured by changes in the Gastrointestinal Symptom Rating Scale (GSRS) total score from baseline visit to the visit at 6-8 weeks. This total score was calculated as the average of the 15 single items (each ranging from 1-7 score points) and thus also had a range from 1-7 score points. Higher values indicate more unfavorable conditions.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

111 participants

Primary outcome timeframe

Baseline and 6 - 8 weeks

Results posted on

2011-04-21

Participant Flow

Participant milestones

Participant milestones
Measure
Enteric-coated Mycophenolate Sodium
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Overall Study
STARTED
111
Overall Study
COMPLETED
104
Overall Study
NOT COMPLETED
7

Reasons for withdrawal

Reasons for withdrawal
Measure
Enteric-coated Mycophenolate Sodium
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Overall Study
Adverse Event
5
Overall Study
Death
1
Overall Study
Unsatisfactory therapeutic effect
1

Baseline Characteristics

Gastrointestinal and Health-related Quality of Life Outcomes in Patients With Autoimmune Diseases Treated With Mycophenolate

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Age Continuous
49.5 years
STANDARD_DEVIATION 14.1 • n=99 Participants
Sex: Female, Male
Female
82 Participants
n=99 Participants
Sex: Female, Male
Male
29 Participants
n=99 Participants

PRIMARY outcome

Timeframe: Baseline and 6 - 8 weeks

Population: Intention to treat (ITT) population consisting of all enrolled participants who received study medication.

Changes in GI symptom severity was measured by changes in the Gastrointestinal Symptom Rating Scale (GSRS) total score from baseline visit to the visit at 6-8 weeks. This total score was calculated as the average of the 15 single items (each ranging from 1-7 score points) and thus also had a range from 1-7 score points. Higher values indicate more unfavorable conditions.

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS)
Baseline (N= 111)
2.28 Scores on a scale
Standard Deviation 1.13
Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS)
6-8 Weeks (N= 102)
2.02 Scores on a scale
Standard Deviation 0.93
Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS)
Change From Baseline (N = 102)
-0.28 Scores on a scale
Standard Deviation 0.92

SECONDARY outcome

Timeframe: Baseline and 6-8 weeks

Population: Intention to treat (ITT) population consisting of all enrolled participants who received study medication.

Changes in GI symptom severity was measured by changes in the total scores of 5 subscales (reflux, diarrhea, constipation, abdominal pain and indigestion) of the Gastrointestinal Symptom Rating Scale (GSRS) from baseline visit to the visit at 6-8 weeks. The GSRS is a 15-item instrument with a mean subscale score ranging from 1 (no discomfort) to 7 (very severe discomfort).

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Reflux [n =102]
-0.24 Scores on a scale
Standard Deviation 1.36
Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Diarrhea [n =104]
-0.27 Scores on a scale
Standard Deviation 1.43
Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Constipation [n = 104]
0.02 Scores on a scale
Standard Deviation 1.18
Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Abdominal pain [n =102]
-0.51 Scores on a scale
Standard Deviation 1.20
Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Indigestion [n = 104]
-0.42 Scores on a scale
Standard Deviation 1.33

SECONDARY outcome

Timeframe: Baseline and 6-8 weeks

Population: Intention to treat (ITT) population consisting of all enrolled participants who received study medication.

Assessed by changes in the Gastrointestinal Quality of Life Index (GIQLI) from Baseline visit to the 6-8 week visit. The GIQLI is a 36-item questionnaire and consists of 5 different subscales. The total score was calculated as the sum of the 36 single items which each ranged from 0-4, leading to a hypothetical range from 0-144 score points (lower scores indicate more unfavorable conditions). The mean change was calculated as (6-8 week visit value) minus (Baseline value).

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Changes in GI-related Quality of Life Index (GIQLI), After Patients Are Converted From MMF to Enteric-coated Mycophenolate Sodium
Baseline (N= 111)
100.2 Scores on a scale
Standard Deviation 23.8
Changes in GI-related Quality of Life Index (GIQLI), After Patients Are Converted From MMF to Enteric-coated Mycophenolate Sodium
6-8 Weeks (N= 104)
105.6 Scores on a scale
Standard Deviation 22.9
Changes in GI-related Quality of Life Index (GIQLI), After Patients Are Converted From MMF to Enteric-coated Mycophenolate Sodium
Change from Baseline (N= 104)
5.8 Scores on a scale
Standard Deviation 18.6

SECONDARY outcome

Timeframe: Baseline and 6-8 weeks

Population: Intention to treat (ITT) population consisting of all enrolled participants who received study medication.

The 5 different subscales of the GI-related Quality of Life (GIQLI) were analyzed separately by calculating the average value of the items that were included in the respective subscore. Thus, the theoretical range for each of the subscores was the same as for the single items, i.e. 0-4 score points. An increase in the subscale score indicates an improvement in symptoms.

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Changes in the GI-related Quality of Life Subscales After Conversion to Enteric-coated Mycophenolate Sodium
GI symptoms [n=103]
0.18 Scores on a scale
Standard Deviation 0.53
Changes in the GI-related Quality of Life Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Emotional status [n=103]
0.15 Scores on a scale
Standard Deviation 0.68
Changes in the GI-related Quality of Life Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Physical function [n=102]
0.22 Scores on a scale
Standard Deviation 0.68
Changes in the GI-related Quality of Life Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Social function [n=102]
0.10 Scores on a scale
Standard Deviation 0.66
Changes in the GI-related Quality of Life Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Stress of medical treatment [n=102]
0.19 Scores on a scale
Standard Deviation 1.22

SECONDARY outcome

Timeframe: Baseline and 6-8 weeks

Population: Intention to treat (ITT) population consisting of all enrolled participants who received study medication.

The PGWB consists of 22 single items (each ranging from 0-5) with 7 dimensions (including the total score) to be calculated. Lower scores indicate more unfavorable conditions. The total raw score is calculated by summing up all of the single items and thus has a hypothetical range from 0-110 score points. This raw score is further transformed using the formula: (raw score / 110) x 100 to fit a range from 0-100.

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=110 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Changes in Psychological General Well-Being Index (PGWB) After Conversion to Enteric-coated Mycophenolate Sodium
Baseline (N= 110)
65.3 Scores on a scale
Standard Deviation 16.1
Changes in Psychological General Well-Being Index (PGWB) After Conversion to Enteric-coated Mycophenolate Sodium
6-8 Weeks (N= 103)
66.8 Scores on a scale
Standard Deviation 15.6
Changes in Psychological General Well-Being Index (PGWB) After Conversion to Enteric-coated Mycophenolate Sodium
Change From Baseline (N= 103)
2.0 Scores on a scale
Standard Deviation 11.1

SECONDARY outcome

Timeframe: Baseline and 6-8 weeks

Population: Intention to treat (ITT) population consisting of all enrolled participants who received study medication.

The change from baseline to the 6-8 week visit for each of the six subscores (each ranging from 0-5) of the PGWB were analyzed individually. Each of the subscores was transformed to fit a range from 0-100. Lower scores indicate more unfavorable conditions, so an increase in score indicates an improvement in symptoms.

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium
General health [n=104]
3.4 Scores on a scale
Standard Deviation 15.1
Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Vitality [n=104]
2.7 Scores on a scale
Standard Deviation 16.0
Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Anxiety [n=103]
1.3 Scores on a scale
Standard Deviation 14.1
Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Depressed mood [n=104]
1.5 Scores on a scale
Standard Deviation 14.1
Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Positive well-being [n=104]
0.7 Scores on a scale
Standard Deviation 14.4
Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Self control [n=104]
1.5 Scores on a scale
Standard Deviation 15.0

SECONDARY outcome

Timeframe: 6-8 week

Population: Intention to treat population consisting of all enrolled participants who received study medication.

Assessed using the Overall Treatment Effects for GI symptoms questionnaire. The question was: "Has there been any change in the participant's GI symptoms since his/her last study visit? Please indicate if there has been any change in his/her symptoms." The possible answers were: "Improved", "about the same", or "worse. The questionnaire was completed by the physician.

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Overall Treatment Effects for GI Symptoms Assessed by the Physician
Improved
49 Participants
Overall Treatment Effects for GI Symptoms Assessed by the Physician
About the same
43 Participants
Overall Treatment Effects for GI Symptoms Assessed by the Physician
Worsened
13 Participants
Overall Treatment Effects for GI Symptoms Assessed by the Physician
Missing data
6 Participants

SECONDARY outcome

Timeframe: 6-8 week

Population: Intention to treat population consisting of all enrolled participants who received study medication.

Assessed using the Overall Treatment Effects for GI symptoms questionnaire. The question was: "Has there been any change in the participant's GI symptoms since his/her last study visit? Please indicate if there has been any change in his/her symptoms." The possible answers were: "Improved", "about the same", or "worse. The questionnaire was completed by the patient.

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Overall Treatment Effects for GI Symptoms Assessed by the Patient
Missing data
11 Participants
Overall Treatment Effects for GI Symptoms Assessed by the Patient
Improved
38 Participants
Overall Treatment Effects for GI Symptoms Assessed by the Patient
About the same
53 Participants
Overall Treatment Effects for GI Symptoms Assessed by the Patient
Worsened
9 Participants

SECONDARY outcome

Timeframe: 6-8 week

Population: Intention to treat population consisting of all enrolled participants who received study medication.

Assessed using the Overall Treatment Effects for health-related quality of life questionnaire. Possible answers were: "Improved", "about the same", or "worse. The questionnaire was completed by the patient.

Outcome measures

Outcome measures
Measure
Enteric-coated Mycophenolate Sodium
n=111 Participants
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Overall Treatment Effects for for Health-related Quality of Life Assessed by the Patient
Improved
31 Participants
Overall Treatment Effects for for Health-related Quality of Life Assessed by the Patient
Worsened
10 Participants
Overall Treatment Effects for for Health-related Quality of Life Assessed by the Patient
About the same
61 Participants
Overall Treatment Effects for for Health-related Quality of Life Assessed by the Patient
Missing data
9 Participants

Adverse Events

Enteric-coated Mycophenolate Sodium

Serious events: 6 serious events
Other events: 38 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Enteric-coated Mycophenolate Sodium
n=111 participants at risk
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Blood and lymphatic system disorders
Nephrogenic anaemia
0.90%
1/111
Blood and lymphatic system disorders
Thrombocytopenia
0.90%
1/111
Gastrointestinal disorders
Diverticulum
0.90%
1/111
Infections and infestations
Bronchitis
0.90%
1/111
Infections and infestations
Endocarditis
0.90%
1/111
Infections and infestations
Pneumonia
0.90%
1/111
Infections and infestations
Septic shock
0.90%
1/111
Nervous system disorders
Dementia
0.90%
1/111
Nervous system disorders
Disturbance in attention
0.90%
1/111
Nervous system disorders
Dizziness
0.90%
1/111
Nervous system disorders
Syncope
0.90%
1/111
Respiratory, thoracic and mediastinal disorders
Dyspnoea
0.90%
1/111
Respiratory, thoracic and mediastinal disorders
Pulmonary fibrosis
0.90%
1/111

Other adverse events

Other adverse events
Measure
Enteric-coated Mycophenolate Sodium
n=111 participants at risk
Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily in a dose that was equimolar to the dose of Mycophenolate mofetil the participant was taking at the time of study entry. The planned duration of treatment 6 to 8 weeks.
Gastrointestinal disorders
Abdominal distension
7.2%
8/111
Gastrointestinal disorders
Diarrhoea
14.4%
16/111
Gastrointestinal disorders
Dyspepsia
7.2%
8/111
Gastrointestinal disorders
Nausea
11.7%
13/111
Gastrointestinal disorders
Vomiting
9.0%
10/111
Infections and infestations
Nasopharyngitis
9.0%
10/111

Additional Information

Study Director

Novartis Pharmaceuticals

Phone: 862-778-8300

Results disclosure agreements

  • Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial.
  • Publication restrictions are in place

Restriction type: OTHER