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

Measurement and Analysis of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Patients With Autoimmune Diseases Treated With Mycophenolate (MPA)

Treatment with the immunosuppressive drug mycophenolate mofetil (MMF) may result in gastrointestinal (GI) complications in some patients. This study will 1) determine the proportion of patients with autoimmune diseases who are experiencing any GI complaints under MMF-based immunosuppressive treatment and 2) assess if a switch from MMF to enteric-coated mycophenolate sodium (EC-MPS) results in improved GI and/or health-related quality of life outcomes.

Study Overview

• Status: Completed
• Conditions: Autoimmune Disease
• Intervention / Treatment: Drug: Enteric-coated Mycophenolate Sodium

• Study Type: Interventional
• Enrollment (Actual): 111
• Phase: Phase 3

Contacts and Locations

Study Locations

• Germany
  • Various cities, Germany
    • Novartis Investigational site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion criteria:

1. Patients with autoimmune diseases;
2. receiving immunosuppressive therapy that includes MMF at time of study enrollment;
3. receiving immunosuppressive regimen that includes MMF at a stable dose for at least 1 month prior to enrollment. Patients can only be enrolled into the study if it is expected that treatment will continue at the same dose until study end (6-8 weeks after enrollment).

Exclusion criteria:

1. If applicable, GI symptoms assumed or known not to be caused by Mycophenolic acid (MPA) therapy (e.g. oral biphosphonates induced, infectious diarrhea);
2. Women of child-bearing potential who are planning to become pregnant or are pregnant and/or lactating or who are unwilling to use effective means of contraception;
3. Presence of psychiatric illness (i.e., schizophrenia, major depression) that, in the opinion of the site investigator, would interfere with study requirements;
4. Current acute medical intervention or hospitalization;
5. Presence of a medical condition not related to a GI event at time of visit, which requires immediate medical intervention.

Other protocol-defined inclusion/exclusion criteria may apply

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Drug: Enteric-coated Mycophenolate Sodium; Enteric-coated Mycophenolate Sodium (EC-MPS) 180 mg and 360 mg tablets were administered orally in divided doses twice daily.; Other Names: Myfortic

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS)	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.	Baseline and 6 - 8 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium	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).	Baseline and 6-8 weeks
Changes in GI-related Quality of Life Index (GIQLI), After Patients Are Converted From MMF to Enteric-coated Mycophenolate Sodium	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).	Baseline and 6-8 weeks
Changes in the GI-related Quality of Life Subscales After Conversion to Enteric-coated Mycophenolate Sodium	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.	Baseline and 6-8 weeks
Changes in Psychological General Well-Being Index (PGWB) After Conversion to Enteric-coated Mycophenolate Sodium	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.	Baseline and 6-8 weeks
Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium	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.	Baseline and 6-8 weeks
Overall Treatment Effects for GI Symptoms Assessed by the Physician	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.	6-8 week
Overall Treatment Effects for GI Symptoms Assessed by the Patient	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.	6-8 week
Overall Treatment Effects for for Health-related Quality of Life Assessed by the Patient	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.	6-8 week

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start: June 1, 2006
• Primary Completion (Actual): June 1, 2009
• Study Completion (Actual): June 1, 2009

Study Registration Dates

• First Submitted: July 10, 2006
• First Submitted That Met QC Criteria: July 10, 2006
• First Posted (Estimate): July 12, 2006

Study Record Updates

• Last Update Posted (Estimate): April 21, 2011
• Last Update Submitted That Met QC Criteria: April 19, 2011
• Last Verified: April 1, 2011

More Information

Terms related to this study

• Keywords: Autoimmune disease, mycophenolate, GI problems
• Additional Relevant MeSH Terms: Immune System Diseases; Autoimmune Diseases; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Enzyme Inhibitors; Antineoplastic Agents; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antitubercular Agents; Antibiotics, Antitubercular; Mycophenolic Acid
• Other Study ID Numbers: CERL080ADE08