Kuvan® (sapropterin dihydrochloride) for Improving Gastric Accommodation in Women with Diabetic Gastroparesis (KIGA-DG)
Patients are invited to participate in a research study of Kuvan® (sapropterin dihydrochloride). We hope to learn whether treatment with Kuvan® is safe and effective in improving the ability of the stomach to relax after eating and improving the symptoms of diabetic gastroparesis. Women are selected as a possible participant in this study because they have diabetes and moderate to severe gastroparesis (meaning stomach empties slowly).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
9
sapropterin dihydrochloride: 10mg/kg/day
Stanford University School of Medicine
Stanford, California, United States
Gastric Accommodation
Gastric Accommodation refers to the reflexive relaxation of the upper stomach after swallowing as measured by the Satiety Test at baseline, 4 weeks, and 8 weeks. Increased gastric accommodation is considered a positive outcome.
Time frame: Baseline, 4 Weeks, 8 Weeks
Secondary Outcome Measures Will Include Change in Symptom Severity.
Change in symptom severity over the past 2 weeks as measured by the patient reported Gastroparesis Cardinal Symptom Index (GCSI) The GCSI is composed of 9 questions. Each question asks about symptom severity on a scale of 1-5 listed below. 0=None 1= Very mild 2= Mild 3=Moderate 4=Severe 5= Very severe The 9 scores are summed together for cumulative GCSI score. The minimum cumulative score is '"0" and the maximum cumulative score is "45." Once we calculated the cumulative score for each participant, we took the average of all participants cumulative scores giving us the sample mean. This was done at baseline, week 4, and week 8. Higher total scores indicate higher symptom severity. Lower total scores indicate lower symptom severity.
Time frame: Baseline, 4 Weeks, 8 Weeks
Secondary Outcome Measures Will Include Change in Quality of Life.
Quality of life as impacted by patients with upper gastrointestinal disorders (PAGI-QOL) The PAGI-QOl is composed of 30 questions. Each question asks about the degree to which a patient's quality of life is impacted by upper gastrointestinal disorders. Questions measure quality of life impact on a scale of 1-5 listed below. 0=none of the time, 1=a little of the time, 2=some of the time, 3=a good bit of the time, 4= most of the time, 5= all of time The 30 items are summed together for a cumulative PAGI-QOL score. The minimum cumulative score is "0" and the maximum score is "150." Once we calculated the cumulative score for each participant, we took the average of all participants cumulative scores giving us the sample mean. This was done at baseline, week 4, and week 8. Lower scores indicate an improved overall quality of life. Higher scores indicate a diminished overall quality of life.
Time frame: Baseline, 4 Weeks, 8 Weeks
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Secondary Outcome Measures Will Include Change in Symptom Severity as Measured by the Patient Assessment of Gastrointestinal Disorder-Symptom Severity Index Disorders Symptom Severity Index (PAGI-SYM)
Symptom severity as measured by the Patient Assessment of Upper Gastrointestinal Disorder- Symptom Severity Index. (PAGI-SYM) The PAGI-SYM is compose of 20 questions. Each question can be answered on a scale of 1-5 (below) 0 = none, 1=very mild, 2=mild, 3=moderate, 4=severe, 5=very severe Cumulative scores were calculated by summing all 20 questions. The minimum cumulative scores would be "0" while the maximum cumulative score would be "100" Once we calculated the cumulative score for each participant, we took the average of all the cumulative scores giving us the sample mean. This was done at baseline, week 4, and week 8. Lower score indicates decreased symptom severity. Higher score indicates increased symptom severity.
Time frame: Baseline, 4 Weeks, 8 Weeks