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Impact of KUVAN® on Gastric Relaxation in Women With Diabetic Gastroparesis.

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Linda Nguyen, Stanford University
ClinicalTrials.gov Identifier:
NCT01135186
First received: May 28, 2010
Last updated: February 9, 2017
Last verified: February 2017
  Purpose
Kuvan® (sapropterin dihydrochloride) for Improving Gastric Accommodation in Women with Diabetic Gastroparesis (KIGA-DG)

Condition Intervention
Gastroparesis Drug: sapropterin dihydrochloride

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Kuvan® (Sapropterin Dihydrochloride) for Improving Gastric Accommodation in Women With Diabetic Gastroparesis (KIGA-DG)

Resource links provided by NLM:


Further study details as provided by Linda Nguyen, Stanford University:

Primary Outcome Measures:
  • Gastric Accommodation [ Time Frame: Baseline, 4 Weeks, 8 Weeks ]

    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.



Secondary Outcome Measures:
  • Secondary Outcome Measures Will Include Change in Symptom Severity. [ Time Frame: Baseline, 4 Weeks, 8 Weeks ]

    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.


  • Secondary Outcome Measures Will Include Change in Quality of Life. [ Time Frame: Baseline, 4 Weeks, 8 Weeks ]

    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.


  • 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) [ Time Frame: Baseline, 4 Weeks, 8 Weeks ]

    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.



Enrollment: 9
Study Start Date: May 2010
Study Completion Date: November 2012
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Sapropterin
open label study of sapropterin dihydrochloride
Drug: sapropterin dihydrochloride
sapropterin dihydrochloride: 10mg/kg/day
Other Name: KUVAN

Detailed Description:
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).
  Eligibility

Ages Eligible for Study:   18 Years to 65 Years   (Adult)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:Inclusion Criteria

Patients with moderate to severe symptoms of diabetic gastroparesis will be studied (GCSI >21). Patients should not have post-surgical gastroparesis or dyspeptic symptoms with normal gastric emptying. In order to qualify for inclusion in the trial, patients must satisfy the following inclusion criteria:

  1. Diagnosis of diabetes mellitus > 5 years requiring medical therapy
  2. Female gender
  3. Ages 18-65 years
  4. Documentation of delayed gastric emptying on gastric emptying scintigraphy (within 2 years of enrollment)
  5. Symptoms of gastroparesis for at least 6 months with Gastroparesis Cardinal Symptom Index (GCSI) score > 21 indicating moderate to severe symptoms
  6. Recent negative upper endoscopy or upper GI series within 2 years of enrollment (no evidence of mechanical obstruction or peptic ulcer disease)

Exclusion Criteria:Exclusion Criteria

Patients who satisfy any of the following exclusion criteria will be ineligible for enrollment in the study:

  1. Diabetes diagnosed < 5 years prior to the study
  2. Male gender
  3. Normal gastric emptying
  4. Gastroparesis from post-surgical etiologies
  5. Another active disorder, which could explain symptoms in the opinion of the investigator
  6. Pregnancy
  7. History of significant cardiac arrhythmias and/or prolonged QTc
  8. Daily use of narcotic analgesics for abdominal pain
  9. Contraindications to gastric emptying breath test: patients with a known allergy to egg, wheat, or algae.
  10. Underlying seizure disorder
  11. Known history of cardiac ischemia
  12. Recent clinically significant gastrointestinal bleeding
  13. Patients taking Levodopa
  14. Any other condition, which in the opinion of the investigator would impede compliance or hinder the completion of the study
  15. Failure to give informed consent
  16. Surgery for placement of a gastric stimulator within the past 6 months (patients > 6 months post-op with persistent symptoms and delayed gastric emptying are eligible).
  17. A normal upper endoscopy not performed within 2 year of study entry.
  18. Patients taking phosphodiesterase inhibitors such as sildenafil, vardenafil, tadalafil
  19. Patients with renal impairment (i.e. Creatinine > 2.5 mg/dL)
  20. Patients with hepatic dysfunction (i.e. ALT and AST values > 2.5x ULN and T. bilirubin > 1.5x ULN)
  21. Patients with uncontrolled diabetes, such as HbA1c > 10 mg/dl at screening/baseline will be excluded.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01135186

Locations
United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Investigators
OverallOfficial: Dr. Linda Nguyen Stanford University
Principal Investigator: Pankaj Jay Pasricha Stanford University
  More Information

Publications:
Responsible Party: Linda Nguyen, Principle Investigator, Stanford University
ClinicalTrials.gov Identifier: NCT01135186     History of Changes
Other Study ID Numbers: SU-03252010-5462
Study First Received: May 28, 2010
Results First Received: October 13, 2016
Last Updated: February 9, 2017

Additional relevant MeSH terms:
Gastroparesis
Stomach Diseases
Gastrointestinal Diseases
Digestive System Diseases
Paralysis
Neurologic Manifestations
Signs and Symptoms
Verapamil
Anti-Arrhythmia Agents
Calcium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Vasodilator Agents

ClinicalTrials.gov processed this record on September 18, 2017