GastroparesisNCT01135186

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

A study testing sapropterin dihydrochloride in people with Gastroparesis, run by Stanford University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Kuvan® (sapropterin dihydrochloride) for Improving Gastric Accommodation in Women with Diabetic Gastroparesis (KIGA-DG) Read more about this study

Who is it for?: Ages 18 to 65 years, women only
Conditions the study is for: Gastroparesis
What is being tested?: sapropterin dihydrochloride
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 9 participants
Where: 1 siteUnited States
Study dates: May 2010to Nov 2012
Phase: Interventional
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 65 years
Women only
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

Patients who satisfy any of the following exclusion criteria will be ineligible for enrollment in the study:
Diabetes diagnosed < 5 years prior to the study
Male gender
Normal gastric emptying
Gastroparesis from post-surgical etiologies
Another active disorder, which could explain symptoms in the opinion of the investigator

Show all 22Show fewer

Pregnancy
History of significant cardiac arrhythmias and/or prolonged QTc
Daily use of narcotic analgesics for abdominal pain
Contraindications to gastric emptying breath test: patients with a known allergy to egg, wheat, or algae.
Underlying seizure disorder
Known history of cardiac ischemia
Recent clinically significant gastrointestinal bleeding
Patients taking Levodopa
Any other condition, which in the opinion of the investigator would impede compliance or hinder the completion of the study
Failure to give informed consent
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).
A normal upper endoscopy not performed within 2 year of study entry.
Patients taking phosphodiesterase inhibitors such as sildenafil, vardenafil, tadalafil
Patients with renal impairment (i.e. Creatinine > 2.5 mg/dL)
Patients with hepatic dysfunction (i.e. ALT and AST values > 2.5x ULN and T. bilirubin > 1.5x ULN)
Patients with uncontrolled diabetes, such as HbA1c > 10 mg/dl at screening/baseline will be excluded.

Trial locations and contacts

1 site in 1 country.

United States1 site

Stanford University School of Medicine
Stanford, California, 94305

Other trials in United States

About this study

Official title: Kuvan® (Sapropterin Dihydrochloride) for Improving Gastric Accommodation in Women With Diabetic Gastroparesis (KIGA-DG)

Kuvan® (sapropterin dihydrochloride) for Improving Gastric Accommodation in Women with Diabetic Gastroparesis (KIGA-DG)

Read the 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).

Conditions: Gastroparesis
Being tested: sapropterin dihydrochloride
Medical subject headings: Gastroparesis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

sapropterin dihydrochlorideDrug
Also called KUVAN
sapropterin dihydrochloride: 10mg/kg/day

Study groups in NCT01135186
Study group	Role	What they receive
Sapropterin	Other	open label study of sapropterin dihydrochloride Receives: Drug: sapropterin dihydrochloride

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline, 4 Weeks, 8 Weeks

Secondary outcomes (3)

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.
Measured: 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.
Measured: Baseline, 4 Weeks, 8 Weeks
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.
Measured: Baseline, 4 Weeks, 8 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

May 2010Nov 2012

First posted2 Jun 2010 (estimated)
Study startMay 2010
Primary completionNov 2012
Study completionNov 2012

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (6)Papers the sponsor has linked to this study.
Other trials for GastroparesisStudies of the same condition, open and under way.
More trials run by Stanford UniversityThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase N/A
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 9 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01135186
Sponsor's protocol ID: SU-03252010-5462
Status last verified: Sep 2019
First submitted: 28 May 2010
Last updated: 10 Sep 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Soykan I, Sivri B, Sarosiek I, Kiernan B, McCallum RW. Demography, clinical characteristics, psychological and abuse profiles, treatment, and long-term follow-up of patients with gastroparesis. Dig Dis Sci. 1998 Nov;43(11):2398-404. doi: 10.1023/a:1026665728213.
PubMed 9824125
Bell RA, Jones-Vessey K, Summerson JH. Hospitalizations and outcomes for diabetic gastroparesis in North Carolina. South Med J. 2002 Nov;95(11):1297-9.
PubMed 12539997
Stacher G. Diabetes mellitus and the stomach. Diabetologia. 2001 Sep;44(9):1080-93. doi: 10.1007/s001250100619.
PubMed 11596661
Gangula PR, Maner WL, Micci MA, Garfield RE, Pasricha PJ. Diabetes induces sex-dependent changes in neuronal nitric oxide synthase dimerization and function in the rat gastric antrum. Am J Physiol Gastrointest Liver Physiol. 2007 Mar;292(3):G725-33. doi: 10.1152/ajpgi.00406.2006.
PubMed 17347455
Werner ER, Gorren AC, Heller R, Werner-Felmayer G, Mayer B. Tetrahydrobiopterin and nitric oxide: mechanistic and pharmacological aspects. Exp Biol Med (Maywood). 2003 Dec;228(11):1291-302. doi: 10.1177/153537020322801108.
PubMed 14681545
Smirnova VI, Gridchik IE, Tregubenko AD, Khachaturova EA. [Analgesia and intensive therapy during hemicorporectomy]. Anesteziol Reanimatol. 1991 Sep-Oct;(5):46-8. Russian.
PubMed 1767961

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Not yet recruiting
Stomach-Partitioning Versus Conventional Gastrojejunostomy for Malignant Gastric Outlet Obstruction: A Multicenter Randomized Controlled Trial
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To Study the Efficacy and Safety of Medtronic 780G Insulin Pump in People With Gastroparesis and Type 1 Diabetes.
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All Gastroparesis trials

Study at a glance

From the registry record, last updated by the sponsor on 10 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Kuvan® (Sapropterin Dihydrochloride) for Improving Gastric Accommodation in Women With Diabetic Gastroparesis (KIGA-DG)
ClinicalTrials.gov ID: NCT01135186
Acronym / sponsor ID: SU-03252010-5462
Conditions: Gastroparesis
Study type: Interventional
Recruitment status: Completed
Enrolment: 9 participants (actual)
Lead sponsor: Stanford UniversityUniversity, hospital or other
Study start: May 2010
Primary completion: Nov 2012 (actual)
Study completion: Nov 2012 (actual)
First posted: 2 Jun 2010 (estimated)
Results: Posted 29 Mar 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Kuvan® (sapropterin dihydrochloride) for Improving Gastric Accommodation in Women with Diabetic Gastroparesis (KIGA-DG)

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Sapropterin	Other	Drug: sapropterin dihydrochlorideopen label study of sapropterin dihydrochloride

Who may qualify

Ages: 18 to 65 years
Sex: Female only
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

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:
Diagnosis of diabetes mellitus > 5 years requiring medical therapy
Female gender
Ages 18-65 years
Documentation of delayed gastric emptying on gastric emptying scintigraphy (within 2 years of enrollment)

Key exclusion criteria (22 listed)

Patients who satisfy any of the following exclusion criteria will be ineligible for enrollment in the study:
Diabetes diagnosed < 5 years prior to the study
Male gender
Normal gastric emptying
Gastroparesis from post-surgical etiologies

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
sapropterin dihydrochlorideAlso: KUVANsapropterin dihydrochloride: 10mg/kg/day	Drug	Sapropterin

Primary outcome

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.

3 secondary outcomes

Secondary Outcome Measures Will Include Change in Symptom Severity.
Time frame: Baseline, 4 Weeks, 8 Weeks
Secondary Outcome Measures Will Include Change in Quality of Life.
Time frame: Baseline, 4 Weeks, 8 Weeks
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

Results and safety

Results posted to the registry on 29 Mar 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Open Label Study	9	9

Primary outcome: Gastric Accommodation

Time frame: Baseline, 4 Weeks, 8 Weeks. Mean (Standard Deviation). Unit: ml

Measure	Open Label Study n = 9
Baseline	322.22 (137.18)
4 Weeks	402.78 (197.03)
8 Weeks	450.63 (301.50)

3 secondary outcomes are reported in the full results.

Adverse events

Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Open Label Study	1 of 9 (11.1%)	0 of 9 (0%)	Not reported

Limitations and caveats (from the sponsor): A larger sample size is required to confirm the efficacy of saproptetin in increasing gastric accommodation and decreasing gastroparesis symptoms. This would assist in optimal dosing and duration of therapy.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 9824125Soykan I, Sivri B, Sarosiek I, Kiernan B, McCallum RW. Demography, clinical characteristics, psychological and abuse profiles, treatment, and long-term follow-up of patients with gastroparesis. Dig Dis Sci. 1998 Nov;43(11):2398-404. doi: 10.1023/a:1026665728213.
Background: PubMed 12539997Bell RA, Jones-Vessey K, Summerson JH. Hospitalizations and outcomes for diabetic gastroparesis in North Carolina. South Med J. 2002 Nov;95(11):1297-9.
Background: PubMed 11596661Stacher G. Diabetes mellitus and the stomach. Diabetologia. 2001 Sep;44(9):1080-93. doi: 10.1007/s001250100619.
Background: PubMed 17347455Gangula PR, Maner WL, Micci MA, Garfield RE, Pasricha PJ. Diabetes induces sex-dependent changes in neuronal nitric oxide synthase dimerization and function in the rat gastric antrum. Am J Physiol Gastrointest Liver Physiol. 2007 Mar;292(3):G725-33. doi: 10.1152/ajpgi.00406.2006.
Background: PubMed 14681545Werner ER, Gorren AC, Heller R, Werner-Felmayer G, Mayer B. Tetrahydrobiopterin and nitric oxide: mechanistic and pharmacological aspects. Exp Biol Med (Maywood). 2003 Dec;228(11):1291-302. doi: 10.1177/153537020322801108.
Background: PubMed 1767961Smirnova VI, Gridchik IE, Tregubenko AD, Khachaturova EA. [Analgesia and intensive therapy during hemicorporectomy]. Anesteziol Reanimatol. 1991 Sep-Oct;(5):46-8. Russian.

Related trials for Gastroparesis

Recruiting
Gastric Focused Ultrasound Stimulation
Gastroparesis, Delayed Gastric Emptying
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Enrolling by invitationObservational study
Body Surface Gastric Mapping to Evaluate Patients With Upper Gastrointestinal Symptoms and Controls
Motility Disorder, Gastroparesis
Not yet recruiting
Stomach-Partitioning Versus Conventional Gastrojejunostomy for Malignant Gastric Outlet Obstruction: A Multicenter Randomized Controlled Trial
Gastric Cancer, Gastric Outlet Obstruction
Recruiting
To Study the Efficacy and Safety of Medtronic 780G Insulin Pump in People With Gastroparesis and Type 1 Diabetes.
Type 1 Diabetes, Gastroparesis Due to Diabetes Mellitus Type I

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Linda A Nguyen principal investigator

Stanford University

Pankaj Jay Pasricha principal investigator

Stanford University

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A study testing sapropterin dihydrochloride in people with Gastroparesis, run by Stanford University at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 10 Sep 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 29 Mar 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Gastric Accommodation (time frame: Baseline, 4 Weeks, 8 Weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01135186, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2019.