Topiramate for the Prophylactic Treatment of Cyclic Vomiting Syndrome in Children

• ClinicalTrials.gov Identifier: NCT00286988
• Recruitment Status: Terminated
• First Posted: February 6, 2006
• Last Update Posted: January 6, 2011
• Sponsor: Monarch Medical Research
• Collaborator: Ortho-McNeil Neurologics, Inc.
• Information provided by: Monarch Medical Research

Tracking Information

• First Submitted Date: February 1, 2006
• First Posted Date: February 6, 2006
• Last Update Posted Date: January 6, 2011
• Study Start Date: March 2006
• Actual Primary Completion Date: May 2007 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: February 1, 2006): Reduction of cycle frequency as measured by number of days between cycles.
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: June 17, 2010)

• Decreased duration of attacks (measured in hours)
• Decreased intensity of attacks (0-4 point scale; none, mild, moderate, severe, excruciating)
• Decreased associated symptoms (0-4 point scales; none, mild, moderate, severe, excruciating)
• Nausea
• Vomiting
• Headache
• Abdominal pain
• Decreased disability
• PedMidas
• Missed school (days per attack)
• Missed work (parent(s)) (days per attack)
• Decreased use of acute therapies (e.g. anti-emetics)
• Decreased emergency department visits.

Original Secondary Outcome Measures (submitted: February 1, 2006)

• 1.Decreased duration of attacks (measured in hours)
• 2.Decreased intensity of attacks (0-4 point scale; none, mild, moderate, severe, excruciating)
• 3.Decreased associated symptoms (0-4 point scales; none, mild, moderate, severe, excruciating)
• a. Nausea
• b. Vomiting
• c. Headache
• d. Abdominal pain
• 4.Decreased disability
• a. PedMidas
• b. Missed school (days per attack)
• c. Missed work (parent(s)) (days per attack)
• 5.Decreased use of acute therapies (e.g. anti-emetics)
• 6.Decreased emergency department visits.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Topiramate for the Prophylactic Treatment of Cyclic Vomiting Syndrome in Children
• Official Title: Topiramate for the Prophylactic Treatment of Cyclic Vomiting Syndrome in Children
• Brief Summary: The purpose of this study is to assess the effectiveness, tolerability and safety of oral topiramate for the preventative management of Cyclic Vomiting Syndrome. It is believed that topiramate will decrease the frequency, duration and severity of attacks experienced by children and adolescents with Cyclic Vomiting Syndrome.

Detailed Description

There is very little controlled data on the preventative treatment for Cyclic Vomiting Syndrome. The existing evidence consists of small, retrospective clinical series that evaluate symptomatic responses to five medications including cyproheptadine, propranolol, amitriptyline, phenobarbital and pizotifen (n >10). These published data consist of uncontrolled or retrospective reports. In addition, varying inclusion criteria and outcomes (i.e. obtained by family recall) were used in these studies limiting the basis upon which to compare relative effectiveness.

During the prospective baseline period, the subject will maintain cyclical vomiting records in which all headache occurrences will be recorded and characterized. (Cyclical vomiting records will be maintained throughout the study). The purpose of this study is to assess the effectiveness, tolerability and safety of oral topiramate for the preventative management of Cyclic Vomiting Syndrome using a prospective design, established diagnostic criteria (ICHD 2004), and defined, objective primary and secondary endpoints.

• Study Type: Interventional
• Study Phase: Phase 4
• Study Design: Allocation: Non-Randomized; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Prevention
• Condition: Vomiting
• Intervention: Drug: Topiramate
• Study Arms: Not Provided

Publications *

• CULLEN KJ, MA CDONALD WB. The periodic syndrome: its nature and prevalence. Med J Aust. 1963 Aug 3;50(2):167-73.
• Abu-Arafeh I, Russell G. Prevalence and clinical features of abdominal migraine compared with those of migraine headache. Arch Dis Child. 1995 May;72(5):413-7.

Recruitment Information

• Recruitment Status: Terminated
• Estimated Enrollment (submitted: February 1, 2006): 12
• Original Enrollment: Same as current
• Actual Study Completion Date: June 2007
• Actual Primary Completion Date: May 2007 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Meet the2004 ICHD Criteria for Cyclical Vomiting.

  • 1 vomiting attack lasting > 24 hours duration for 3 consecutive months
• 4-12 years of age.

• If female, subjects must:

  1. be premenarchal or otherwise incapable of pregnancy, or
  2. have practiced one of the following methods of contraception for at least one month prior to study entry: hormonal contraceptives, spermicide and barrier, intrauterine device, partner sterility, or
  3. be practicing abstinence and agree to continue abstinence or to use an acceptable method of contraception (as listed above) should sexual activity commence.
• Able to take oral medication in tablet form or sprinkle form
• Subjects or their guardians must be willing and able to: a) read and comprehend written instructions, b) complete the assessment forms, c) return for regular visits, and d) adhere to medication regimens.
• Subjects (or their legally acceptable representative) must have signed an informed consent document

Exclusion Criteria

• Have taken topiramate within 14 days prior to the start of the prospective baseline period.
• Have taken certain medications for cyclical vomiting prophylaxis
• Have progressive neurological disorder or a structural disorder of the brain from birth, trauma or past infection.
• Subjects who have taken any medications for migraine prophylaxis, within 2 weeks of the start of the prospective baseline period.
• Subjects starting non-pharmacologic prophylactic approaches (e.g., acupuncture, biofeedback, chiropractic methods) within 1 month prior to Visit 1.
• Require continuing treatment with anticonvulsant therapy for a non-migraine condition.
• Significant major psychiatric disorder (e.g., major depression) or subjects receiving anti-psychotic medication.
• History of attempted suicide or suicidal tendencies.
• History of substance abuse.
• Pregnant or lactating females.
• Have clinically unstable neurological, cardiovascular, gastrointestinal, musculoskeletal, pulmonary or other disease.
• Have any disease or condition that could compromise the function of those body systems that could result in altered absorption, excess accumulation, or impaired metabolism or excretion of the test medications (e.g., abnormal renal and/or hepatic function).
• Have active liver disease.
• Have received an investigational drug or used an investigational device within 30 days of study entry.
• Employees of the investigator, study center, or sponsor (i.e., principal investigator, sub-investigator(s), study coordinators, other study staff, employees, or contractors of each), with direct involvement in the proposed study or other studies under the direction of that investigator, study center or sponsor, as well as family members of the employees or the investigator.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 4 Years to 12 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT00286988
• Other Study ID Numbers: TOPMAT-EME-4001
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Donald W Lewis, MD, Children's Specialty Group
• Study Sponsor: Monarch Medical Research
• Collaborators: Ortho-McNeil Neurologics, Inc.

Investigators

• Principal Investigator: Donald W Lewis, MD; Monarch Medical Research

• PRS Account: Monarch Medical Research
• Verification Date: April 2007