Placebo Controlled Study of Baclofen for GERD in Children With Cerebral Palsy

August 26, 2017 updated by: Samuel Nurko, Boston Children's Hospital
Despite the use of proton pump inhibitors and available prokinetics, medical therapy is ineffective in an important number of children with cerbreal palsy (CP) and gastroesophageal reflux disease (GERD), and failure of medical therapy is associated with a substantial morbidity. Many patients, particularly children with CP and GERD, continue to experience complications despite aggressive therapy because antisecretory medications do not address the primary reflux mechanism (TLESR). Furthermore, in patients with CP, surgical options are fraught with serious complications and long-term morbidity. Because the available treatment options for children with CP and intractable GERD are limited, new therapies are urgently needed. Baclofen, which has been shown in animals and humans to decrease TLESRs, may be a good alternative for the treatment of children with CP with intractable GERD.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Detailed Description

Despite the use of proton pump inhibitors and available prokinetics, medical therapy is ineffective in an important number of children with cerbreal palsy (CP) and gastroesophageal reflux disease (GERD), and failure of medical therapy is associated with a substantial morbidity. Many patients, particularly children with CP and GERD, continue to experience complications despite aggressive therapy because antisecretory medications do not address the primary reflux mechanism (TLESR).

This is a placebo controlled trial of baclofen for the treatment of GERD in children with CP.

Study Type

Interventional

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Children's Hospital Boston

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • 3-18 years old
  • Diagnosis of Cerebral Palsy
  • Symptoms of GERD for at least 3 months
  • At least 8 weeks on BID therapy with a PPI at a therapeutic dose at time of enrollment
  • Normal upper gastrointestinal barium contrast study (UGI)
  • Have a g-tube that is used for more than 75% of calories and a stable feeding schedule for at least 2 weeks
  • If seizures are present, they need to be controlled and on stable medications for 4 weeks

Exclusion Criteria:

  • Underlying electrolyte disturbance
  • History of Nissen fundoplication
  • Renal insufficiency
  • Currently receiving baclofen
  • Baclofen allergy
  • Uncontrolled seizure disorder
  • Lack of informed consent

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: QUADRUPLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: baclofen
Baclofen suspension
Drug: Baclofen
The final baclofen dose will be 0.7 mg/kg (to a maximum of 40 mg/day) divided in three doses.
Other Names:
  • Lioresal
Placebo Comparator: placebo
Identical palcebo suspension
Drug: placebo
placebo. No other names

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Symptom control
Time Frame: 3 weeks
Symptom frequency during the 2 weeks of placebo and baclofen administration.
3 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
GERD control
Time Frame: 3 weeks

Frequency and duration of reflux episodes detected by MII during 24 hours of recording after two weeks of maximum dose.

Proportion of full-column and non-acid reflux episodes detected by MII during 24 hours after two weeks of maximum dose.

Frequency of TLESRs and reflux associated with TLESRs at half of the maximum dose, and after two weeks of maximum dose are administered
3 weeks
side effects of baclofen
Time Frame: 3 weeks
Incidence of side effects and rate of discontinuation of the study
3 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Boston Children's Hospital

Collaborators

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2009

Primary Completion (Actual)

April 1, 2012

Study Completion (Actual)

April 30, 2012

Study Registration Dates

First Submitted

June 29, 2011

First Submitted That Met QC Criteria

June 29, 2011

First Posted (Estimate)

July 1, 2011

Study Record Updates

Last Update Posted (Actual)

August 29, 2017

Last Update Submitted That Met QC Criteria

August 26, 2017

Last Verified

August 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Baclofen-07-02-0077
  • 1R21DK077678-01 (U.S. NIH Grant/Contract)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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