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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

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

Conditions
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Cerebral Palsy, GERD

Intervention / Treatment
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* Drug: Baclofen
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

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

What is the study measuring?
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Primary Outcome Measures

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

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| GERD control | 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 | Incidence of side effects and rate of discontinuation of the study | 3 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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GERD