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

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• Status: Withdrawn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: Cerebral Palsy; GERD
• Intervention / Treatment: Drug: Baclofen; Drug: placebo

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

Study Locations

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

Participation Criteria

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

How is the study designed?

Design Details

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

Number of Arms

2

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	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

Collaborators and Investigators

• Sponsor: Boston Children's Hospital
• Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Study record dates

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: GERD
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Damage, Chronic; Cerebral Palsy; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; GABA Agents; Neuromuscular Agents; Muscle Relaxants, Central; GABA Agonists; GABA-B Receptor Agonists; Baclofen
• Other Study ID Numbers: Baclofen-07-02-0077; 1R21DK077678-01 (U.S. NIH Grant/Contract)