Cerebral PalsyPhase 4NCT01386255
Placebo Controlled Study of Baclofen for GERD in Children With Cerebral Palsy
A Phase 4 study testing Baclofen in people with Cerebral Palsy and GERD, run by Boston Children's Hospital at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Cerebral Palsy, GERD
- What is being tested?
- Baclofen
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2009to 30 Apr 2012
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- Underlying electrolyte disturbance
- History of Nissen fundoplication
- Renal insufficiency
- Currently receiving baclofen
- Baclofen allergy
- Uncontrolled seizure disorder
- Lack of informed consent
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Boston
Boston, Massachusetts, 02115
About this study
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.
Read the 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.
- Conditions
- Cerebral Palsy, GERD
- Being tested
- Baclofen, placebo
- Keywords
- GERD
- Medical subject headings
- Cerebral Palsy, Gastroesophageal Reflux
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BaclofenDrug
Also called Lioresal
The final baclofen dose will be 0.7 mg/kg (to a maximum of 40 mg/day) divided in three doses.
placeboDrug
placebo. No other names
| Study group | Role | What they receive |
|---|---|---|
| baclofen | Active comparator | Baclofen suspension Receives: Drug: Baclofen |
| placebo | Placebo comparator | Identical palcebo suspension Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Symptom control
Symptom frequency during the 2 weeks of placebo and baclofen administration.
Measured: 3 weeks
Secondary outcomes (2)
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
Measured: 3 weeks
side effects of baclofen
Incidence of side effects and rate of discontinuation of the study
Measured: 3 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2011 (estimated)
- Study startApr 2009
- Primary completionApr 2012
- Study completion30 Apr 2012
Why it stopped: No participants were enrolled
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cerebral PalsyStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Samuel Nurko, Boston Children's Hospital
- Investigators
- Samuel Nurko, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01386255
- Sponsor's protocol ID
- Baclofen-07-02-0077
- Other IDs
- 1R21DK077678-01
- Status last verified
- Aug 2017
- First submitted
- 29 Jun 2011
- Last updated
- 29 Aug 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Placebo Controlled Study of Baclofen for GERD in Children With Cerebral Palsy
- ClinicalTrials.gov ID
- NCT01386255
- Acronym / sponsor ID
- Baclofen-07-02-0077
- Conditions
- Cerebral Palsy, GERD
- Keywords
- GERD
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No participants were enrolled
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
- Study start
- Apr 2009
- Primary completion
- Apr 2012 (actual)
- Study completion
- 30 Apr 2012 (actual)
- First posted
- 1 Jul 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| baclofen | Active comparator | Drug: BaclofenBaclofen suspension |
| placebo | Placebo comparator | Drug: placeboIdentical palcebo suspension |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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)
Key exclusion criteria (7 listed)
- Underlying electrolyte disturbance
- History of Nissen fundoplication
- Renal insufficiency
- Currently receiving baclofen
- Baclofen allergy
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 |
|---|---|---|
| BaclofenAlso: LioresalThe final baclofen dose will be 0.7 mg/kg (to a maximum of 40 mg/day) divided in three doses. | Drug | baclofen |
| placeboplacebo. No other names | Drug | placebo |
Primary outcome
Symptom control
Time frame: 3 weeks
Symptom frequency during the 2 weeks of placebo and baclofen administration.
2 secondary outcomes
GERD control
Time frame: 3 weeks
side effects of baclofen
Time frame: 3 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Aug 2017.
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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.
Samuel Nurko, MD principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Baclofen in people with Cerebral Palsy and GERD, run by Boston Children's Hospital 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 7 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: withdrawn, as of the sponsor's last update on 29 Aug 2017. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Symptom control (time frame: 3 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 29 Aug 2017.