DystoniaPhase 2NCT00122044
Childhood Hypertonia of Central Origin: A Trial of Anticholinergic Treatment Effects
A Phase 2 study testing trihexyphenidyl in people with Dystonia, run by University of Southern California at 7 sites in United States.
Trial summary
This study is an open-label trial of trihexyphenidyl in children with upper extremity dystonia due to cerebral palsy. It is hypothesized that trihexyphenidyl in doses up to 0.75mg/kg/day would be well-tolerated and show significant changes on the Melbourne scale of upper extremity function. Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Dystonia
- What is being tested?
- trihexyphenidyl
- How many people
- 35 participants
- Where
- 7 sitesUnited States
- Study dates
- Jan 2003to Dec 2004
- Phase
- Phase 2
- 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 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Dystonia in the dominant upper extremity
Who cannot join
- Complete absence of voluntary movement in the affected hands, wrists, and elbows
- Severe weakness in the dominant upper extremity (MRC grade < 4)
- Passive range of motion at the hand, wrist or elbow less than 80% of normal
- Current use of medications for dystonia (anticholinergics, L-dopa, baclofen, diazepam, tizanidine, tetrabenazine, reserpine, and others)
- Changes in the subject's physical therapy regimen for the duration of the 15-week study
- Prior use of trihexyphenidyl or other anticholinergic therapy for dystonia.
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- History of surgery on the dominant upper extremity or cervical spine
- Botulinum toxin injection in the dominant upper extremity within the previous 6 months
- Current or prior implantation of an intrathecal baclofen pump, deep brain stimulator, or other device to treat dystonia or spasticity
- Concurrent acute or chronic medical condition (such as frequent seizures, heart disease, or asthma) that could adversely affect motor performance or the safety of testing
- Presence of diurnal fluctuations or other clinical signs and symptoms suggesting an inborn error of metabolism, a family history of dystonia suggesting a genetic dystonia, or dystonia due to injury after the neonatal period (including toxin exposure, trauma, or medication-induced)
- History of allergic or adverse reaction to trihexyphenidyl or other anticholinergic medications
- Current complaint of urinary retention requiring treatment.
- History of glaucoma, or family history of glaucoma with onset before age 40
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21205
University of Alabama School of Medicine
Birmingham, Alabama, 35233
Rehabilitation Institute of Chicago
Chicago, Illinois, 60611
Texas Scottish Rite Hospital for Children
Dallas, Texas, 75219
University of Rochester Medical Center
Rochester, New York, 14642
Washington University School of Medicine
St Louis, Missouri, 63110
Stanford University
Stanford, California, 94305-5235
About this study
Official title: Childhood Hypertonia of Central Origin: An Open Label Trial of Anticholinergic Treatment Effects
This study is an open-label trial of trihexyphenidyl in children with upper extremity dystonia due to cerebral palsy. It is hypothesized that trihexyphenidyl in doses up to 0.75mg/kg/day would be well-tolerated and show significant changes on the Melbourne scale of upper extremity function.
Read the detailed description
BACKGROUND: Although trihexyphenidyl has been used to treat both primary and secondary dystonia in children, previous studies have not investigated efficacy in secondary dystonia. We describe the results of a prospective, open-label, multi-center trial of high-dose trihexyphenidyl in children with secondary dystonia of the arms due to cerebral palsy.
METHODS: Twenty-six children age 4-15 years with cerebral palsy and dystonia that impairs function of the dominant upper extremity were enrolled. All children were given trihexyphenidyl at increasing doses over 9 weeks up to 0.75mg/kg/day. Trihexyphenidyl was subsequently tapered over 5 weeks. Visits occurred at baseline, 9 weeks, and 15 weeks. The primary outcome measure was the Melbourne assessment of upper extremity function, tested in the dominant arm.
RESULTS: Three children withdrew due to non-serious adverse events (chorea, drug rash, hyperactivity). 3 children reduced dosage due to non-serious adverse events. The 23 children who completed the study showed a significant improvement in arm function at 15 weeks (p=0.045) but not at 9 weeks. Post-hoc analysis showed that a subgroup (N=10) with hyperkinetic dystonia worsened at 9 weeks (p=0.04) but subsequently returned to baseline following taper of the medicine.
CONCLUSIONS: Trihexyphenidyl appears to be safe and effective for treatment of arm dystonia in children with cerebral palsy. Children with hyperkinetic dystonia may worsen. A larger randomized prospective trial is needed to confirm these results.
- Conditions
- Dystonia
- Being tested
- trihexyphenidyl
- Keywords
- childhood, cerebral palsy, dystonia, secondary, trihexyphenidyl, pediatric dystonia
- Medical subject headings
- Dystonia, Cerebral Palsy, Neoplasm Metastasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
trihexyphenidylDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
Melbourne assessment of upper extremity function
Secondary outcomes (5)
Barry-Albright Dystonia Scale
Burke-Fahn-Marsden Dystonia Scale
Pediatric Outcomes Data Collection Instrument
Pediatric Quality of Life
Gross Motor Function Measure
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2005 (estimated)
- Study startJan 2003
- Primary completionDec 2004
- Study completionDec 2004
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DystoniaStudies of the same condition, open and under way.
- More trials run by University of Southern CaliforniaThe 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 35 participants
Sponsor and investigators
- Lead sponsor
- University of Southern California
- Sponsor type
- University, hospital or other
- Collaborators
- United Cerebral Palsy Foundation, Don and Linda Carter Foundation, Crowley Carter Foundation
- Responsible party
- Principal investigator, Terence Sanger, University of Southern California
- Investigators
- Terence D Sanger, MD, PhD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00122044
- Sponsor's protocol ID
- CHOCOLATE
- Status last verified
- May 2014
- First submitted
- 18 Jul 2005
- Last updated
- 23 May 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Childhood Hypertonia of Central Origin: An Open Label Trial of Anticholinergic Treatment Effects
- ClinicalTrials.gov ID
- NCT00122044
- Acronym / sponsor ID
- CHOCOLATE
- Conditions
- Dystonia
- Keywords
- childhood, cerebral palsy, dystonia, secondary, trihexyphenidyl, pediatric dystonia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants
- Lead sponsor
- University of Southern CaliforniaUniversity, hospital or other
- Collaborators
- United Cerebral Palsy Foundation, Don and Linda Carter Foundation, Crowley Carter Foundation
- Study start
- Jan 2003
- Primary completion
- Dec 2004 (actual)
- Study completion
- Dec 2004
- First posted
- 21 Jul 2005 (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.
This study is an open-label trial of trihexyphenidyl in children with upper extremity dystonia due to cerebral palsy. It is hypothesized that trihexyphenidyl in doses up to 0.75mg/kg/day would be well-tolerated and show significant changes on the Melbourne scale of upper extremity function.
Detailed description
BACKGROUND: Although trihexyphenidyl has been used to treat both primary and secondary dystonia in children, previous studies have not investigated efficacy in secondary dystonia. We describe the results of a prospective, open-label, multi-center trial of high-dose trihexyphenidyl in children with secondary dystonia of the arms due to cerebral palsy.
METHODS: Twenty-six children age 4-15 years with cerebral palsy and dystonia that impairs function of the dominant upper extremity were enrolled. All children were given trihexyphenidyl at increasing doses over 9 weeks up to 0.75mg/kg/day. Trihexyphenidyl was subsequently tapered over 5 weeks. Visits occurred at baseline, 9 weeks, and 15 weeks. The primary outcome measure was the Melbourne assessment of upper extremity function, tested in the dominant arm.
RESULTS: Three children withdrew due to non-serious adverse events (chorea, drug rash, hyperactivity). 3 children reduced dosage due to non-serious adverse events. The 23 children who completed the study showed a significant improvement in arm function at 15 weeks (p=0.045) but not at 9 weeks. Post-hoc analysis showed that a subgroup (N=10) with hyperkinetic dystonia worsened at 9 weeks (p=0.04) but subsequently returned to baseline following taper of the medicine.
CONCLUSIONS: Trihexyphenidyl appears to be safe and effective for treatment of arm dystonia in children with cerebral palsy. Children with hyperkinetic dystonia may worsen. A larger randomized prospective trial is needed to confirm these results.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Dystonia in the dominant upper extremity
Key exclusion criteria (14 listed)
- Complete absence of voluntary movement in the affected hands, wrists, and elbows
- Severe weakness in the dominant upper extremity (MRC grade < 4)
- Passive range of motion at the hand, wrist or elbow less than 80% of normal
- Current use of medications for dystonia (anticholinergics, L-dopa, baclofen, diazepam, tizanidine, tetrabenazine, reserpine, and others)
- Changes in the subject's physical therapy regimen for the duration of the 15-week study
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 |
|---|---|---|
| trihexyphenidyl | Drug | – |
Primary outcome
Melbourne assessment of upper extremity function
5 secondary outcomes
Barry-Albright Dystonia Scale
Burke-Fahn-Marsden Dystonia Scale
Pediatric Outcomes Data Collection Instrument
Pediatric Quality of Life
Gross Motor Function Measure
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2014.
- Information on childhood dystonia and other movement disordersLink provided by the sponsor.
Related trials for Dystonia
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Intrathecal Baclofen and Pediatric Dystonia
Dystonic Cerebral Palsy
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Focused Ultrasound Unilateral Pallidotomy for Medication-Refractory Limb Dystonia
Limb Dystonia, Idiopathic Dystonia
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Deep Brain Stimulation Surgery for Movement Disorders
Parkinson's Disease, Essential Tremor
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Deep Brain Stimulation Surgery for Focal Hand Dystonia
Dystonia, Focal Dystonia
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.
Terence D Sanger, MD, PhD principal investigator
Stanford University
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing trihexyphenidyl in people with Dystonia, run by University of Southern California at 7 sites 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 1 inclusion and 14 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: finished, as of the sponsor's last update on 23 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Melbourne assessment of upper extremity function. 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 23 May 2014.