NCT00607542

Brief Summary

Oral baclofen is used commonly to treat spasticity in children with cerebral palsy. Although for adults there is dosing,safety and efficacy information in the package insert, this is not the case for children. The purpose of this study is to determine how fast the drug is cleared from the body, the correct dose, and long-term safety and efficacy for children with spasticity.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
61

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Nov 2008

Typical duration for phase_1

Geographic Reach
1 country

11 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 22, 2008

Completed
14 days until next milestone

First Posted

Study publicly available on registry

February 5, 2008

Completed
9 months until next milestone

Study Start

First participant enrolled

November 1, 2008

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2011

Completed
Last Updated

December 6, 2011

Status Verified

February 1, 2011

Enrollment Period

2.2 years

First QC Date

January 22, 2008

Last Update Submit

December 2, 2011

Conditions

SpasticityCerebral Palsy

Keywords

spasticitycerebral palsybaclofenpharmacokineticspharmacodynamicsdosingsafetyefficacy

Outcome Measures

Primary Outcomes (3)

  • Determine pharmacokinetic parameters of oral baclofen in children with spasticity associated with cerebral palsy (CP).

    1 year

  • Describe the relationship between plasma concentrations of oral baclofen and clinical measures of spasticity.

    1 year

  • Determine optimal dosing range and interval for administration of oral baclofen for use in a randomized clinical trial of safety and efficacy.

    1 year

Secondary Outcomes (3)

  • Describe the relationship between plasma concentrations of oral baclofen and measures of strength, function, ease of care, pain/comfort and health related quality of life.

    1 year

  • Describe the safety and tolerability of oral baclofen in children with spasticity associated with CP.

    1 year

  • Investigate preliminarily whether oral baclofen improves dystonia

    1 year

Study Arms (1)

1

ACTIVE COMPARATOR

starting dose of baclofen 2.5 mg PO TID with dose escalation as tolerated

Drug: baclofen

Interventions

baclofenDRUG

2.5 mg oral baclofen tablets given three times a day; dose gradually escalated as specified in the protocol

Also known as: Lioresal
1

Eligibility Criteria

Age2 Years - 16 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Males and females aged 2-16 years, inclusive.
  • Triceps skinfold thickness between the 5th and 95th percentiles for age (Refer to Appendix 3).
  • Gross Motor Function Classification Scale (GMFCS) Level II - V (GMFCS classifies children by functional mobility with Level I indicating minimal motor disability and V indicating total body involvement and dependence on others for mobility (Palisano et al, 1997).
  • Ashworth score of 2 or higher in at least one arm and one leg (knee + elbow flexors and/or extensors).
  • Cerebral Palsy: Motor disability due to a static, non-progressive brain injury/ malformation occurring prenatally or any time prior to the age of 2 years.
  • No history of baclofen use within the past 4 months.
  • Female subject, is premenarchal, or is incapable of pregnancy because of a hysterectomy or tubal ligation; or female subject who is sexually active and capable of pregnancy, has been using an acceptable method of contraception (hormonal contraceptives, intrauterine device, spermicide and barrier) for at least one month prior to study entry and agrees to continue to use one of these for the duration of the study; or female subject who is sexually abstinent and capable of pregnancy, agrees to continued abstinence or to use an acceptable method of birth control (either intrauterine device or spermicide and barrier) should sexual activity commence.
  • Subject ≥10 years of age has negative urine tests at screening and baseline for alcohol, non-medically prescribed drugs of abuse, and no history of tobacco use.

You may not qualify if:

  • Hypersensitivity to baclofen.
  • Selective dorsal rhizotomy.
  • Active intrathecal baclofen pump within the past 6 months.
  • Use of botulinum toxin in past 4 months or use any time during the study.
  • Use of tone altering medications (e.g. baclofen, benzodiazepines, levodopa, trihexyphenidyl) for \>3 consecutive days duration within the past 4 months.
  • Start of any drug or product known to be a significant cytochrome P450 enzyme inducer or inhibitor within the past 30 days.
  • Orthopaedic surgery within the past year or any time during the study.
  • Abdominal surgery within the past six months or any time during the study.
  • Uncontrolled seizures (baseline seizure frequency \>1 per month or history of more than 2 prolonged seizures lasting longer than 5 minutes duration within the past year.
  • Severe behavior difficulties or psychiatric disturbance
  • Proven gastric dysmotility: known history of abnormal gastric emptying study and/or history of vomiting 3 or more times per week.
  • Severe Gastroesophageal Reflux Disease: known history of esophagitis (documented on abnormal endoscopy or biopsy).
  • Malnutrition: defined as triceps skin fold thickness less than 5th or greater than 95th percentile for age.
  • Renal or Liver disease: Elevated bilirubin, LFTs greater than twice the upper limit of normal, reduced BUN/Cr ratio (\<5), or abnormal creatinine clearance that is clinically significant as determined by the investigator.
  • Abnormal CBC: Anemia, polycythemia, neutropenia, leukocytosis, thrombocytopenia, or thrombocytosis clinically significant as determined by the investigator.
  • +9 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (11)

Rehabilitation Institute of Chicago

Chicago, Illinois, 60611, United States

Location

Children's Hospital of Lousiana

New Orleans, Louisiana, 70118, United States

Location

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

Gillette Children's Speciality Healthcare

Saint Paul, Minnesota, 55101, United States

Location

Children's Mercy Hospital and Clinics

Kansas City, Missouri, 64108, United States

Location

Washington Univeristy - St. Louis Children's hospital

St Louis, Missouri, 63110-1093, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13210, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Kluge Children's Rehabilitation Center - University of Virginia

Charlottesville, Virginia, 22903, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Related Publications (2)

  • McLaughlin MJ, He Y, Brunstrom-Hernandez J, Thio LL, Carleton BC, Ross CJD, Gaedigk A, Lewandowski A, Dai H, Jusko WJ, Leeder JS. Pharmacogenomic Variability of Oral Baclofen Clearance and Clinical Response in Children With Cerebral Palsy. PM R. 2018 Mar;10(3):235-243. doi: 10.1016/j.pmrj.2017.08.441. Epub 2017 Sep 1.

    PMID: 28867665DERIVED

  • He Y, Brunstrom-Hernandez JE, Thio LL, Lackey S, Gaebler-Spira D, Kuroda MM, Stashinko E, Hoon AH Jr, Vargus-Adams J, Stevenson RD, Lowenhaupt S, McLaughlin JF, Christensen A, Dosa NP, Butler M, Schwabe A, Lopez C, Roge D, Kennedy D, Tilton A, Krach LE, Lewandowski A, Dai H, Gaedigk A, Leeder JS, Jusko WJ. Population pharmacokinetics of oral baclofen in pediatric patients with cerebral palsy. J Pediatr. 2014 May;164(5):1181-1188.e8. doi: 10.1016/j.jpeds.2014.01.029. Epub 2014 Mar 5.

    PMID: 24607242DERIVED

MeSH Terms

Conditions

Muscle SpasticityCerebral Palsy

Interventions

Baclofen

Condition Hierarchy (Ancestors)

Muscular DiseasesMusculoskeletal DiseasesMuscle HypertoniaNeuromuscular ManifestationsNeurologic ManifestationsNervous System DiseasesSigns and SymptomsPathological Conditions, Signs and SymptomsBrain Damage, ChronicBrain DiseasesCentral Nervous System Diseases

Intervention Hierarchy (Ancestors)

gamma-Aminobutyric AcidAminobutyratesButyratesAcids, AcyclicCarboxylic AcidsOrganic Chemicals

Study Officials

  • Janice Brunstrom, MD

    Washington University of St. Louis

    PRINCIPAL INVESTIGATOR

  • Richard Stevenson, MD

    University of Virginia

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 22, 2008

First Posted

February 5, 2008

Study Start

November 1, 2008

Primary Completion

January 1, 2011

Study Completion

January 1, 2011

Last Updated

December 6, 2011

Record last verified: 2011-02

Locations

US(11)