Back to resultsBaclofen Treatment of Ataxia Telangiectasia
Primary Purpose
Ataxia Telangiectasia
Status
Completed
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Baclofen
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Ataxia Telangiectasia focused on measuring AT, Ataxia, Telangiectasia
Eligibility Criteria
Inclusion Criteria:
- Teenagers over 12 years old and young adults with A-T may join if they have been evaluated previously in the A-T Clinical Center at Johns Hopkins Hospital and have a measurable abnormality of eye movement.
- Patients who are presently taking Baclofen will be eligible for the study if they are presently receiving the medication under the direction of Dr. Crawford in the ATCC, and are willing to withdraw from the medication for a period of one month prior to the initial screening visit.
- Female patients who are sexually active will be given a standard serum HCG pregnancy test.
- Those who are sexually active will be counseled about necessary precautions against pregnancy during the duration of the trial.
Exclusion Criteria:
- A positive pregnancy test.
Sites / Locations
- Johns Hopkins Hospital
- Johns Hopkins Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
1
2
Arm Description
Outcomes
Primary Outcome Measures
Improvement in the decay constant for velocity storage as assessed by quantitative video examination of eye movement during and after rotation.
Secondary Outcome Measures
Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination.
Full Information
NCT ID
NCT00640003
First Posted
March 14, 2008
Last Updated
December 18, 2017
Sponsor
Johns Hopkins University
1. Study Identification
Unique Protocol Identification Number
NCT00640003
Brief Title
Baclofen Treatment of Ataxia Telangiectasia
Study Type
Interventional
2. Study Status
Record Verification Date
December 2017
Overall Recruitment Status
Completed
Study Start Date
April 2007 (undefined)
Primary Completion Date
February 2011 (Actual)
Study Completion Date
February 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Johns Hopkins University
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This research is being done to find out if Baclofen, a medicine that is often used for the treatment of abnormal stiffness, might also be useful to treat some of the neurologic problems caused by ataxia telangiectasia (A-T). The investigators also want to find out if there are better ways to measure the problems of ataxia and abnormal eye movement for future studies of medication in ataxia telangiectasia.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Ataxia Telangiectasia
Keywords
AT, Ataxia, Telangiectasia
7. Study Design
Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
10 (Actual)
8. Arms, Groups, and Interventions
Arm Title
1
Arm Type
Experimental
Arm Title
2
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Baclofen
Other Intervention Name(s)
Butanoic Acid
Intervention Description
Drug given by orally via capsules. Escalation steps in 4-day intervals. Step 1: 5mg QAM. Step 2: 5mg BID. Step 3: 5mg TID. Step 4: 10mg QAM, 5mg BID. Step 5: 10mg BID, 5mg QMid-day. Step 6: 10mg TID. Step 6 will last up to 2 weeks. De-escalation will start from highest dose step and work backwards in 2-day intervals. Complete drug administration of escalating, staying constant at 10mg TID and de-escalating will be done with both drug and placebo.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
placebo drug
Primary Outcome Measure Information:
Title
Improvement in the decay constant for velocity storage as assessed by quantitative video examination of eye movement during and after rotation.
Time Frame
3 months
Secondary Outcome Measure Information:
Title
Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination.
Time Frame
3 months
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Teenagers over 12 years old and young adults with A-T may join if they have been evaluated previously in the A-T Clinical Center at Johns Hopkins Hospital and have a measurable abnormality of eye movement.
Patients who are presently taking Baclofen will be eligible for the study if they are presently receiving the medication under the direction of Dr. Crawford in the ATCC, and are willing to withdraw from the medication for a period of one month prior to the initial screening visit.
Female patients who are sexually active will be given a standard serum HCG pregnancy test.
Those who are sexually active will be counseled about necessary precautions against pregnancy during the duration of the trial.
Exclusion Criteria:
A positive pregnancy test.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Thomas O Crawford, M.D.
Organizational Affiliation
Johns Hopkins School of Medicine
Official's Role
Principal Investigator
Facility Information:
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21108
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Baclofen Treatment of Ataxia Telangiectasia
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