Baclofen Treatment of Ataxia Telangiectasia
1 other identifier
interventional
10
1 country
2
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for early_phase_1
Started Apr 2007
Longer than P75 for early_phase_1
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2007
CompletedFirst Submitted
Initial submission to the registry
March 14, 2008
CompletedFirst Posted
Study publicly available on registry
March 20, 2008
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
February 1, 2011
CompletedDecember 20, 2017
December 1, 2017
3.8 years
March 14, 2008
December 18, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Improvement in the decay constant for velocity storage as assessed by quantitative video examination of eye movement during and after rotation.
3 months
Secondary Outcomes (1)
Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination.
3 months
Study Arms (2)
1
EXPERIMENTAL2
EXPERIMENTALInterventions
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.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- A positive pregnancy test.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (2)
Johns Hopkins Hospital
Baltimore, Maryland, 21108, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Thomas O Crawford, M.D.
Johns Hopkins School of Medicine
Study Design
- Study Type
- interventional
- Phase
- early phase 1
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 14, 2008
First Posted
March 20, 2008
Study Start
April 1, 2007
Primary Completion
February 1, 2011
Study Completion
February 1, 2011
Last Updated
December 20, 2017
Record last verified: 2017-12