Baclofen Treatment of Ataxia Telangiectasia

Early Phase 1

Completed

• Conditions: Ataxia Telangiectasia
• Interventions: Drug: Baclofen; Drug: Placebo

• Registration Number: NCT00640003
• Lead Sponsor: Johns Hopkins University

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2007-04
• Study First Submitted: 2008-03-14
• Study First Submitted QC: 2008-03-19
• Study First Posted: 2008-03-20
• Primary Completion: 2011-02
• Study Completion: 2011-02
• Status Verified: 2017-12
• Last Update Submitted: 2017-12-18
• Last Update Posted: 2017-12-20

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
1	Placebo	-
2	Placebo	-
1	Baclofen	-
2	Baclofen	-

Primary Outcome Measures

Name	Time	Method
Improvement in the decay constant for velocity storage as assessed by quantitative video examination of eye movement during and after rotation.	3 months

Secondary Outcome Measures

Name	Time	Method
Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination.	3 months

Trial Locations

Locations (1)

Johns Hopkins Hospital; 🇺🇸 Baltimore, Maryland, United States