Baclofen Treatment of Ataxia Telangiectasia

Sponsor

Johns Hopkins University (Other)

Overall Status

Completed

CT.gov ID

NCT00640003

Collaborator

(none)

Enrollment

Locations

Arms

46.1

Duration (Months)

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Ataxia Telangiectasia	Drug: Baclofen Drug: Placebo	Early Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

10 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

Study Start Date :

Apr 1, 2007

Actual Primary Completion Date :

Feb 1, 2011

Actual Study Completion Date :

Feb 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 1	Drug: Baclofen 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. Other Names: Butanoic Acid Drug: Placebo placebo drug
Experimental: 2	Drug: Baclofen 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. Other Names: Butanoic Acid Drug: Placebo placebo drug

Arm

Intervention/Treatment

Experimental: 1

Drug: Baclofen

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.

Other Names:

Butanoic Acid

Drug: Placebo

placebo drug

Experimental: 2

Drug: Baclofen

Other Names:

Butanoic Acid

Drug: Placebo

placebo drug

Outcome Measures

Primary Outcome Measures

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

Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination. [3 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.