Baclofen Treatment of Ataxia Telangiectasia

Early Phase 1CompletedNCT00640003

Johns Hopkins University10 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Ataxia Telangiectasia

Interventions

BaclofenDRUG

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.

PlaceboDRUG

placebo drug

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

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.

Locations (2)

Johns Hopkins Hospital

Baltimore, Maryland, United States

Johns Hopkins Hospital

Baltimore, Maryland, United States

Outcomes

Primary Outcomes

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 Outcomes

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

Time frame: 3 months

Data from ClinicalTrials.gov

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