Overview

This trial is active, not recruiting.

Condition ataxia telangiectasia
Treatments baclofen, placebo
Phase phase 0
Sponsor Johns Hopkins University
Start date April 2007
End date February 2011
Trial size 12 participants
Trial identifier NCT00640003, NA_00002180

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model crossover assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
Arm
(Experimental)
baclofen Butanoic Acid
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.
placebo
placebo drug
(Experimental)
baclofen Butanoic Acid
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.
placebo
placebo drug

Primary Outcomes

Measure
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

Measure
Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination.
time frame: 3 months

Eligibility Criteria

Male or female participants at least 12 years old.

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.

Additional Information

Principal investigator Thomas O Crawford, M.D.
Trial information was received from ClinicalTrials.gov and was last updated in March 2011.
Information provided to ClinicalTrials.gov by Johns Hopkins University.