Baclofen Treatment of Ataxia Telangiectasia

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.

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Key information

Conditions

Ataxia Telangiectasia Ataxia Brain Diseases Cardiovascular Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities DNA Repair-Deficiency Disorders Dyskinesias Genetic Diseases, Inborn Immune System Diseases Immunologic Deficiency Syndromes Metabolic Diseases Nervous System Diseases Neurocutaneous Syndromes Neurologic Manifestations Nutritional and Metabolic Diseases Pathological Conditions, Signs and Symptoms Primary Immunodeficiency Diseases Signs and Symptoms Spinocerebellar Ataxias Telangiectasis Vascular Diseases

Age range

12 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Johns Hopkins Hospital, Baltimore, Maryland, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Baclofen

Drug

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

Placebo

Drug

placebo drug

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

Sponsors and collaborators

Lead sponsor

Johns Hopkins University

Other

Registry information

Important dates

Study start: 2007
Primary completion: 2011
Study completion: 2011
First posted: Mar 20, 2008
Registry last updated: Dec 20, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed