Baclofen Treatment of Ataxia Telangiectasia - Full Text View

Sponsor:	Johns Hopkins University
Information provided by:	Johns Hopkins University
ClinicalTrials.gov Identifier:	NCT00640003

Purpose

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	Intervention	Phase
Ataxia Telangiectasia	Drug: Baclofen Drug: Placebo	Phase 0

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator) Primary Purpose: Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: ataxia-telangiectasia autosomal recessive cerebellar ataxia type 1 complement factor I deficiency Friedreich ataxia infantile-onset spinocerebellar ataxia Marinesco-Sjögren syndrome spinocerebellar ataxia type 1 spinocerebellar ataxia type 2 spinocerebellar ataxia type 3 spinocerebellar ataxia type 6 VLDLR-associated cerebellar hypoplasia

MedlinePlus related topics: Ataxia Telangiectasia

Drug Information available for: Baclofen

U.S. FDA Resources

Further study details as provided by Johns Hopkins University:

Primary Outcome Measures:

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination. [ Time Frame: 3 months ] [ Designated as safety issue: No ]

Estimated Enrollment:	12
Study Start Date:	April 2007
Estimated Study Completion Date:	February 2011
Estimated Primary Completion Date:	February 2011 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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 Name: 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 Name: Butanoic Acid Drug: Placebo placebo drug

Arms

Assigned Interventions

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 Name: 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 Name: Butanoic Acid

Drug: Placebo

placebo drug

Eligibility

Ages Eligible for Study:	12 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00640003

Locations

United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21108

Sponsors and Collaborators

Johns Hopkins University

Investigators

Principal Investigator:

Thomas O Crawford, M.D.

Johns Hopkins School of Medicine

More Information

No publications provided

Responsible Party:	Thomas Owen Crawford, M.D./Principal Investigator, Johns Hopkins School of Medicine
ClinicalTrials.gov Identifier:	NCT00640003 History of Changes
Other Study ID Numbers:	NA_00002180
Study First Received:	March 14, 2008
Last Updated:	March 3, 2011
Health Authority:	United States: Institutional Review Board

Keywords provided by Johns Hopkins University:

AT
Ataxia
Telangiectasia

Additional relevant MeSH terms:

Ataxia
Ataxia Telangiectasia
Telangiectasis
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Ataxias
Cerebellar Ataxia
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Neurocutaneous Syndromes
Vascular Diseases
Cardiovascular Diseases

Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases
Baclofen
GABA-B Receptor Agonists
GABA Agonists
GABA Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Muscle Relaxants, Central
Neuromuscular Agents

ClinicalTrials.gov processed this record on February 09, 2012