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Baclofen Treatment of Ataxia Telangiectasia

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified December 2010 by Johns Hopkins University.
Recruitment status was:  Active, not recruiting
Sponsor:
ClinicalTrials.gov Identifier:
NCT00640003
First Posted: March 20, 2008
Last Update Posted: March 4, 2011
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
Johns Hopkins University
  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 Early Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment

Resource links provided by NLM:


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 ]

Secondary Outcome Measures:
  • Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination. [ Time Frame: 3 months ]

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

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00640003


Locations
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21108
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Sponsors and Collaborators
Johns Hopkins University
Investigators
Principal Investigator: Thomas O Crawford, M.D. Johns Hopkins School of Medicine
  More Information

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
First Submitted: March 14, 2008
First Posted: March 20, 2008
Last Update Posted: March 4, 2011
Last Verified: December 2010

Keywords provided by Johns Hopkins University:
AT
Ataxia
Telangiectasia

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Telangiectasis
Ataxia Telangiectasia
Spinocerebellar Ataxias
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Neurocutaneous Syndromes
Genetic Diseases, Inborn
DNA Repair-Deficiency Disorders
Metabolic Diseases
Immunologic Deficiency Syndromes
Immune System Diseases