Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Baclofen Treatment of Ataxia Telangiectasia

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.
 
ClinicalTrials.gov Identifier: NCT00640003
Recruitment Status : Completed
First Posted : March 20, 2008
Last Update Posted : December 20, 2017
Sponsor:
Information provided by (Responsible Party):
Johns Hopkins University

Tracking Information
First Submitted Date  ICMJE March 14, 2008
First Posted Date  ICMJE March 20, 2008
Last Update Posted Date December 20, 2017
Study Start Date  ICMJE April 2007
Actual Primary Completion Date February 2011   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 3, 2011)
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 ]
Original Primary Outcome Measures  ICMJE
 (submitted: March 19, 2008)
Improvement in the decay constant for velocity storage as assessed by quantitative video examination of eye movement during and after rotation. [ Time Frame: months ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 3, 2011)
Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination. [ Time Frame: 3 months ]
Original Secondary Outcome Measures  ICMJE
 (submitted: March 19, 2008)
Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination. [ Time Frame: months ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Baclofen Treatment of Ataxia Telangiectasia
Official Title  ICMJE Not Provided
Brief 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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Early Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Ataxia Telangiectasia
Intervention  ICMJE
  • 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
Study Arms  ICMJE
  • Experimental: 1
    Interventions:
    • Drug: Baclofen
    • Drug: Placebo
  • Experimental: 2
    Interventions:
    • Drug: Baclofen
    • Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: December 18, 2017)
10
Original Estimated Enrollment  ICMJE
 (submitted: March 19, 2008)
12
Actual Study Completion Date  ICMJE February 2011
Actual Primary Completion Date February 2011   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00640003
Other Study ID Numbers  ICMJE NA_00002180
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Johns Hopkins University
Study Sponsor  ICMJE Johns Hopkins University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Thomas O Crawford, M.D. Johns Hopkins School of Medicine
PRS Account Johns Hopkins University
Verification Date December 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP