Baclofen Treatment of Ataxia Telangiectasia

Ataxia Telangiectasia Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00640003
• Other study ID #: NA_00002180
• Status: Completed
• Phase: Early Phase 1
• First received: March 14, 2008
• Last updated: December 18, 2017
• Start date: April 2007
• Est. completion date: February 2011

Study information

• Verified date: December 2017
• Source: Johns Hopkins University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: February 2011
• Est. primary completion date: February 2011
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Ataxia
• Ataxia Telangiectasia
• Cerebellar Ataxia
• Telangiectasis

Intervention

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.
• Placebo
placebo drug

Locations

Country	Name	City	State
United States	Johns Hopkins Hospital	Baltimore	Maryland
United States	Johns Hopkins Hospital	Baltimore	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Improvement in the decay constant for velocity storage as assessed by quantitative video examination of eye movement during and after rotation.		3 months
Secondary	Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination.		3 months