NCT00640003

Baclofen Treatment of Ataxia Telangiectasia

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Johns Hopkins University · Academic / Other
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Ataxia Telangiectasia

Interventions

Type	Name	Description
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.
DRUG	Placebo	placebo drug

Timeline

• Start date: 2007-04-01
• Primary completion: 2011-02-01
• Completion: 2011-02-01
• First posted: 2008-03-20
• Last updated: 2017-12-20

Locations

2 sites across 1 country: United States