Ampicillin for DYT-1 Dystonia Motor Symptoms

Phase 1

Completed

• Conditions: DYT-1; Dystonia
• Interventions: Drug: Ampicillin; Drug: Sugar pill

• Registration Number: NCT01433757
• Lead Sponsor: University of Florida

Brief Summary

The purpose of this study is to evaluate if the antibiotic Ampicillin is safe and tolerated in patients that have generalized dystonia caused by the DYT-1 gene mutation, as compared to patients treated with a placebo. A placebo is a pill that looks and tastes the same as the real drug, but without the active ingredient. The second objective of this study is to determine if dystonia symptoms improve while on the study drug.

Detailed Description

This is a double-blinded and randomized drug study: neither the patient nor the investigator know if patients are taking study drug (Ampicillin) or placebo.

Three study visits will include neurological exams, review of medical history, genetic test results and video-taped BFM-DRS motor scales. Patients will receive medication during baseline visit, consisting of either Ampicillin drug or placebo, which will be consumed twice daily for 28 days. Following a washout period (no drugs) of 7 days, patient will return to clinic for second study visit and receive second set of medication (placebo -vs- Ampicillin). Medication will be consumed twice daily for 28 days, with patient returning to clinic after a washout period of 7 days for final study visit in clinic.

Study Timeline

• Study First Submitted: 2011-08-05
• Study Start: 2011-09
• Study First Submitted QC: 2011-09-13
• Study First Posted: 2011-09-14
• Primary Completion: 2017-06
• Study Completion: 2017-06
• Status Verified: 2017-07
• Last Update Submitted: 2017-07-03
• Last Update Posted: 2017-07-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 2

Inclusion Criteria

• DYT-1 dystonia, confirmed by genetic testing
• Between ages of 7 and 80 years
• BFM-DRS score greater than 6

Exclusion Criteria

• Negative DYT-1 dystonia gene test
• Allergy to penicillins or cephalosporins
• Concurrent bacterial, viral or fungal infection at time of enrollment
• Pregnancy
• Inability to follow study protocol
• Lactose intolerance (placebo contains lactose powder)

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Ampicillin	Ampicillin	Patients who are randomly selected to receive the active drug will receive Ampicillin (2mg daily for adults and 1 mg daily for children).
Placebo	Sugar pill	Patients who are randomly selected to receive the placebo will be given a sugar pill that resembles the active drug.

Primary Outcome Measures

Name	Time	Method
Safety and tolerability of Ampicillin in treating DYT-1 dystonia	70 days	The primary objective of this study is to evaluate the safety and tolerability of treating DYT-1 dystonia with the use of ampicillin as compared to placebo.

Secondary Outcome Measures

Name	Time	Method
Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS)	70 days	We will determine if there is a reduction in Burke-Fahn Marsden Dystonia Rating Scale motor scores at the end of the study period and compare with the placebo treated group.

Trial Locations

Locations (1)

UF Center for Movement Disorders and Neurorestoration; 🇺🇸 Gainesville, Florida, United States