Ampicillin for DYT-1 Dystonia Motor Symptoms
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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
|Official Title:||A Pilot, Randomized, Double-Blind, Placebo-Controlled Phase I Study to Determine the Safety and Efficacy of Ampicillin in Treating Primary Dystonia Symptoms|
- Safety and tolerability of Ampicillin in treating DYT-1 dystonia [ Time Frame: 70 days ] [ Designated as safety issue: Yes ]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.
- Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS) [ Time Frame: 70 days ] [ Designated as safety issue: No ]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.
|Study Start Date:||September 2011|
|Estimated Study Completion Date:||August 2014|
|Estimated Primary Completion Date:||June 2014 (Final data collection date for primary outcome measure)|
Active Comparator: Ampicillin
Patients who are randomly selected to receive the active drug will receive Ampicillin (2mg daily for adults and 1 mg daily for children).
Ampicillin will be given in capsule form. Dosage will be 2 mg daily for adults and 1 mg daily for children. One capsule will be taken each day for 28 consecutive days. After the first 28 days, there will be a 7 day 'washout' period during which no capsules are taken. Following the 'washout' period, another 28-day supply of Ampicillin will be given to the patient, to be taken in the same manner as described above.
Placebo Comparator: Placebo
Patients who are randomly selected to receive the placebo will be given a sugar pill that resembles the active drug.
Drug: Sugar pill
The sugar pill will be given in capsule form. Dosage will be 2 mg daily for adults and 1 mg daily for children. One capsule will be taken each day for 28 consecutive days. After the first 28 days, there will be a 7 day 'washout' period during which no capsules are taken. Following the 'washout' period, another 28-day supply of the sugar pill will be given to the patient, to be taken in the same manner as described above.
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01433757
|Contact: Kyle H Rizer, B.A.||firstname.lastname@example.org|
|Contact: Stacy Merritt, MAemail@example.com|
|United States, Florida|
|UF Center for Movement Disorders and Neurorestoration||Recruiting|
|Gainesville, Florida, United States, 32607|
|Contact: Kyle H Rizer, B.A. 352-294-5194 firstname.lastname@example.org|
|Contact: Stacy S Merritt, MA 352-273-5614 email@example.com|
|Principal Investigator: Ramon L. Rodriguez, M.D.|
|Principal Investigator:||Ramon L Rodriguez, M.D.||University of Florida|