Ampicillin for DYT-1 Dystonia Motor Symptoms
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|ClinicalTrials.gov Identifier: NCT01433757|
Recruitment Status : Completed
First Posted : September 14, 2011
Last Update Posted : July 5, 2017
|Condition or disease||Intervention/treatment||Phase|
|DYT-1 Dystonia||Drug: Ampicillin Drug: Sugar pill||Phase 1|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||2 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Investigator)|
|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|
|Study Start Date :||September 2011|
|Primary Completion Date :||June 2017|
|Study Completion Date :||June 2017|
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.
- Safety and tolerability of Ampicillin in treating DYT-1 dystonia [ Time Frame: 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.
- Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS) [ Time Frame: 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01433757
|United States, Florida|
|UF Center for Movement Disorders and Neurorestoration|
|Gainesville, Florida, United States, 32607|
|Principal Investigator:||Irene Malaty, M.D.||University of Florida|