Ampicillin for DYT-1 Dystonia Motor Symptoms
Information source: University of Florida
ClinicalTrials.gov processed this data on August 20, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: DYT-1; Dystonia
Intervention: Ampicillin (Drug); Sugar pill (Drug)
Phase: Phase 1
Status: Recruiting
Sponsored by: University of Florida Official(s) and/or principal investigator(s): Michael S Okun, M.D., Principal Investigator, Affiliation: University of Florida
Overall contact: Kyle H Rizer, B.A., Phone: 352-294-5194, Email: kyle.rizer@neurology.ufl.edu
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.
Clinical Details
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 design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Primary outcome: Safety and tolerability of Ampicillin in treating DYT-1 dystonia
Secondary outcome: Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS)
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.
Eligibility
Minimum age: 7 Years.
Maximum age: 80 Years.
Gender(s): Both.
Criteria:
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)
Locations and Contacts
Kyle H Rizer, B.A., Phone: 352-294-5194, Email: kyle.rizer@neurology.ufl.edu
UF Center for Movement Disorders and Neurorestoration, Gainesville, Florida 32607, United States; Recruiting Kyle H Rizer, B.A., Phone: 352-294-5194, Email: kyle.rizer@neurology.ufl.edu Julie Segura, B.A., Phone: 352-273-5566, Email: Julie.Segura@neurology.ufl.edu Michael S Okun, M.D., Principal Investigator
Additional Information
Starting date: September 2011
Last updated: June 2, 2015
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