Trial record 5 of 165 for:
dystonia
Ampicillin for DYT-1 Dystonia Motor Symptoms
This study is currently recruiting participants.
Verified May 2013 by University of Florida
Sponsor:
University of Florida
Collaborator:
Tyler's Hope for a Dystonia Cure, Inc
Information provided by (Responsible Party):
University of Florida
ClinicalTrials.gov Identifier:
NCT01433757
First received: August 5, 2011
Last updated: May 10, 2013
Last verified: May 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
DYT-1 Dystonia |
Drug: Ampicillin Drug: Sugar pill |
Phase 1 |
| Study Type: | Interventional |
| 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 |
Resource links provided by NLM:
Further study details as provided by University of Florida:
Primary Outcome Measures:
- 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.
Secondary Outcome Measures:
- 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.
| Estimated Enrollment: | 14 |
| Study Start Date: | September 2011 |
| Estimated Study Completion Date: | December 2013 |
| Estimated Primary Completion Date: | September 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
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).
|
Drug: Ampicillin
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.
|
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| Ages Eligible for Study: | 7 Years to 80 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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)
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01433757
Contacts
| Contact: Kyle H Rizer, B.A. | 352-294-5194 | kyle.rizer@neurology.ufl.edu |
| Contact: Stacy Merritt, MA | 352-273-5614 | stacy.merritt@neurology.ufl.edu |
Locations
| 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 kyle.rizer@neurology.ufl.edu | |
| Contact: Stacy S Merritt, MA 352-273-5614 stacy.merritt@neurology.ufl.edu | |
| Principal Investigator: Ramon L. Rodriguez, M.D. | |
Sponsors and Collaborators
University of Florida
Tyler's Hope for a Dystonia Cure, Inc
Investigators
| Principal Investigator: | Ramon L Rodriguez, M.D. | University of Florida |
More Information
No publications provided
| Responsible Party: | University of Florida |
| ClinicalTrials.gov Identifier: | NCT01433757 History of Changes |
| Other Study ID Numbers: | 301-2011 |
| Study First Received: | August 5, 2011 |
| Last Updated: | May 10, 2013 |
| Health Authority: | United States: Food and Drug Administration United States: Institutional Review Board |
Keywords provided by University of Florida:
|
dystonia DYT-1 AMPICILLIN |
Additional relevant MeSH terms:
|
Dystonia Dystonic Disorders Dyskinesias Neurologic Manifestations Nervous System Diseases Signs and Symptoms Movement Disorders |
Central Nervous System Diseases Ampicillin Anti-Bacterial Agents Anti-Infective Agents Therapeutic Uses Pharmacologic Actions |
ClinicalTrials.gov processed this record on June 18, 2013