Ampicillin for DYT-1 Dystonia Motor Symptoms

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by University of Florida
Sponsor:
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 20, 2014
Last verified: May 2014
  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: August 2014
Estimated Primary Completion Date: June 2014 (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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

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 20, 2014
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
Central Nervous System Diseases
Dyskinesias
Movement Disorders
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
Ampicillin
Anti-Bacterial Agents
Anti-Infective Agents
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on October 20, 2014