Ampicillin for DYT-1 Dystonia Motor Symptoms

Dystonia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01433757
• Other study ID #: 301-2011
• Status: Completed
• Phase: Phase 1
• First received: August 5, 2011
• Last updated: July 3, 2017
• Start date: September 2011
• Est. completion date: June 2017

Study information

• Verified date: July 2017
• Source: University of Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 2
• Est. completion date: June 2017
• Est. primary completion date: June 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 7 Years to 80 Years

Eligibility

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)

Related Conditions & MeSH terms

• Dystonia
• Dystonic Disorders
• DYT-1

Intervention

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.
• 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.

Locations

Country	Name	City	State
United States	UF Center for Movement Disorders and Neurorestoration	Gainesville	Florida

Sponsors (2)

Lead Sponsor	Collaborator
University of Florida	Tyler's Hope for a Dystonia Cure, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability of Ampicillin in treating DYT-1 dystonia	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.	70 days
Secondary	Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS)	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.	70 days