Ampicillin for DYT-1 Dystonia Motor Symptoms

NCT ID: NCT01433757

Last Updated: 2017-07-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-09-30

Study Completion Date

2017-06-30

Brief Summary

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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.

Detailed Description

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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.

Conditions

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DYT-1 Dystonia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Ampicillin

Patients who are randomly selected to receive the active drug will receive Ampicillin (2mg daily for adults and 1 mg daily for children).

Group Type ACTIVE_COMPARATOR

Ampicillin

Intervention Type DRUG

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

Patients who are randomly selected to receive the placebo will be given a sugar pill that resembles the active drug.

Group Type PLACEBO_COMPARATOR

Sugar pill

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type 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.

Intervention Type DRUG

Eligibility Criteria

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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)
Minimum Eligible Age

7 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Tyler's Hope for a Dystonia Cure, Inc

UNKNOWN

Sponsor Role collaborator

University of Florida

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Irene Malaty, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Florida

Locations

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UF Center for Movement Disorders and Neurorestoration

Gainesville, Florida, United States

Site Status

Countries

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United States

Other Identifiers

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301-2011

Identifier Type: -

Identifier Source: org_study_id

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