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Completed

NCT Number: NCT01433757

Ampicillin for DYT-1 Dystonia Motor Symptoms

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.

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Key information

Conditions

DYT-1 Dyskinesias Dystonia Nervous System Diseases Neurologic Manifestations Pathological Conditions, Signs and Symptoms Signs and Symptoms

Age range

7 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

UF Center for Movement Disorders and Neurorestoration

Gainesville, Florida, 32607, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

Ampicillin

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.

Sugar pill

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.

Primary outcomes

  1. Safety and tolerability of Ampicillin in treating DYT-1 dystonia

    Time frame: 70 days

    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 outcomes

  1. Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS)

    Time frame: 70 days

    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.

Sponsors and collaborators

Lead sponsor

University of Florida

Other

Collaborators

  • Tyler's Hope for a Dystonia Cure, Inc

Registry information

Official study title

A Pilot, Randomized, Double-Blind, Placebo-Controlled Phase I Study to Determine the Safety and Efficacy of Ampicillin in Treating Primary Dystonia Symptoms

Important dates

Study start
2011
Primary completion
2017
Study completion
2017
First posted
Sep 14, 2011
Registry last updated
Jul 5, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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