Ampicillin for DYT-1 Dystonia Motor Symptoms

NCT01433757 | Completed Phase 1

• 1 other identifier: 301-2011
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

DYT-1; Dystonia

Keywords

dystonia; DYT-1; AMPICILLIN

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS)

  70 days

Study Arms (2)

Ampicillin

ACTIVE COMPARATOR

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

Placebo

PLACEBO COMPARATOR

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

Drug: Sugar pill

Interventions

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.

Ampicillin

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.

Placebo

Eligibility Criteria

• Age: 7 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• DYT-1 dystonia, confirmed by genetic testing
• Between ages of 7 and 80 years
• BFM-DRS score greater than 6

You may not qualify if:

• 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)

Sponsors & Collaborators

• University of Florida: lead
• Tyler's Hope for a Dystonia Cure, Inc: collaborator

Study Sites (1)

UF Center for Movement Disorders and Neurorestoration

Gainesville, Florida, 32607, United States

Location

MeSH Terms

Conditions

Dystonia

Interventions

Ampicillin; Sugars

Condition Hierarchy (Ancestors)

Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Penicillin G; Penicillins; beta-Lactams; Lactams; Amides; Organic Chemicals; Sulfur Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds; Carbohydrates

Study Officials

• Irene Malaty, M.D.

  University of Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 5, 2011
• First Posted: September 14, 2011
• Study Start: September 1, 2011
• Primary Completion: June 1, 2017
• Study Completion: June 1, 2017
• Last Updated: July 5, 2017

Record last verified: 2017-07

Locations

US (1)