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DYT-1 | Ampicillin for DYT-1 Dystonia Motor Symptoms

DYT-1 research study

What is the primary objective of this study?

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.

Who is eligible to participate?

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)

Which medical condition, disease, disorder, syndrome, illness, or injury is researched?

DYT-1

Dystonia

Study Interventions

Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.

Drug:AmpicillinAmpicillin 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.

Drug:Sugar pillThe 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.

Study Arms

Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.

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

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

Study Status

Completed

Start Date: September 2011

Completed Date: June 2017

Phase: Phase 1

Type: Interventional

Design:

Primary Outcome: Safety and tolerability of Ampicillin in treating DYT-1 dystonia

Secondary Outcome: Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS)

Study sponsors, principal investigator, and references

Principal Investigator: Irene Malaty, M.D.

Lead Sponsor: University of Florida

Collaborator: Tyler's Hope for a Dystonia Cure, Inc

More information:https://clinicaltrials.gov/show/NCT01433757

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