DYT-1Phase 1NCT01433757
Ampicillin for DYT-1 Dystonia Motor Symptoms
A Phase 1 study testing Ampicillin and Sugar pill in people with DYT-1 and Dystonia, run by University of Florida at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 7 to 80 years
- What is being tested?
- Ampicillin and Sugar pill
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2011to Jun 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- DYT-1 dystonia, confirmed by genetic testing
- Between ages of 7 and 80 years
- BFM-DRS score greater than 6
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
UF Center for Movement Disorders and Neurorestoration
Gainesville, Florida, 32607
About this study
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
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.
Read the detailed 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.
- Being tested
- Ampicillin, Sugar pill
- Keywords
- dystonia, DYT-1, AMPICILLIN
- Medical subject headings
- Dystonia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AmpicillinDrug
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 pillDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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). Receives: 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. Receives: Drug: Sugar pill |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 70 days
Secondary outcomes (1)
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.
Measured: 70 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2011 (estimated)
- Study startSep 2011
- Primary completionJun 2017
- Study completionJun 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DYT-1Studies of the same condition, open and under way.
- More trials run by University of FloridaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Tyler's Hope for a Dystonia Cure, Inc
- Responsible party
- Sponsor
- Investigators
- Irene Malaty, M.D., Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01433757
- Sponsor's protocol ID
- 301-2011
- Status last verified
- Jul 2017
- First submitted
- 5 Aug 2011
- Last updated
- 5 Jul 2017
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01433757
- Acronym / sponsor ID
- 301-2011
- Keywords
- dystonia, DYT-1, AMPICILLIN
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Tyler's Hope for a Dystonia Cure, Inc
- Study start
- Sep 2011
- Primary completion
- Jun 2017 (actual)
- Study completion
- Jun 2017 (actual)
- First posted
- 14 Sep 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ampicillin | Active comparator | Drug: AmpicillinPatients who are randomly selected to receive the active drug will receive Ampicillin (2mg daily for adults and 1 mg daily for children). |
| Placebo | Placebo comparator | Drug: Sugar pillPatients who are randomly selected to receive the placebo will be given a sugar pill that resembles the active drug. |
Who may qualify
- Ages
- 7 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- DYT-1 dystonia, confirmed by genetic testing
- Between ages of 7 and 80 years
- BFM-DRS score greater than 6
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Ampicillin |
| 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. | Drug | Placebo |
Primary outcome
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.
1 secondary outcome
Burke-Fahn Marsden Dystonia Rating Scale (BFM-DRS)
Time frame: 70 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jul 2017.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Irene Malaty, M.D. principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ampicillin and Sugar pill in people with DYT-1 and Dystonia, run by University of Florida at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of Ampicillin in treating DYT-1 dystonia (time frame: 70 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jul 2017.