Spinocerebellar Ataxias Type 1NCT01811706
Dalfampridine and Gait in Spinocerebellar Ataxias
A study testing Dalfampridine in people with Spinocerebellar Ataxias Type 1 and Spinocerebellar Ataxias Type 2, run by University of Florida at 1 site in United States.
Trial summary
Investigators expect there will be improvement in walking speed and steadiness after taking Dalfampridine, thereby improving activities of daily living and enhancing social and occupational functions for patients with spinocerebellar ataxia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Spinocerebellar Ataxias Type 1, Spinocerebellar Ataxias Type 2, Spinocerebellar Ataxias Type 3
- What is being tested?
- Dalfampridine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2013to Dec 2013
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Individuals at age 18 years or older.
- Individuals who can provide the informed consent
- Genetic confirmed definite spinocerebellar ataxias (SCA)
- Able to complete two trials of the timed 25-foot walk at screening
Who cannot join
- Patients who has severe ataxia and unable to ambulate.
- Any orthopedic condition that would affect motor performance.
- Patients with secondary ataxia from general medical disorders
- Individuals who have major psychiatric disorders that prevents compliance
- History of epilepsy
- Patients with active drug or alcohol use or dependence that would interfere with adherence to study requirements
- Inability or unwillingness of subject or legal guardian/representative to give written informed consent.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Therapeutic Effect of Dalfampridine on Gait Incoordination in Spinocerebellar Ataxias- A Randomized, Double-blinded, Placebo-controlled, Crossover Clinical Trial
Investigators expect there will be improvement in walking speed and steadiness after taking Dalfampridine, thereby improving activities of daily living and enhancing social and occupational functions for patients with spinocerebellar ataxia.
Read the detailed description
Twenty spinocerebellar ataxia patients will be randomized to receive either Dalfampridine or placebo over a total period of 10 weeks. After entering the study, patients will return every 2 weeks for evaluation. After four weeks, intervention will be stopped and patient will enter a 2-week wash out period where they do not take any drug. Then, patients will be given the opposite treatment (Dalfampridine or placebo) and this "crossover" study will be performed for another 4 weeks. Investigators expect there will be improvement in walking speed and steadiness after taking Dalfampridine, thereby improving activities of daily living and enhancing social and occupational functions.
- Conditions
- Spinocerebellar Ataxias Type 1, Spinocerebellar Ataxias Type 2, Spinocerebellar Ataxias Type 3, Spinocerebellar Ataxias Type 6
- Being tested
- Dalfampridine, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DalfampridineDrug
Also called Ampyra
Dalfampridine will be provided at an oral dose of 10mg every 12 hours, for 4 weeks period
PlaceboDrug
Also called Sugar Pill
Placebo will be administered orally every 12 hours, for a 4 week period.
| Study group | Role | What they receive |
|---|---|---|
| Dalfampridine and then placebo | Experimental | Participant first receive Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks. After a washout period of 2 weeks, they then receive Placebo tablet orally every 12 hours, for a 4 weeks period. Receives: Drug: Dalfampridine; Drug: Placebo |
| Placebo, Then Dalfampridine | Experimental | Participant first receive Placebo tablet orally every 12 hours, for a 4 weeks period. After a washout period of 2 weeks, they then receive Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks. Receives: Drug: Dalfampridine; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Timed 25 Feet Walking Test (T25FW)
The patient is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly as possible, but safely. The time, in seconds, is calculated from the initiation of the instruction to start and ends when the patient has reached the 25-foot mark. Baseline values are recorded twice. One was at the beginning of the intervention. The second was 2 weeks after washout period and before the second intervention.
Measured: Baseline and 4 weeks after Dalfampridine or placebo
Secondary outcomes (2)
Change in Scale of Assessment and Rating of Ataxia (SARA)
Scale for the assessment and rating of ataxia (SARA) is a clinical scale that is based on a semiquantitative assessment of cerebellar ataxia on an impairment level. SARA has 8 items that are related to gait, stance, sitting, speech, finger-chase test, nose-finger test, fast alternating movements and heel-shin test. SARA score ranges from 0 to 40, with higher scores indicating more severe disease.
Measured: Baseline and 4 weeks after Dalfampridine or placebo
Biomechanical Assessment of Gait (BAG)-Stride Length
Biomechanical Assessment of Gait is a sensitive, quantitative movement analysis system. Stride length was analyzed. Baseline values are recorded twice. One was at the beginning of the intervention. The second was 2 weeks after washout period and before the second intervention.
Measured: Baseline and 4 weeks after Dalfampridine or placebo
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2013 (estimated)
- Study startFeb 2013
- Primary completionDec 2013
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Spinocerebellar Ataxias Type 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 N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Acorda Therapeutics
- Responsible party
- Sponsor
- Investigators
- Guangbin Xia, MD, PhD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01811706
- Sponsor's protocol ID
- 20121107
- Other IDs
- 1133511
- Status last verified
- Jan 2015
- First submitted
- 6 Mar 2013
- Last updated
- 12 Jan 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Therapeutic Effect of Dalfampridine on Gait Incoordination in Spinocerebellar Ataxias- A Randomized, Double-blinded, Placebo-controlled, Crossover Clinical Trial
- ClinicalTrials.gov ID
- NCT01811706
- Acronym / sponsor ID
- 20121107
- Conditions
- Spinocerebellar Ataxias Type 1, Spinocerebellar Ataxias Type 2, Spinocerebellar Ataxias Type 3, Spinocerebellar Ataxias Type 6
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- Acorda Therapeutics
- Study start
- Feb 2013
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 14 Mar 2013 (estimated)
- Results
- Posted 12 Jan 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Investigators expect there will be improvement in walking speed and steadiness after taking Dalfampridine, thereby improving activities of daily living and enhancing social and occupational functions for patients with spinocerebellar ataxia.
Detailed description
Twenty spinocerebellar ataxia patients will be randomized to receive either Dalfampridine or placebo over a total period of 10 weeks. After entering the study, patients will return every 2 weeks for evaluation. After four weeks, intervention will be stopped and patient will enter a 2-week wash out period where they do not take any drug. Then, patients will be given the opposite treatment (Dalfampridine or placebo) and this "crossover" study will be performed for another 4 weeks. Investigators expect there will be improvement in walking speed and steadiness after taking Dalfampridine, thereby improving activities of daily living and enhancing social and occupational functions.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dalfampridine and then placebo | Experimental | Drug: Dalfampridine, Drug: PlaceboParticipant first receive Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks. After a washout period of 2 weeks, they then receive Placebo tablet orally every 12 hours, for a 4 weeks period. |
| Placebo, Then Dalfampridine | Experimental | Drug: Dalfampridine, Drug: PlaceboParticipant first receive Placebo tablet orally every 12 hours, for a 4 weeks period. After a washout period of 2 weeks, they then receive Dalfampridine at an oral dose of 10mg every 12 hours, for 4 weeks. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Individuals at age 18 years or older.
- Individuals who can provide the informed consent
- Genetic confirmed definite spinocerebellar ataxias (SCA)
- Able to complete two trials of the timed 25-foot walk at screening
Key exclusion criteria (7 listed)
- Patients who has severe ataxia and unable to ambulate.
- Any orthopedic condition that would affect motor performance.
- Patients with secondary ataxia from general medical disorders
- Individuals who have major psychiatric disorders that prevents compliance
- History of epilepsy
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 |
|---|---|---|
| DalfampridineAlso: AmpyraDalfampridine will be provided at an oral dose of 10mg every 12 hours, for 4 weeks period | Drug | Dalfampridine and then placebo, Placebo, Then Dalfampridine |
| PlaceboAlso: Sugar PillPlacebo will be administered orally every 12 hours, for a 4 week period. | Drug | Dalfampridine and then placebo, Placebo, Then Dalfampridine |
Primary outcome
Change in Timed 25 Feet Walking Test (T25FW)
Time frame: Baseline and 4 weeks after Dalfampridine or placebo
The patient is directed to one end of a clearly marked 25-foot course and is instructed to walk 25 feet as quickly as possible, but safely. The time, in seconds, is calculated from the initiation of the instruction to start and ends when the patient has reached the 25-foot mark. Baseline values are recorded twice. One was at the beginning of the intervention. The second was 2 weeks after washout period and before the second intervention.
2 secondary outcomes
Change in Scale of Assessment and Rating of Ataxia (SARA)
Time frame: Baseline and 4 weeks after Dalfampridine or placebo
Biomechanical Assessment of Gait (BAG)-Stride Length
Time frame: Baseline and 4 weeks after Dalfampridine or placebo
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jan 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Guangbin Xia, MD, PhD 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 study testing Dalfampridine in people with Spinocerebellar Ataxias Type 1 and Spinocerebellar Ataxias Type 2, 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 4 inclusion and 7 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 12 Jan 2015. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Jan 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change in Timed 25 Feet Walking Test (T25FW) (time frame: Baseline and 4 weeks after Dalfampridine or placebo). 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 12 Jan 2015.