Cerebral Palsy (CP)Phase 1NCT01468350
Safety and Tolerability of Dalfampridine in Subjects With Cerebral Palsy
A Phase 1 study testing dalfampridine-ER 10mg in people with Cerebral Palsy (CP), run by Acorda Therapeutics at 11 sites in United States.
Trial summary
A double-blind, placebo-controlled, crossover study in subjects with cerebral palsy (CP) to evaluate the safety and tolerability and the effect of dalfampridine extended release (ER) tablets on sensorimotor function Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Cerebral Palsy (CP)
- What is being tested?
- dalfampridine-ER 10mg
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 35 participants
- Where
- 11 sitesUnited States
- Study dates
- Dec 2011to Mar 2013
- Phase
- Phase 1
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A diagnosis of CP
- No previous use of any dalfampridine formulation
- Ability to perform all the required study procedures. Subjects should be capable of fully extending and flexing both hands
Who cannot join
- Presence of any progressive neurological disease
- Severe CP defined as the requirement to use a wheelchair at all times and a care taker for constant assistance in daily activities. This definition includes spastic quadriplegia
- Pregnant or breastfeeding
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
Kennedy Krieger Institute at Johns Hopkins University
Baltimore, Maryland, 21205
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Rehabilitation Institute of Chicago
Chicago, Illinois, 60611
Cleveland Clinic
Cleveland, Ohio, 44195
University of Missouri at Columbia
Columbia, Missouri, 65212
Rancho Los Amigos National Rehabilitation Center
Downey, California, 90242
Detroit Clinical Research Center
Farmington Hills, Michigan, 48334
UCLA/Orthopaedic Hospital Center for Cerebral Palsy
Los Angeles, California, 90095
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
Rady Children's Hospital San Diego
San Diego, California, 92123
Swedish Medical Center
Seattle, Washington, 98122
About this study
Official title: A Double-Blind, Placebo-Controlled, Crossover Study in Subjects With Cerebral Palsy to Evaluate the Safety and Tolerability and the Effect on Sensorimotor Function of Dalfampridine-ER
A double-blind, placebo-controlled, crossover study in subjects with cerebral palsy (CP) to evaluate the safety and tolerability and the effect of dalfampridine extended release (ER) tablets on sensorimotor function
- Conditions
- Cerebral Palsy (CP)
- Being tested
- dalfampridine-ER 10mg, Placebo
- Medical subject headings
- Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dalfampridine-ER 10mgDrug
PlaceboOther
| Study group | Role | What they receive |
|---|---|---|
| (PART A) AB: dalfampridine-ER 10mg then placebo | Placebo comparator | Each subject randomized to the AB arm will receive a single witnessed dose of (A) dalfampridine-ER 10 mg, and a single witnessed dose of (B) placebo, two days apart Receives: Drug: dalfampridine-ER 10mg; Other: Placebo |
| (PART A) BA: placebo then dalfampridine-ER 10mg | Placebo comparator | Each subject randomized to the BA arm will receive a single witnessed dose of (B) placebo, and a single witnessed dose of (A) dalfampridine-ER 10 mg, two days apart Receives: Drug: dalfampridine-ER 10mg; Other: Placebo |
| (PART B) AB: dalfampridine-ER 10mg then placebo | Placebo comparator | Each subject randomized to the AB arm will receive multiple doses of (A) dalfampridine-ER 10mg and multiple doses of (B) placebo Receives: Drug: dalfampridine-ER 10mg; Other: Placebo |
| (PART B) BA: Placebo then dalfampridine-ER 10mg | Placebo comparator | Each subject randomized to the BA arm will receive multiple doses of (B) placebo, and multiple doses of (A) dalfampridine-ER 10mg Receives: Drug: dalfampridine-ER 10mg; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability of Dalfampridine-ER 10mg in Subjects With Cerebral Palsy (CP)
Safety and tolerability will be assessed primarily by monitoring Treatment Emergent Adverse Events (TEAEs)
TEAEs are defined as Adverse Events (AEs) with date of onset (or worsening) on or after the start-date of double-blind treatment and no more than 5 days after the last dose of double-blind treatment for Part A of the study and no more than 9 days for Part B of the study.
The severity categories of mild, moderate or severe, are defined below:
- Mild is defined as causing no limitation of usual activities
- Moderate is defined as causing some limitation of usual activities
- Severe is defined as causing inability to carry out usual activities
Measured: up to 31 days
Secondary outcomes (1)
Measure the Effects of Both Single and Multiple Doses of Dalfampridine-ER 10 mg on Sensorimotor Function
- Hand strength as measured by a composite Z-score derived from the grip test, and key, tip and palmar pinch tests
- Manual dexterity as measured by the Box and Block Test
- Walking speed as measured by the Timed 25 Foot Walk (T25FW)
- Gait as measured by gait analysis equipment (to be performed by sites that have the capability to perform it)
- For Part B only, subjective impressions of treatment as measured by:
- Subject Global Impression (SGI)
- Clinician Global Impression (CGI)
Measured: up to 31 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2011 (estimated)
- Study startDec 2011
- Primary completionJan 2013
- Study completionMar 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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cerebral Palsy (CP)Studies of the same condition, open and under way.
- 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
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Acorda Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Enrique Carrazana, MD, Study director, Acorda Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01468350
- Sponsor's protocol ID
- DALF-CP-1002
- Status last verified
- May 2014
- First submitted
- 7 Nov 2011
- Last updated
- 24 Jun 2014
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Bethoux F, Fatemi A, Fowler E, Marciniak C, Mayadev A, Waksman J, Zackowski K, Suarez G, Blight AR, Rabinowicz AL, Carrazana E. Safety, Tolerability, and Sensorimotor Effects of Extended-release Dalfampridine in Adults With Cerebral Palsy: A Pilot Study. Clin Ther. 2017 Feb;39(2):337-346. doi: 10.1016/j.clinthera.2016.12.015. Epub 2017 Jan 25.
PubMed 28131322
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Double-Blind, Placebo-Controlled, Crossover Study in Subjects With Cerebral Palsy to Evaluate the Safety and Tolerability and the Effect on Sensorimotor Function of Dalfampridine-ER
- ClinicalTrials.gov ID
- NCT01468350
- Acronym / sponsor ID
- DALF-CP-1002
- Conditions
- Cerebral Palsy (CP)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Acorda TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- Dec 2011
- Primary completion
- Jan 2013 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 9 Nov 2011 (estimated)
- Results
- Posted 24 Jun 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A double-blind, placebo-controlled, crossover study in subjects with cerebral palsy (CP) to evaluate the safety and tolerability and the effect of dalfampridine extended release (ER) tablets on sensorimotor function
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| (PART A) AB: dalfampridine-ER 10mg then placebo | Placebo comparator | Drug: dalfampridine-ER 10mg, Other: PlaceboEach subject randomized to the AB arm will receive a single witnessed dose of (A) dalfampridine-ER 10 mg, and a single witnessed dose of (B) placebo, two days apart |
| (PART A) BA: placebo then dalfampridine-ER 10mg | Placebo comparator | Drug: dalfampridine-ER 10mg, Other: PlaceboEach subject randomized to the BA arm will receive a single witnessed dose of (B) placebo, and a single witnessed dose of (A) dalfampridine-ER 10 mg, two days apart |
| (PART B) AB: dalfampridine-ER 10mg then placebo | Placebo comparator | Drug: dalfampridine-ER 10mg, Other: PlaceboEach subject randomized to the AB arm will receive multiple doses of (A) dalfampridine-ER 10mg and multiple doses of (B) placebo |
| (PART B) BA: Placebo then dalfampridine-ER 10mg | Placebo comparator | Drug: dalfampridine-ER 10mg, Other: PlaceboEach subject randomized to the BA arm will receive multiple doses of (B) placebo, and multiple doses of (A) dalfampridine-ER 10mg |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- A diagnosis of CP
- No previous use of any dalfampridine formulation
- Ability to perform all the required study procedures. Subjects should be capable of fully extending and flexing both hands
Key exclusion criteria (3 listed)
- Presence of any progressive neurological disease
- Severe CP defined as the requirement to use a wheelchair at all times and a care taker for constant assistance in daily activities. This definition includes spastic quadriplegia
- Pregnant or breastfeeding
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 |
|---|---|---|
| dalfampridine-ER 10mg | Drug | (PART A) AB: dalfampridine-ER 10mg then placebo, (PART A) BA: placebo then dalfampridine-ER 10mg, (PART B) AB: dalfampridine-ER 10mg then placebo, (PART B) BA: Placebo then dalfampridine-ER 10mg |
| Placebo | Other | (PART A) AB: dalfampridine-ER 10mg then placebo, (PART A) BA: placebo then dalfampridine-ER 10mg, (PART B) AB: dalfampridine-ER 10mg then placebo, (PART B) BA: Placebo then dalfampridine-ER 10mg |
Primary outcome
Safety and Tolerability of Dalfampridine-ER 10mg in Subjects With Cerebral Palsy (CP)
Time frame: up to 31 days
Safety and tolerability will be assessed primarily by monitoring Treatment Emergent Adverse Events (TEAEs) TEAEs are defined as Adverse Events (AEs) with date of onset (or worsening) on or after the start-date of double-blind treatment and no more than 5 days after the last dose of double-blind treatment for Part A of the study and no more than 9 days for Part B of the study. The severity categories of mild, moderate or severe, are defined below: * Mild is defined as causing no limitation of usual activities * Moderate is defined as causing some limitation of usual activities * Severe is defined as causing inability to carry out usual activities
1 secondary outcome
Measure the Effects of Both Single and Multiple Doses of Dalfampridine-ER 10 mg on Sensorimotor Function
Time frame: up to 31 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 28131322Bethoux F, Fatemi A, Fowler E, Marciniak C, Mayadev A, Waksman J, Zackowski K, Suarez G, Blight AR, Rabinowicz AL, Carrazana E. Safety, Tolerability, and Sensorimotor Effects of Extended-release Dalfampridine in Adults With Cerebral Palsy: A Pilot Study. Clin Ther. 2017 Feb;39(2):337-346. doi: 10.1016/j.clinthera.2016.12.015. Epub 2017 Jan 25.
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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.
Enrique Carrazana, MD study director
Acorda Therapeutics
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing dalfampridine-ER 10mg in people with Cerebral Palsy (CP), run by Acorda Therapeutics at 11 sites 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 3 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 24 Jun 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites 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?
Yes. Results are posted in the registry (first posted 24 Jun 2014). 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?
Safety and Tolerability of Dalfampridine-ER 10mg in Subjects With Cerebral Palsy (CP) (time frame: up to 31 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01468350, which also lists 1 linked publication.
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 24 Jun 2014.