Cervical DystoniaPhase 3NCT01753310
Efficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.
A Phase 3 study testing Botulinum toxin type A in people with Cervical Dystonia, run by Ipsen at 42 sites in United States.
Trial summary
The purpose of the protocol is to evaluate the efficacy and safety of Dysport® using 2 mL dilution compared with placebo for the treatment of Cervical Dystonia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cervical Dystonia
- What is being tested?
- Botulinum toxin type A
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 134 participants
- Where
- 42 sitesUnited States
- Study dates
- Jan 2013to Jan 2015
- Phase
- Phase 3
- 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
- Primary diagnosis of Cervical Dystonia at least 9 months since onset and either previously untreated with botulinum toxin or currently treated with Botox at a total dosing range of 100-200 U and ≤60 U in the sternocleidomastoid muscle at the last injection cycle, and having had a satisfactory treatment response in the principal investigator's judgment during the last two sequential Botox treatment cycles.
- TWSTRS total score≥ 20; TWSTRS-severity subscale score> 10;
Who cannot join
- In apparent remission from Cervical Dystonia
- Diagnosis of pure retrocollis or pure anterocollis
- For non-naïve subjects, previous poor response to either of the last two Botox treatments
- Known requirement of <100U or >200U of Botox injected into the neck muscles
Trial locations and contacts
42 sites in 1 country.
42 sites in 1 country
United States42 sites
Emory University
Atlanta, Georgia, 30329
NeuroTrials Research Inc.
Atlanta, Georgia, 30342
University of Colorado at Denver Health Sciences
Aurora, Colorado, 80045
North Texas Movement Disorders Institute
Bedford, Texas, 76201
East Bay Physician's Group
Berkeley, California, 94705
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Parkinson's & Movement Disorders Center of Boca Raton
Boca Raton, Florida, 33486
Tufts Medical Center
Boston, Massachusetts, 02111
Rush University Medical Center
Chicago, Illinois, 60612
University of Cincinnati Physicians Company, LLC
Cincinnati, Ohio, 45267
Rehabilitation Consultants PA
Eagan, Minnesota, 55122
Associated Neurologists of Southern Connecticut
Fairfield, Connecticut, 06824
Advanced Neurosciences Research
Fort Collins, Colorado, 80528
Parkinson's and Movement Disorder Institute
Fountain Valley, California, 92708
University of Florida Center for Movement Disorders and Neurorestoration
Gainesville, Florida, 32607
Guilford Neurologic Associates; Cone Health Medical Group
Greensboro, North Carolina, 27405
Penn State Hershey Neurology
Hershey, Pennsylvania, 17033
Baylor College of Medicine
Houston, Texas, 77030
University of Texas Health Science Center at Houston
Houston, Texas, 77030
Kansas City Bone & Joint Clinic
Kansas City, Kansas, 66211
Kingston Neurological Associates
Kingston, New York, 12401
Loma Linda University Healthcare, Department of Neurology
Loma Linda, California, 92354
USC Keck School of Medicine
Los Angeles, California, 90033
Fazzini Parkinson's Disease & Dystonia Center
New York, New York, 10016
The Ichan School of Medicine at Mount Sinai
New York, New York, 10029
International Clinical Research Institute
Overland Park, Kansas, 66210
Emerald Coast Center for Neurological Disorders
Pensacola, Florida, 32514
Island Neurological Associates
Plainview, New York, 11803
PD Treatment Center of SW FL
Port Charlotte, Florida, 33980
Coastal Neurology
Port Royal, South Carolina, 29935
OHSU Center for Health and Healing
Portland, Oregon, 97239
UC Davis Medical Center
Sacramento, California, 95817
Movement Disorders Center of Arizona, LLC
Scottsdale, Arizona, 85258
University of Medicine and Dentistry of New Jersey
Stratford, New Jersey, 08084
Atlantic Neuroscience Institute
Summit, New Jersey, 07901
Puget Sound Neurology
Tacoma, Washington, 98409
University of South Florida
Tampa, Florida, 33606
University of Arizona
Tucson, Arizona, 85724
Georgetown University Hospital
Washington D.C., District of Columbia, 20007
Guilford Neurologic Associates
West Palm Beach, Florida, 33407
Premiere Research Institute at Palm Beach Neurology
West Palm Beach, Florida, 33407
Wake Forest School of Medicine
Winston-Salem, North Carolina, 27157
About this study
Official title: A Phase 3b, Multicentre, Randomised, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.
The purpose of the protocol is to evaluate the efficacy and safety of Dysport® using 2 mL dilution compared with placebo for the treatment of Cervical Dystonia.
- Conditions
- Cervical Dystonia
- Being tested
- Botulinum toxin type A, Placebo
- Medical subject headings
- Torticollis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Botulinum toxin type ABiological
Also called AbobotulinumtoxinA (Dysport®)
Intramuscular injection, between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only
PlaceboDrug
Up to 2mL
| Study group | Role | What they receive |
|---|---|---|
| Dysport® | Active comparator | Dysport® (intramuscular injection), between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only Receives: Biological: Botulinum toxin type A |
| Placebo | Placebo comparator | Placebo, up to 2mL Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4.
The change from baseline in the TWSTRS total score at Week 4 was determined for the subjects who received a single dose of Dysport® or placebo by intramuscular injection at the baseline visit (Day 1), and is expressed as weighted overall treatment difference. The TWSTRS is an assessment scale used to measure the impact of CD on subjects, and comprises 3 subscales: severity, disability and pain, each of which is scored independently. The total score from the 3 subscales gives the TWSTRS total score with a value from 0 to 85 (best to worst). The score was assessed by the investigator prior to study treatment at baseline and at all post-treatment visits.
Measured: 4 weeks post-treatment
Secondary outcomes (7)
Change From Baseline in TWSTRS Total Score at Week 2.
The change from baseline in the TWSTRS total score at Week 2 was determined for the subjects who received a single dose of Dysport® or placebo by intramuscular injection at the baseline visit (Day 1), and is expressed as weighted overall treatment difference. The TWSTRS is an assessment scale used to measure the impact of CD on subjects, and comprises 3 subscales: severity, disability and pain, each of which is scored independently. The total score from the 3 subscales gives the TWSTRS total score with a value from 0 to 85 (best to worst). The score was assessed by the investigator prior to study treatment at baseline and at all post-treatment visits.
Measured: 2 weeks post-treatment
Change From Baseline in Clinical Global Impression of Change (CGIC) in CD at Week 2.
The CGIC is an investigator-reported assessment of the global clinical change in CD since study treatment administration. The CGIC uses a seven-point Likert scale ranging from +3 (very much improved) to -3 (very much worse), and was assessed by the investigator at the Week 2 and Week 4 visits.
Measured: 2 weeks post-treatment
TWSTRS Responders at Week 2.
Treatment response was determined as the number of responders at Week 2 relative to the baseline TWSTRS total score. A treatment responder is defined as a subject who had at least a 30% reduction in the TWSTRS total score after treatment. This was calculated as ([Week 2 score - baseline score]/baseline score) * 100.
Measured: 2 weeks post-treatment
Change From Baseline in CGIC in CD at Week 4.
The CGIC is an investigator-reported assessment of the global clinical change in CD since study treatment administration. The CGIC uses a seven-point Likert scale ranging from +3 (very much improved) to -3 (very much worse), and was assessed by the investigator at the Week 2 and Week 4 visits.
Measured: 4 weeks post-treatment
TWSTRS Responders at Week 4.
Treatment response was determined as the number of responders at Week 4 relative to the baseline TWSTRS total score. A treatment responder is defined as a subject who had at least a 30% reduction in the TWSTRS total score after treatment. This was calculated as ([Week 4 score - baseline score]/baseline score) * 100.
Measured: 4 weeks post-treatment
Change From Baseline in Cervical Dystonia Impact Profile-58 (CDIP-58) Total Score at Week 4.
The CDIP-58 scale is a subject-based rating scale measuring the health impact of CD measured in 8 health dimensions including head and neck symptoms, pain and discomfort, upper limb activities, walking, sleep, annoyance, mood and psychosocial functioning. Subscale scores were transformed to a common theoretical range of 0 (no impact) to 100 (most impact). Negative changes from the baseline total score indicate improvement in the impact of CD on health whereas postive changes indicate worsening.
Measured: 4 weeks post-treatment
Change From Baseline in CDIP-58 Total Score at Week 2.
The CDIP-58 scale is a subject-based rating scale measuring the health impact of CD measured in 8 health dimensions including head and neck symptoms, pain and discomfort, upper limb activities, walking, sleep, annoyance, mood and psychosocial functioning. Subscale scores were transformed to a common theoretical range of 0 (no impact) to 100 (most impact). Negative changes from the baseline total score indicate improvement in the impact of CD on health whereas postive changes indicate worsening. The hierarchical testing procedure would only be conducted if the previous secondary efficacy endpoint (change from baseline in CDIP-58 total score at Week 4) reached a statistically significant treatment effect. This secondary efficacy endpoint (change from baseline in CDIP-58 total score at Week 2) was performed to characterise the full clinical effect.
Measured: 2 weeks post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2012 (estimated)
- Study startJan 2013
- Primary completionOct 2014
- Study completionJan 2015
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 Cervical DystoniaStudies of the same condition, open and under way.
- More trials run by IpsenThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 134 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Neurology, M.D., Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01753310
- Sponsor's protocol ID
- A-TL-52120-169
- Status last verified
- Jul 2019
- First submitted
- 17 Dec 2012
- Last updated
- 7 Aug 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Patel AT, Lew MF, Dashtipour K, Isaacson S, Hauser RA, Ondo W, Maisonobe P, Wietek S, Rubin B, Brashear A. Sustained functional benefits after a single set of injections with abobotulinumtoxinA using a 2-mL injection volume in adults with cervical dystonia: 12-week results from a randomized, double-blind, placebo-controlled phase 3b study. PLoS One. 2021 Feb 1;16(2):e0245827. doi: 10.1371/journal.pone.0245827. eCollection 2021.
PubMed 33524060
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3b, Multicentre, Randomised, Double-Blind, Placebo-Controlled Study Evaluating the Efficacy and Safety of DYSPORT® Using 2mL Dilution in Adults With Cervical Dystonia.
- ClinicalTrials.gov ID
- NCT01753310
- Acronym / sponsor ID
- A-TL-52120-169
- Conditions
- Cervical Dystonia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 134 participants (actual)
- Lead sponsor
- IpsenIndustry (pharma, biotech, devices)
- Study start
- Jan 2013
- Primary completion
- Oct 2014 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 20 Dec 2012 (estimated)
- Results
- Posted 16 Mar 2017
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 the protocol is to evaluate the efficacy and safety of Dysport® using 2 mL dilution compared with placebo for the treatment of Cervical Dystonia.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dysport® | Active comparator | Biological: Botulinum toxin type ADysport® (intramuscular injection), between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo, up to 2mL |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Primary diagnosis of Cervical Dystonia at least 9 months since onset and either previously untreated with botulinum toxin or currently treated with Botox at a total dosing range of 100-200 U and ≤60 U in the sternocleidomastoid muscle at the last injection cycle, and having had a satisfactory treatment response in the principal investigator's judgment during the last two sequential Botox treatment cycles.
- TWSTRS total score≥ 20; TWSTRS-severity subscale score> 10;
Key exclusion criteria (4 listed)
- In apparent remission from Cervical Dystonia
- Diagnosis of pure retrocollis or pure anterocollis
- For non-naïve subjects, previous poor response to either of the last two Botox treatments
- Known requirement of <100U or >200U of Botox injected into the neck muscles
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 |
|---|---|---|
| Botulinum toxin type AAlso: AbobotulinumtoxinA (Dysport®)Intramuscular injection, between 250 and 500 units (U)/vial using 2mL dilution, 1 cycle only | Biological | Dysport® |
| PlaceboUp to 2mL | Drug | Placebo |
Primary outcome
Change From Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4.
Time frame: 4 weeks post-treatment
The change from baseline in the TWSTRS total score at Week 4 was determined for the subjects who received a single dose of Dysport® or placebo by intramuscular injection at the baseline visit (Day 1), and is expressed as weighted overall treatment difference. The TWSTRS is an assessment scale used to measure the impact of CD on subjects, and comprises 3 subscales: severity, disability and pain, each of which is scored independently. The total score from the 3 subscales gives the TWSTRS total score with a value from 0 to 85 (best to worst). The score was assessed by the investigator prior to study treatment at baseline and at all post-treatment visits.
7 secondary outcomes
Change From Baseline in TWSTRS Total Score at Week 2.
Time frame: 2 weeks post-treatment
Change From Baseline in Clinical Global Impression of Change (CGIC) in CD at Week 2.
Time frame: 2 weeks post-treatment
TWSTRS Responders at Week 2.
Time frame: 2 weeks post-treatment
Change From Baseline in CGIC in CD at Week 4.
Time frame: 4 weeks post-treatment
TWSTRS Responders at Week 4.
Time frame: 4 weeks post-treatment
Change From Baseline in Cervical Dystonia Impact Profile-58 (CDIP-58) Total Score at Week 4.
Time frame: 4 weeks post-treatment
Change From Baseline in CDIP-58 Total Score at Week 2.
Time frame: 2 weeks post-treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33524060Patel AT, Lew MF, Dashtipour K, Isaacson S, Hauser RA, Ondo W, Maisonobe P, Wietek S, Rubin B, Brashear A. Sustained functional benefits after a single set of injections with abobotulinumtoxinA using a 2-mL injection volume in adults with cervical dystonia: 12-week results from a randomized, double-blind, placebo-controlled phase 3b study. PLoS One. 2021 Feb 1;16(2):e0245827. doi: 10.1371/journal.pone.0245827. eCollection 2021.
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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.
Medical Director, Neurology, M.D. study director
Ipsen
42 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Botulinum toxin type A in people with Cervical Dystonia, run by Ipsen at 42 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 2 inclusion and 4 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 7 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 42 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 16 Mar 2017). 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 From Baseline in Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) Total Score at Week 4. (time frame: 4 weeks post-treatment). 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 NCT01753310, 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 7 Aug 2019.