Dystonia, FocalRoyalNCT04560101
Motor and Non-motor Symptoms in Cervical Dystonia
An observational study of Dystonia, Focal, run by University of Roma La Sapienza at 1 site in Italy.
Trial summary
In this monocenter, observational, non-interventional, prospective, open label study investigators will enrol 43 CD patients from the outpatient Movement Disorders Clinic of the Department of Human Neurosciences, Sapienza University of Rome. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Dystonia, Focal
- What is being tested?
- botulinum toxin A
- Type of treatment
- Injection or infusion
- How many people
- 43 participants
- Where
- 1 siteItaly
- Study dates
- 1 Aug 2020to Jun 2021
- Phase
- Observational
- 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 18 to 80 years
- All sexes
- Adult, Older adult
Study population: Patients with diagnosis of Cervical dystonia
Who may be able to join
- Cervical dystonia patients (primary dystonia).
- BoNT-A naïve or pre-treated with any BoNT-A product.
- Signed informed consent prior to participation in the study. If previously treated with any BoNT-A, at least a 3-4 months interval between last injection and inclusion.
Who cannot join
- - All secondary causes of dystonia (e.g. parkinsonian syndromes, or exposition of dopamine receptor blocking drugs) will be excluded.
- Major psychiatric disorders.
- Sensitivity to BoNT-A or to its excipients
- Other contraindications as given in the local SmPC for BoNT-A
- Pregnant \& lactating women
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
Department of Human Neurosciences
Rome, 00185
Giovanni Fabbrini, MD, +393386369901, giovanni.fabbrini@uniroma1.it
About this study
Official title: Motor and Non-motor Symptoms in Patients With Cervical Dystonia and the Effect of Treatment With Botulinum Toxin A
In this monocenter, observational, non-interventional, prospective, open label study investigators will enrol 43 CD patients from the outpatient Movement Disorders Clinic of the Department of Human Neurosciences, Sapienza University of Rome.
As this is a non-interventional study, no diagnostic, therapeutic or experimental intervention is involved. Subjects will receive clinical assessments, medications and treatments solely as determined by their study physician. The BoNT-A injection will be performed in CD patients at baseline.
As this is an observational, non-interventional study, the injection protocol for BoNT-A treatment is upon physicians' decision. All CD patients will undergo up to three evaluations of motor and non-motor symptoms: before (baseline) and 1 month and 3 months after botulinum toxin treatment. Both evaluations will be carried out under the same conditions. Motor symptoms will be assessed in all CD using the Comprehensive Cervical Dystonia Rating scale (CCDRS) (Comella et al, 2015). Non-motor symptoms including psychiatric, psychological and sleep disorders will be investigated. Psychiatric symptoms will be assessed with CCDS, Hamilton Rating Scale for Anxiety (HAM-A) and the Hamilton Rating Scale for Depression (HAM-D); the psychological symptoms will be assessed with the demoralization scale (Kissane et al, 2004) and the Italian Perceived Disability Scale (Innamorati et al,2009). Sleep disorders will be investigated with the Pittisburg Sleep Quality Index (PSQI) (Buysse et al, 1989).
Read the detailed description
Cervical dystonia (CD) is a focal dystonia, characterized by sustained muscle contractions of the neck causing repetitive movements, or abnormal postures. The muscles' activation pattern may lead to neck rotation (torticollis), flexion (anterocollis), extension (retrocollis), or head tilt (laterocollis) or a combination of these. Patients with CD may also have head tremor.
In addition to motor symptoms, CD patients may have non-motor features including psychiatric disturbances, sleep difficulties, cognitive impairment and neck pain.
The first line of treatment for CD is botulinum toxin-A (BoNT-A) injection therapy administered approximately at every 12 weeks. Several studies showed that one month after BoNT-A treatment there is an improvement of motor symptoms in the majority of CD patients. Conversely, regarding the effect of BoNT-A on non-motor symptoms previous studies in CD focused only on the possible effect on depressive symptoms and on neck pain.
No study has so far investigated whether the treatment with BoNT-A may improve non-motor symptoms in CD, including psychological aspects, such as demoralization and perceived disability.
Main aim of this study is therefore to assess the effect of botulinum neurotoxin type A (BoNT-A) treatment on motor and non-motor symptoms, including psychiatric and psychological symptoms (anxiety, depression, demoralization, perceived disability) and sleep. To this aim CD patients will be re-tested at 1 month and at 3 months after the treatment with BoNT-A. Investigators will also assess a possible relationship between the improvements of motor symptoms with that of non-motor symptoms at 1 month and at 3 months after BoNT-A treatment and the frequency and severity of non-motor symptoms in a large population of CD patients.
To see whether non-motor symptoms in CD are the consequence of motor symptoms, investigators will compare the severity of non-motor symptoms with the severity of motor symptoms, as well as with the different clinical patterns of CD and with the presence or absence of head tremor.
- Conditions
- Dystonia, Focal
- Being tested
- botulinum toxin A
- Keywords
- depression, anxiety, sleep, cognitive
- Medical subject headings
- Dystonic Disorders, Depression, Anxiety Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
botulinum toxin ADrug
Intramuscolar injection of botulinum toxin in affected muscles
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in depression scores from baseline to month 1 and 3
Changes in the Hamilton Rating Scale for depression (HAM-D) before and after three months from treatment
Measured: From baseline to month 1 and 3 after treatment
Changes in anxiety scores from baseline to month 1 and 3
Changes in the Hamilton Rating Scale for anxiety (HAM-A) before and after three months from treatment
Measured: From baseline to month 1 and 3 after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2020
- Study start1 Aug 2020
- Primary completionFeb 2021 (estimated)
- Study completionJun 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Dystonia, FocalStudies of the same condition, open and under way.
- More trials run by University of Roma La SapienzaThe 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 43 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Roma La Sapienza
- Sponsor type
- University, hospital or other
- Collaborators
- Ipsen
- Responsible party
- Principal investigator, Giovanni Fabbrini, University of Roma La Sapienza
- Investigators
- Giovanni Fabbrini, MD, Principal investigator, Depatment Human Neurosciences Sapienza University of Rome
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04560101
- Sponsor's protocol ID
- 5491
- Acronym
- Royal
- Status last verified
- Sep 2020
- First submitted
- 9 Sep 2020
- Last updated
- 23 Sep 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Single center study
Publications
Fabbrini G, Berardelli I, Moretti G, Pasquini M, Colosimo C, Berardelli A. Nonmotor symptoms in adult-onset focal dystonia: psychiatric abnormalities. Mov Disord. 2011 Aug 1;26(9):1765-6. doi: 10.1002/mds.23668. Epub 2011 Mar 25. No abstract available.
PubMed 21442656Berardelli I, Ferrazzano G, Pasquini M, Biondi M, Berardelli A, Fabbrini G. Clinical course of psychiatric disorders in patients with cervical dystonia. Psychiatry Res. 2015 Sep 30;229(1-2):583-5. doi: 10.1016/j.psychres.2015.07.076. Epub 2015 Jul 29.
PubMed 26239770Conte A, Berardelli I, Ferrazzano G, Pasquini M, Berardelli A, Fabbrini G. Non-motor symptoms in patients with adult-onset focal dystonia: Sensory and psychiatric disturbances. Parkinsonism Relat Disord. 2016 Jan;22 Suppl 1:S111-4. doi: 10.1016/j.parkreldis.2015.09.001. Epub 2015 Sep 3.
PubMed 26360238Ferrazzano G, Berardelli I, Conte A, Suppa A, Fabbrini G, Berardelli A. Interoceptive sensitivity in patients with cervical dystonia. Parkinsonism Relat Disord. 2017 Nov;44:129-132. doi: 10.1016/j.parkreldis.2017.08.019. Epub 2017 Aug 16.
PubMed 28827008Ferrazzano G, Berardelli I, Conte A, Baione V, Concolato C, Belvisi D, Fabbrini G, Defazio G, Berardelli A. Motor and non-motor symptoms in blepharospasm: clinical and pathophysiological implications. J Neurol. 2019 Nov;266(11):2780-2785. doi: 10.1007/s00415-019-09484-w. Epub 2019 Jul 29.
PubMed 31359200Berardelli I, Ferrazzano G, Belvisi D, Pompili M, Fabbrini G. Psychiatric disorders in blepharospasm: A 10-year follow-up study. Psychiatry Res. 2020 Aug;290:113092. doi: 10.1016/j.psychres.2020.113092. Epub 2020 May 26. No abstract available.
PubMed 32505028Ferrazzano G, Berardelli I, Belvisi D, De Bartolo MI, Di Vita A, Conte A, Fabbrini G. Awareness of Dystonic Posture in Patients With Cervical Dystonia. Front Psychol. 2020 Jun 23;11:1434. doi: 10.3389/fpsyg.2020.01434. eCollection 2020.
PubMed 32655462Berardelli I, Ferrazzano G, Belvisi D, Baione V, Fabbrini G, Innamorati M, Berardelli A, Pompili M. Suicidal ideation, hopelessness, and affective temperament in patients with blepharospasm. Int J Psychiatry Clin Pract. 2021 Nov;25(4):344-349. doi: 10.1080/13651501.2020.1790613. Epub 2020 Jul 15.
PubMed 32669012
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Motor and Non-motor Symptoms in Patients With Cervical Dystonia and the Effect of Treatment With Botulinum Toxin A
- ClinicalTrials.gov ID
- NCT04560101
- Acronym / sponsor ID
- Royal / 5491
- Conditions
- Dystonia, Focal
- Keywords
- depression, anxiety, sleep, cognitive
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 43 participants (estimated)
- Lead sponsor
- University of Roma La SapienzaUniversity, hospital or other
- Collaborators
- Ipsen
- Study start
- 1 Aug 2020 (actual)
- Primary completion
- Feb 2021 (estimated)
- Study completion
- Jun 2021 (estimated)
- First posted
- 23 Sep 2020 (actual)
- 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.
In this monocenter, observational, non-interventional, prospective, open label study investigators will enrol 43 CD patients from the outpatient Movement Disorders Clinic of the Department of Human Neurosciences, Sapienza University of Rome.
As this is a non-interventional study, no diagnostic, therapeutic or experimental intervention is involved. Subjects will receive clinical assessments, medications and treatments solely as determined by their study physician. The BoNT-A injection will be performed in CD patients at baseline.
As this is an observational, non-interventional study, the injection protocol for BoNT-A treatment is upon physicians' decision. All CD patients will undergo up to three evaluations of motor and non-motor symptoms: before (baseline) and 1 month and 3 months after botulinum toxin treatment. Both evaluations will be carried out under the same conditions. Motor symptoms will be assessed in all CD using the Comprehensive Cervical Dystonia Rating scale (CCDRS) (Comella et al, 2015). Non-motor symptoms including psychiatric, psychological and sleep disorders will be investigated. Psychiatric symptoms will be assessed with CCDS, Hamilton Rating Scale for Anxiety (HAM-A) and the Hamilton Rating Scale for Depression (HAM-D); the psychological symptoms will be assessed with the demoralization scale (Kissane et al, 2004) and the Italian Perceived Disability Scale (Innamorati et al,2009). Sleep disorders will be investigated with the Pittisburg Sleep Quality Index (PSQI) (Buysse et al, 1989).
Detailed description
Cervical dystonia (CD) is a focal dystonia, characterized by sustained muscle contractions of the neck causing repetitive movements, or abnormal postures. The muscles' activation pattern may lead to neck rotation (torticollis), flexion (anterocollis), extension (retrocollis), or head tilt (laterocollis) or a combination of these. Patients with CD may also have head tremor.
In addition to motor symptoms, CD patients may have non-motor features including psychiatric disturbances, sleep difficulties, cognitive impairment and neck pain.
The first line of treatment for CD is botulinum toxin-A (BoNT-A) injection therapy administered approximately at every 12 weeks. Several studies showed that one month after BoNT-A treatment there is an improvement of motor symptoms in the majority of CD patients. Conversely, regarding the effect of BoNT-A on non-motor symptoms previous studies in CD focused only on the possible effect on depressive symptoms and on neck pain.
No study has so far investigated whether the treatment with BoNT-A may improve non-motor symptoms in CD, including psychological aspects, such as demoralization and perceived disability.
Main aim of this study is therefore to assess the effect of botulinum neurotoxin type A (BoNT-A) treatment on motor and non-motor symptoms, including psychiatric and psychological symptoms (anxiety, depression, demoralization, perceived disability) and sleep. To this aim CD patients will be re-tested at 1 month and at 3 months after the treatment with BoNT-A. Investigators will also assess a possible relationship between the improvements of motor symptoms with that of non-motor symptoms at 1 month and at 3 months after BoNT-A treatment and the frequency and severity of non-motor symptoms in a large population of CD patients.
To see whether non-motor symptoms in CD are the consequence of motor symptoms, investigators will compare the severity of non-motor symptoms with the severity of motor symptoms, as well as with the different clinical patterns of CD and with the presence or absence of head tremor.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Patients with diagnosis of Cervical dystonia
Key inclusion criteria (3 listed)
- Cervical dystonia patients (primary dystonia).
- BoNT-A naïve or pre-treated with any BoNT-A product.
- Signed informed consent prior to participation in the study. If previously treated with any BoNT-A, at least a 3-4 months interval between last injection and inclusion.
Key exclusion criteria (5 listed)
- - All secondary causes of dystonia (e.g. parkinsonian syndromes, or exposition of dopamine receptor blocking drugs) will be excluded.
- Major psychiatric disorders.
- Sensitivity to BoNT-A or to its excipients
- Other contraindications as given in the local SmPC for BoNT-A
- Pregnant \& lactating women
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 AIntramuscolar injection of botulinum toxin in affected muscles | Drug | – |
Primary outcome
Changes in depression scores from baseline to month 1 and 3
Time frame: From baseline to month 1 and 3 after treatment
Changes in the Hamilton Rating Scale for depression (HAM-D) before and after three months from treatment
Changes in anxiety scores from baseline to month 1 and 3
Time frame: From baseline to month 1 and 3 after treatment
Changes in the Hamilton Rating Scale for anxiety (HAM-A) before and after three months from treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2020.
- Results publication: PubMed 21442656Fabbrini G, Berardelli I, Moretti G, Pasquini M, Colosimo C, Berardelli A. Nonmotor symptoms in adult-onset focal dystonia: psychiatric abnormalities. Mov Disord. 2011 Aug 1;26(9):1765-6. doi: 10.1002/mds.23668. Epub 2011 Mar 25. No abstract available.
- Results publication: PubMed 26239770Berardelli I, Ferrazzano G, Pasquini M, Biondi M, Berardelli A, Fabbrini G. Clinical course of psychiatric disorders in patients with cervical dystonia. Psychiatry Res. 2015 Sep 30;229(1-2):583-5. doi: 10.1016/j.psychres.2015.07.076. Epub 2015 Jul 29.
- Results publication: PubMed 26360238Conte A, Berardelli I, Ferrazzano G, Pasquini M, Berardelli A, Fabbrini G. Non-motor symptoms in patients with adult-onset focal dystonia: Sensory and psychiatric disturbances. Parkinsonism Relat Disord. 2016 Jan;22 Suppl 1:S111-4. doi: 10.1016/j.parkreldis.2015.09.001. Epub 2015 Sep 3.
- Results publication: PubMed 28827008Ferrazzano G, Berardelli I, Conte A, Suppa A, Fabbrini G, Berardelli A. Interoceptive sensitivity in patients with cervical dystonia. Parkinsonism Relat Disord. 2017 Nov;44:129-132. doi: 10.1016/j.parkreldis.2017.08.019. Epub 2017 Aug 16.
- Results publication: PubMed 31359200Ferrazzano G, Berardelli I, Conte A, Baione V, Concolato C, Belvisi D, Fabbrini G, Defazio G, Berardelli A. Motor and non-motor symptoms in blepharospasm: clinical and pathophysiological implications. J Neurol. 2019 Nov;266(11):2780-2785. doi: 10.1007/s00415-019-09484-w. Epub 2019 Jul 29.
- Results publication: PubMed 32505028Berardelli I, Ferrazzano G, Belvisi D, Pompili M, Fabbrini G. Psychiatric disorders in blepharospasm: A 10-year follow-up study. Psychiatry Res. 2020 Aug;290:113092. doi: 10.1016/j.psychres.2020.113092. Epub 2020 May 26. No abstract available.
- Results publication: PubMed 32655462Ferrazzano G, Berardelli I, Belvisi D, De Bartolo MI, Di Vita A, Conte A, Fabbrini G. Awareness of Dystonic Posture in Patients With Cervical Dystonia. Front Psychol. 2020 Jun 23;11:1434. doi: 10.3389/fpsyg.2020.01434. eCollection 2020.
- Results publication: PubMed 32669012Berardelli I, Ferrazzano G, Belvisi D, Baione V, Fabbrini G, Innamorati M, Berardelli A, Pompili M. Suicidal ideation, hopelessness, and affective temperament in patients with blepharospasm. Int J Psychiatry Clin Pract. 2021 Nov;25(4):344-349. doi: 10.1080/13651501.2020.1790613. Epub 2020 Jul 15.
- Individual participant dataSharing planned: No. Single center study
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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.
Giovanni Fabbrini, MD principal investigator
Depatment Human Neurosciences Sapienza University of Rome
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Dystonia, Focal, run by University of Roma La Sapienza at 1 site in Italy.
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 5 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: status not confirmed, as of the sponsor's last update on 23 Sep 2020. 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 Italy. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Alfredo Berardelli, MD (+390649914700, alfredo.berardelli@uniroma1.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in depression scores from baseline to month 1 and 3 (time frame: From baseline to month 1 and 3 after treatment). The study lists 2 primary outcomes in all. 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 NCT04560101, which also lists 8 linked publications.
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 23 Sep 2020.