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An RCT of a Patient-initiated Treatment Service for BEB and HFS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02577224
Recruitment Status : Unknown
Verified September 2016 by Moorfields Eye Hospital NHS Foundation Trust.
Recruitment status was:  Active, not recruiting
First Posted : October 16, 2015
Last Update Posted : March 22, 2017
Merz Pharmaceuticals
City, University of London
Information provided by (Responsible Party):
Moorfields Eye Hospital NHS Foundation Trust

Brief Summary:
Blepharospasm and hemifacial spasm are debilitating conditions which significantly impact on patient quality of life. Cyclical treatment with botulinum toxin injections offers temporary relief but the duration of treatment efficacy is variable. The standard model of patient care defines routine fixed- time based scheduled treatment cycles which may lead to unnecessarily frequent treatment for some patients and experience of distressing symptoms in others if symptoms return before the scheduled follow up period. The present study aims to compare a patient-initiated model of care, where patients determine treatment timing, with the standard model of care by using a two-arm randomised controlled trial design. It is expected that patients able to initiate their own treatment will experience less variation in disease severity and disability over a treatment cycle as they will be empowered to take the initiative when they feel they need to have treatment. A range of other outcomes will be assessed including: confidence and satisfaction in the service, depression, anxiety, quality of life and, illness and treatment beliefs. If the patient-led care model is shown to be effective in preventing distress and reducing disability in these populations; and is found to be acceptable to service users, this will have significant implications for service organisation across the NHS.

Condition or disease Intervention/treatment Phase
Blepharospasm Hemi Facial Spasm Other: Patient-initiated treatment Not Applicable

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 130 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Health Services Research
Official Title: A Randomised Controlled Trial to Explore the Effectiveness and Cost-effectiveness of a Patient-initiated Botulinum Toxin Treatment Model for Blepharospasm and Hemifacial Spasm Compared to Treatment as Usual
Actual Study Start Date : August 2015
Estimated Primary Completion Date : October 2017
Estimated Study Completion Date : October 2017

Arm Intervention/treatment
Experimental: Intervention
Participants randomized to the intervention group (patient-initiated treatment) will be asked to initiate their own treatment during the nine months they are taking part in the trial. Intervention group participants will receive information about when and how to initiate an appointment. Contact details for the service will be provided along with information on how quickly an appointment will be made, with whom and the procedure in the case of an emergency. All patients requesting an appointment will be booked in to the next available slot within the twice weekly ring‐fenced nurse-led clinics. Any subsequent scheduled appointments will be cancelled and all future treatment will be initiated by the patient.
Other: Patient-initiated treatment
Please see Intervention Arm

No Intervention: Control
Participants in the control group will receive treatment as usual. This consists of scheduled appointments in the hospital-based nurse-led botulinum toxin clinic. The frequency with which these appointments takes place are based on clinical judgement, but tend to range between every 6 weeks to every 4 months.

Primary Outcome Measures :
  1. Disease severity [ Time Frame: 18 months ]
    For patients with blepharospasm and hemifacial spasm, disease severity and frequency of symptoms will be assessed using the Jankovic Rating Scale (JRS), a clinician completed measure. The JRS consists of two subscales that measure severity and frequency separately. A 2-point improvement in the JRS sum score is considered a clinically relevant improvement. For patients with hemifacial spasm an additional rating scale for severity and frequency of cheek involvement will be included, which has been recommended by the questionnaire developers.

  2. Disability [ Time Frame: 18 months ]
    The Blepharospasm Disability Index (BSDI)© is a patient self-report measure which asks patients' about six daily activities: reading, driving a vehicle, watching TV, shopping, walking and doing everyday activities. The measure is also recommended for use when assessing the impact of Botulinum toxin in hemifacial spasm. The measure has been found to possess good convergent validity with the JRS, good internal consistency (Cronbach's alpha = 0.88) and adequate test-retest reliability by its developers.

  3. Patient satisfaction [ Time Frame: 18 months ]
    Patient satisfaction will be measured using the 8-item Client Satisfaction Questionnaire (CSQ). The scale has good internal reliability, with Cronbach's alpha ranging from 0.93 to 0.93 and good construct validity

Secondary Outcome Measures :
  1. Side effects [ Time Frame: 18 months ]
    Individual side effects including ptosis, tearing, blurred vision, double vision, hematoma, foreign body sensation, will be recorded at each clinic visit. The total number of side effects will be recorded for each patient.

  2. Confidence in system of care [ Time Frame: 18 months ]
    Confidence in the system of care will be assessed using a 10cm visual analogue scale (VAS). Patients will be asked 'How confident are you that if you required treatment this system of care would be able to support you?' ranging from 'not at all confident' to 'completely confident'.

  3. Quality of life [ Time Frame: 18 months ]
    Quality of life will be measured using the Craniocervical dystonia questionnaire (CDQ-24) which was developed and validated in patients with blepharospasm, and has also been used with success in patients with hemifacial spasm. This 24 item measure assesses quality of life across five domains: stigma, emotional well-being, pain, activities of daily living and social/family life. The measure has been found to possess good internal consistency with Cronbach's alphas ranging from 0.77 to 0.89, good construct validity when compared who the SF-36, good discriminant validity and test retest reliability.

  4. Mood [ Time Frame: 18 months ]
    The Hospital Anxiety and Depression Scale (HADS) will be used to assess mood. The HADS is a 14-item self-screening questionnaire for depression and anxiety in patients with physical health problems. The two 7-item subscales measure how a person has been feeling in the past week. The scale provides cut-off scores for the presence of potentially clinical levels of anxiety and depression. A systematic review of the HADS has confirmed the factor structure, found the cut-off points to be valid against clinical interviews, reports excellent internal consistency (Cronbach's alpha: anxiety 0.68-0.93; depression 0.67-0.90)

  5. Illness perceptions [ Time Frame: 18 months ]
    Illness perceptions are cognitive representations or beliefs that a patient has about their illness. These concepts will be measured using the revised illness perceptions questionnaire (IPQ-R), which has been widely used and is a valid and reliable measure.

  6. Treatment beliefs [ Time Frame: 18 months ]
    Beliefs about botulinum toxin will be measured using the Treatment Representations Inventory (TRI). A 27-item measure, consisting of four subscales including 'treatment-value', 'treatment-concerns', 'decision-satisfaction' and 'cure'.

  7. Acceptability [ Time Frame: 18 months ]
    Acceptability of the new patient-initiated service and standard care will be measured using the 7-item Acceptability Questionnaire developed by the study team.

  8. Cost effectiveness [ Time Frame: 18 months ]
    An adapted version of the Client Service Receipt Inventory (CSRI) has been employed, which has been widely employed in previous studies, particularly in the mental health setting. The impact of the service on direct and indirect costs will be estimated at the end of the trial period.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Diagnosis of hemifacial spasm or blepharospasm
  • Attending a hospital-based botulinum toxin clinic at Moorfields Eye Hospital NHS Foundation Trust.
  • Patients stable on botulinum toxin treatment, defined as receiving toxin treatment at stable doses over two previous cycles and free from and side effects.
  • Aged 18 years old and over.
  • Capacity to give informed consent to participate in the study, judged by the Research Nurse.

Exclusion Criteria:

  • Participants with significant co-morbidities (i.e. their predominant treatment is for another illness).
  • Inability to communicate fluently in written and/or spoken English, to complete study measures.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02577224

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United Kingdom
Moorfields Eye Hospital NHS Foundation Trust
London, United Kingdom, EC1V 2PD
Sponsors and Collaborators
Moorfields Eye Hospital NHS Foundation Trust
Merz Pharmaceuticals
City, University of London
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Principal Investigator: Daniel Ezra, MD FRCOph Moorfields Eye Hospital NHS Foundation Trust

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Responsible Party: Moorfields Eye Hospital NHS Foundation Trust Identifier: NCT02577224    
Other Study ID Numbers: EZRD1024
First Posted: October 16, 2015    Key Record Dates
Last Update Posted: March 22, 2017
Last Verified: September 2016
Keywords provided by Moorfields Eye Hospital NHS Foundation Trust:
Patient-led care
Botulinum toxin
Randomised controlled trial
Additional relevant MeSH terms:
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Hemifacial Spasm
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Eyelid Diseases
Eye Diseases
Mouth Diseases
Stomatognathic Diseases