Cervical Dystonia MedDRA version: 21.0 Level: LLT Classification code 10064124 Term: Cervical dystonia System Organ Class: 100000004859
Conditions
Interventions
Product Name: DaxibotulinumtoxinA for injection
Product Code: RT002
Pharmaceutical Form: Powder for solution for injection
INN or Proposed INN: Daxibotu
Sponsors
Revance Therapeutics Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Adults, 18 to 80 years of age 2. Meets diagnostic criteria for isolated CD (idiopathic; dystonic symptoms localized to the head, neck, shoulder areas) with at least moderate severity at Baseline (Day 1), defined as a TWSTRS-total score of at least 20, with at least 15 on the TWSTRS-Severity subscale, at least 3 on the TWSTRS-Disability subscale, and at least 1 on the TWSTRS-Pain subscale (minimum TWSTRS subscale criteria applicable only to subjects not previously enrolled in Study Protocol 1720302) 3. Subjects who were previously enrolled in Study Protocol 1720302, who completed the study, including: a) Those with no reduction or have an increase from baseline in the average TWSTRS-total score at Weeks 4 and 6 (i.e., improvement or worsened disease status), and the investigator agreed that there was a need for retreatment based on the subject’s symptoms and neurologic examination findings b) Those who benefited from study treatment and complete follow-up study visits up to the time point of when their TWSTRS-total score reached/exceeded their target TWSTRS score c) Those who benefit from study treatment but subsequently experienced significant recurrence of CD symptoms (e.g., pain) during the study before their TWSTRS-total score reached their target TWSTRS score and requested retreatment, which the investigator determined was warranted due based on the subject’s symptoms and neurologic examination findings d) Those who completed study visits up to Week 36 and their TWSTRS-total score never reached their target TWSTRS score and they never requested another treatment. The investigator determined that these subjects can be followed in the OLS until their TWSTRS-total score is the same or higher than their target TWSTRS score or until they request retreatment, which the investigator determined is clinically indicated 4. De novo subjects (not previously enrolled in Study Protocol 1720302): a) Naïve to BoNT treatment b) BoNT treatment-experienced; if previously treated with BoNTA, the subject must have demonstrated a clinically meaningful response to the last BoNTA treatment based on the clinical judgment of the investigator 5. Written informed consent including authorization to release health information Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 245 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 105
Exclusion criteria
Exclusion criteria: 1. Cervical dystonia attributable to an underlying etiology, (e.g., traumatic torticollis or tardive torticollis) 2. Predominant retrocollis or anterocollis CD 3. Significant dystonia in other body areas, or is currently being treated with BoNT for dystonia in areas other than those associated with isolated CD 4. Severe dysphagia (Grade 3 or 4 on the Dysphagia Severity Scale) at Screening or Baseline (prior to study treatment) 5. Any neuromuscular neurological conditions that may place the subject at increased risk of morbidity with exposure to BoNT, including peripheral motor neuropathic diseases (e.g., amyotrophic lateral sclerosis and motor neuropathy, and neuromuscular junctional disorders such as Lambert-Eaton syndrome and myasthenia gravis) 6. Previous treatment with any BoNT product, except investigational daxibotulinumtoxinA, for any condition within the 16 weeks prior to Screening (applicable only to de novo subjects) 7. Botulinum neurotoxin treatment-experienced subjects who have historically required 450 msec (males) or > 470 msec (females), heart block (i.e., second degree AV block Mobitz Type 2, third degree AV block or complete heart block), or ven
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - To evaluate the long-term safety of multiple continuous treatments of DAXI for injection - To assess immunogenicity to BoNTA and RTP004 after multiple treatments of DAXI for injection ;Timepoint(s) of evaluation of this end point: It will be assessed at weeks 4 and 6; Secondary Objective: - To evaluate the long-term efficacy of multiple continuous treatments of DAXI for injection - To establish the inter-treatment time interval or duration of effect - To evaluate changes in symptom burden, daily activities, and psychosocial functioning after multiple continuous treatments of DAXI for injection ; Primary end point(s): - The dose- and cycle-specific incidence of drug-related AEs - The dose- and cycle-specific incidence of study drug discontinuation due to drug-related AEs - The dose and cycle-specific incidence of treatment-emergent immunogenicity | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Inter-treatment time interval or duration of effect • The dose- and cycle-specific average of the change in the TWSTRS-total score at Weeks 4 and 6 of each treatment cycle • Percentage of subjects with at least “moderate” (a 2-point) improvement on CGIC at Week 4 or Week 6 of the treatment cycle • Percentage of subjects with at least “moderate” (a 2-point) improvement on PGIC at Week 4 or Week 6 of the treatment cycle • Changes in quality of life measures based on the CDIP-58 ;Timepoint(s) of evaluation of this end point: It will be assessed at Week 6. | — |
Countries
Austria, Canada, Czech Republic, France, Germany, Hungary, Italy, Poland, Spain, United Kingdom, United States
Contacts
Public ContactRegulatory Affairs Manager
Revance Therapeutics Inc
Outcome results
None listed