Cervical Dystonia
Conditions
Keywords
spasmodic torticollis, focal dystonia
Brief summary
The goal of this retrospective, international, multi-center chart abstraction is to learn about the long-term impact of product-specific immunogenicity-related factors in different botulinum neurotoxin type A formulations in patients suffering from cervical dystonia. The main question it aims to answer is: Do complex-containing (CC) botulinum toxin formulations impact the long-term clinical outcome in cervical dystonia patients compared to a complex-free (CF) formulation? Researchers will compare differences observed in years 2 and 7 between two toxin groups, i.e., botulinum neurotoxins type A containing complexing proteins (CC) and without complexing proteins (CF).
Detailed description
Botulinum neurotoxin type A (BoNT/A) is first-line treatment in patients suffering from cervical dystonia. Effect of BoNT/A is temporary and must be repeated to maintain clinical effect. As for all biologics, repeated treatment bears the risk of activating an immune response due to the immunogenic nature of foreign proteins. Clinical signs of a potential immune response are reduced, or loss of efficacy, decreased duration of effect, and the need of a dose increase to maintain effect. Due to the different degree of purity and protein content, it is reasonable to assume that commercial BoNT/A formulations differ in immunogenic properties. Pivotal clinical trials and monocentric real-world studies demonstrated an increased incidence of neutralizing antibodies (NAbs) and NAb-associated partial or complete secondary non-response. However, the clinical relevance of potential immunogenicity-related mechanisms has not been demonstrated in a larger multicentric cohort in a real-world setting. This chart abstraction is designed to address this gap.
Interventions
BIOLOGICALCC BoNT/A
Complex-containing BotulinumtoxinA (BoNT/A) formulations
BIOLOGICALCF BoNT/A
Complex-free BotulinumtoxinA (BoNT/A) formulation
BIOLOGICALCF to CC BoNT/A
Switch from complex-free to complex-containing BoNT/A formulations
BIOLOGICALCC to CF BoNT/A
Switch from complex-containing to complex-free BoNT/A formulations
Sponsors
Heinrich-Heine University, Duesseldorf
Merz Therapeutics GmbH
Study design
Observational model
CASE_CONTROL
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 64 Years
Healthy volunteers
No
Inclusion criteria
* Clinical diagnosis of cervical dystonia * Adults (m/f) 18-64 years of age at start of BoNT/A treatment * Patient's written informed consent if required by local and/or national law.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of patients with a clinically meaningful change in dose-effect at year 7 compared to reference year 2 between complex-free and complex-containing BoNT/A monotherapy | Year 2 and year 7 | Dose-effect is a change in treatment response following dose adjustment. |
Secondary
| Measure | Time frame |
|---|---|
| Clinical meaningfulness of change in efficacy from baseline (first visit on record) at each visit in years 2, 5, 7, 10 in all treatment groups | Baseline (first visit on record), years 2, 5, 7, and 10 |
Other
| Measure | Time frame |
|---|---|
| Health-related quality of life measured by the SF-36 | Years 2, 5,7, and 10 |
| Incidence of frequent AEs overall and in patients with altered dose-effect | Years 2, 5,7, and 10 |
| Sub-analysis of all outcome measures in switcher population (patients treated with more than one BoNT/A formulation) | Years 2, 5,7, and 10 |
| Health-related quality of life measured by the CDQ24 | Years 2, 5,7, and 10 |
| Health-related quality of life measured by the EQ-5D | Years 2, 5,7, and 10 |
Countries
Germany
Outcome results
None listed