Assessment of Long-term Clinical Response to BoNT in Cervical Dystonia

NCT05884528 | Completed

• 1 other identifier: M602011073
• Study Type: observational
• Target: 270
• Locations: 1 country
• Sites: 1

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).

Conditions

Cervical Dystonia

Keywords

spasmodic torticollis, focal dystonia

Outcome Measures

Primary Outcomes (1)

• 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

  Dose-effect is a change in treatment response following dose adjustment.

  Year 2 and year 7

Secondary Outcomes (1)

• 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 Outcomes (5)

• Incidence of frequent AEs overall and in patients with altered dose-effect

  Years 2, 5,7, and 10

• Health-related quality of life measured by the EQ-5D

  Years 2, 5,7, and 10

• Health-related quality of life measured by the SF-36

  Years 2, 5,7, and 10

Study Arms (4)

Complex-free BoNT/A formulation

Patients exclusively treated with the complex-free (CF) formulation (incobotulinumtoxinA). A maximum of 328 patients will be enrolled in this group.

Biological: CF BoNT/A

Complex-containing BoNT/A formulations

Patients exclusively treated with a single complex-containing (CC) formulation (onabotulinumtoxinA or abobotulinumtoxinA). A maximum of 328 patients will be enrolled in this group.

Biological: CC BoNT/A

Switcher CF to CC BoNT/A formulations

The CF to CC switcher group includes all patients that were switched from a CF to a CC BoNT/A formulation. If more than one switch occurred, the first switch determines the group. Both switcher groups will in sum not exceed 325 patients.

Biological: CF to CC BoNT/A

Switcher CC to CF BoNT/A formulations

The CC to CF switcher group includes all patients that were switched from a CC to a CF BoNT/A formulation. If more than one switch occurred, the first switch determines the group. Both switcher groups will in sum not exceed 325 patients.

Biological: CC to CF BoNT/A

Interventions

CC BoNT/A BIOLOGICAL

Complex-containing BotulinumtoxinA (BoNT/A) formulations

Also known as: onabotulinumtoxinA, abobotulinumtoxinA

Complex-containing BoNT/A formulations

CF BoNT/A BIOLOGICAL

Complex-free BotulinumtoxinA (BoNT/A) formulation

Also known as: incobotulinumtoxinA, NT 201, Botulinum toxin type A (150 kiloDalton), free from complexing proteins

Complex-free BoNT/A formulation

CF to CC BoNT/A BIOLOGICAL

Switch from complex-free to complex-containing BoNT/A formulations

Also known as: incobotulinumtoxinA, onabotulinumtoxinA, abobotulinumtoxinA

Switcher CF to CC BoNT/A formulations

CC to CF BoNT/A BIOLOGICAL

Switch from complex-containing to complex-free BoNT/A formulations

Also known as: onabotulinumtoxinA, abobotulinumtoxinA, incobotulinumtoxinA

Switcher CC to CF BoNT/A formulations

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

The study population will comprise of patients with a diagnosis of cervical dystonia who received regular BoNT/A injections for symptomatic treatment of the disease. The sub-populations consist of long-term patients treated with only ever one BoNT/A formulation (monotherapy) or 2 BoNT/A formulations (switcher).

* 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.

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Merz Therapeutics GmbH: lead
• Heinrich-Heine University, Duesseldorf: collaborator

Study Sites (1)

Düsseldorf University Hospital

Düsseldorf, North Rhine-Westphalia, 40255, Germany

Location

Related Publications (4)

• Ware JE Jr, Kosinski M, Gandek B, Aaronson NK, Apolone G, Bech P, Brazier J, Bullinger M, Kaasa S, Leplege A, Prieto L, Sullivan M. The factor structure of the SF-36 Health Survey in 10 countries: results from the IQOLA Project. International Quality of Life Assessment. J Clin Epidemiol. 1998 Nov;51(11):1159-65. doi: 10.1016/s0895-4356(98)00107-3.

  PMID: 9817133 BACKGROUND

• Albanese A, Bhatia K, Bressman SB, Delong MR, Fahn S, Fung VS, Hallett M, Jankovic J, Jinnah HA, Klein C, Lang AE, Mink JW, Teller JK. Phenomenology and classification of dystonia: a consensus update. Mov Disord. 2013 Jun 15;28(7):863-73. doi: 10.1002/mds.25475. Epub 2013 May 6.

  PMID: 23649720 BACKGROUND

• Albrecht P, Jansen A, Lee JI, Moll M, Ringelstein M, Rosenthal D, Bigalke H, Aktas O, Hartung HP, Hefter H. High prevalence of neutralizing antibodies after long-term botulinum neurotoxin therapy. Neurology. 2019 Jan 1;92(1):e48-e54. doi: 10.1212/WNL.0000000000006688. Epub 2018 Nov 21.

  PMID: 30464031 BACKGROUND

• Carr WW, Jain N, Sublett JW. Immunogenicity of Botulinum Toxin Formulations: Potential Therapeutic Implications. Adv Ther. 2021 Oct;38(10):5046-5064. doi: 10.1007/s12325-021-01882-9. Epub 2021 Sep 13.

  PMID: 34515975 BACKGROUND

MeSH Terms

Conditions

Torticollis; Dystonic Disorders

Interventions

Botulinum Toxins, Type A; abobotulinumtoxinA; incobotulinumtoxinA

Condition Hierarchy (Ancestors)

Dystonia; Dyskinesias; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Movement Disorders; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Botulinum Toxins; Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors

Study Officials

• Merz Medical Expert

  Merz Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2023
• First Posted: June 1, 2023
• Study Start: July 8, 2023
• Primary Completion: May 31, 2024
• Study Completion: July 31, 2024
• Last Updated: August 21, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

DE (1)