Assessment of Long-term Clinical Response to BoNT in Cervical Dystonia (RELY-CD)

August 20, 2024 updated by: Merz Therapeutics GmbH

Assessment of Long-term Clinical Response to BoNT in Cervical Dystonia - Real-world Evidence of LongevitY of Botulinum Toxin in Cervical Dystonia

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Observational

Enrollment (Actual)

270

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
    • North Rhine-Westphalia
      • Düsseldorf, North Rhine-Westphalia, Germany, 40255
        • Düsseldorf University Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

No

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).

Description

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
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-free BotulinumtoxinA (BoNT/A) formulation
Other Names:
  • NT 201
  • Botulinum toxin type A (150 kiloDalton), free from complexing proteins
  • incobotulinumtoxinA
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
Complex-containing BotulinumtoxinA (BoNT/A) formulations
Other Names:
  • onabotulinumtoxinA
  • abobotulinumtoxinA
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
Switch from complex-free to complex-containing BoNT/A formulations
Other Names:
  • onabotulinumtoxinA
  • abobotulinumtoxinA
  • incobotulinumtoxinA
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
Switch from complex-containing to complex-free BoNT/A formulations
Other Names:
  • onabotulinumtoxinA
  • abobotulinumtoxinA
  • incobotulinumtoxinA

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Time Frame: Year 2 and year 7
Dose-effect is a change in treatment response following dose adjustment.
Year 2 and year 7

Secondary Outcome Measures

Outcome 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
Time Frame: Baseline (first visit on record), years 2, 5, 7, and 10
Baseline (first visit on record), years 2, 5, 7, and 10

Other Outcome Measures

Outcome Measure
Time Frame
Incidence of frequent AEs overall and in patients with altered dose-effect
Time Frame: Years 2, 5,7, and 10
Years 2, 5,7, and 10
Health-related quality of life measured by the EQ-5D
Time Frame: Years 2, 5,7, and 10
Years 2, 5,7, and 10
Health-related quality of life measured by the SF-36
Time Frame: Years 2, 5,7, and 10
Years 2, 5,7, and 10
Health-related quality of life measured by the CDQ24
Time Frame: Years 2, 5,7, and 10
Years 2, 5,7, and 10
Sub-analysis of all outcome measures in switcher population (patients treated with more than one BoNT/A formulation)
Time Frame: Years 2, 5,7, and 10
Years 2, 5,7, and 10

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Merz Therapeutics GmbH

Collaborators

Heinrich-Heine University, Duesseldorf

Investigators

  • Study Director: Merz Medical Expert, Merz Therapeutics

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 8, 2023

Primary Completion (Actual)

May 31, 2024

Study Completion (Actual)

July 31, 2024

Study Registration Dates

First Submitted

May 8, 2023

First Submitted That Met QC Criteria

May 22, 2023

First Posted (Actual)

June 1, 2023

Study Record Updates

Last Update Posted (Actual)

August 21, 2024

Last Update Submitted That Met QC Criteria

August 20, 2024

Last Verified

August 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • M602011073

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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