Factors Determining the Efficacy of Botulinum Toxin for Arm Tremor in Dystonia: An Exploratory Study

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Stichting Radboud universitair medisch centrum

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

20 Dec 2024 → ongoing

Decision date (initial)

2024-11-11

Transition trial

No

Low-intervention

Yes

Rare-disease indication

No

Vulnerable population

No

Funding sources

Radboud University Medical Centre · Canisius-Wilhelmina Hospital

External identifiers

EU CT number

2024-515970-28-00

ClinicalTrials.gov

NCT06411028

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To explore the associations between clinical, electrophysiological, ultrasonographic and (functional) magnetic resonance imaging tremor characteristics and botulinum neurotoxin efficacy in dystonic tremor syndrome of the upper extremity.

Secondary objectives 2

1. To explore the clinical, electrophysiological, ultrasonographic and (functional) magnetic resonance imaging differences between dystonic tremor and tremor associated with dystonia of the upper extremity.
2. To explore the agreement between a 1) clinical assessment, 2) polymyography and 3) muscle ultrasound on muscle selection in dystonic tremor syndrome of the upper extremity.

Conditions and MedDRA coding

Dystonic tremor syndrome

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Clinical diagnosis of dystonic tremor or tremor associated with dystonia according to the 2018 consensus statement on the classification of tremors
2. Tremor of one or both upper extremities
3. Starting botulinum neurotoxin injections as part of normal clinical practice
4. Age ≥ 18 years

Exclusion criteria 11

1. Acquired aetiology of dystonic tremor syndrome
2. Previous botulinum neurotoxin treatment of the to be treated upper extremity for ≥ 4 consecutive sessions
3. In case of previous botulinum neurotoxin treatment of the to be treated upper extremity for ≤3 consecutive sessions: the last botulinum neurotoxin injections ≤ 6 months before study enrolment
4. Unstable dose medications for dystonia and tremor ≤ 1 month before study enrolment
5. Deep brain stimulation implantation ≤ 6 months before study enrolment
6. Unstable deep brain stimulation variables ≤ 1 month before study enrolment
7. Comorbidity interfering with study participation
8. Known hypersensitivity for components of Dysport
9. Infection at the upper extremity
10. Pregnancy, trying to conceive and breastfeeding
11. Insufficient knowledge of the Dutch or English language

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The associations between clinical (e.g. dystonic tremor versus tremor associated with dystonia, jerkiness, dominant tremor pattern, sensory trick), electrophysiological (e.g. frequency-width at half-width power, intermuscular coherence), ultrasonographic (e.g. echogenicity, muscle thickness, tremulous muscles) and (functional) magnetic resonance imaging tremor characteristics (e.g. ratio of tremor-related activity in the basal ganglia versus cerebello-thalamo-cortical circuit)

Secondary endpoints 2

1. The clinical, electrophysiological, ultrasonographic and (functional) magnetic resonance imaging differences between dystonic tremor and tremor associated with dystonia at baseline (e.g. jerkiness, dominant tremor pattern, frequency-width at half-width power, intermuscular coherence, tremulous muscles, ratio of tremor-related activity in the basal ganglia versus cerebello-thalamo-cortical circuit).
2. The agreement between a clinical assessment, polymyography and muscle ultrasound on muscle selection

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Stichting Radboud universitair medisch centrum

Sponsor organisation

Stichting Radboud universitair medisch centrum

Address

Geert Grooteplein Zuid 10

City

Nijmegen

Postcode

6525 GA

Country

Netherlands

Scientific contact point

Organisation

Stichting Radboud universitair medisch centrum

Contact name

Anke Snijders

Public contact point

Organisation

Stichting Radboud universitair medisch centrum

Contact name

Anke Snijders

Locations

1 EU/EEA country · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 10 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications