A Study of IPN10200 for the Treatment of Cervical Dystonia in Adults
ConditionCervical Dystonia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-80
SponsorIpsen
About this trial
The purpose of this study is to evaluate the efficacy and safety of the study drug, Corabotase (also known as IPN10200), and to assess how well it works when compared with placebo in treating Cervical Dystonia (CD) in adults.
CD can cause a series of abnormalities and symptoms in the head and neck that can lead to neck pain and stiffness, and headaches. CD is believed to involve deep parts within the brain that control movement, but genetic factors, environmental factors, and abnormalities in the brain may also play a role.
The usual treatment for CD includes injecting BoNT into the affected muscles, but the treatment only lasts about 3 months. Corabotase is designed to last for a longer period.
The study will consist of two periods:
1. A Screening Period of up to 4 weeks (28 days) to assess whether a participant can take part in the study and requires at least one visit. 2. A Treatment Period of 36 weeks.
On Day 1 of the treatment period, participants will receive either Corabotase Dose A or Dose B (additional participants may receive Corabotase Dose C) of the study drug, or placebo distributed into different muscles in the head, neck and shoulders. Participants may continue some other medications, but details need to be recorded.
There will be 10 visits to the clinic in person and one remote visits (phone call) (12 visits to the clinic for participants who receive Dose C). Participants will undergo blood samplings, urine collections, physical/neurological examinations, and clinical evaluations. Participants will also need to complete questionnaires throughout the study.
The total study duration for a participant will be up to 40 weeks (approximately 9 months).
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
(a) Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS)-Total score ≥20
(b) TWSTRS-Severity subscale score ≥15
(c) TWSTRS-Disability subscale score ≥3
(d) TWSTRS-Pain subscale score ≥ 1
Disqualifiers
(a) Grade 3 or 4 on the Dysphagia Severity Scale (severe dysphagia) with swallowing difficulties and requiring a change in diet.
Predominant anterocollis.
Predominant retrocollis.
Traumatic torticollis or tardive torticollis.
Trial design
Design model
Parallel
Treatments tested in this trial
Corabotase
Biological/VaccineStudy intervention will be administered in a single treatment cycle after randomisation (Day 1). The administration cycle consists of intramuscular injection.
Placebo
Other interventionExcipients without active substance will be administered in a single treatment cycle after randomisation (Day 1). The administration cycle consists of intramuscular injection.
Treatment groups
132 Participants
are divided into 5 treatment groupsGroup A: Group 1: Treatment Arm AExperimental treatment 1 intervention
Group B: Group 1: Treatment Arm BExperimental treatment 1 intervention
Group C: Group 1: Placebo ComparatorPlacebo comparator 1 intervention
Group D: Group 2: Treatment Arm CExperimental treatment 1 intervention
Group E: Group 2: Placebo ComparatorPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Change from Baseline in the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score
The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is a rating scale for Cervical Dystonia (CD) consisting of three subscales: severity, disability and pain scales. The total score is the sum of each of the subscales, with a range of 0 to 85, where higher scores are indicative of greater impairment.
Time frame
At Week 4Secondary outcomes
1
Change from Baseline in the TWSTRS total score at all other scheduled timepoints post injection until Week 36
The Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) is a rating scale for Cervical Dystonia (CD) consisting of three subscales: severity, disability and pain scales. The total score is the sum of each of the subscales, with a range of 0 to 85, where higher scores are indicative of greater impairment.
Time frame
At all timepoints post injection until Week 36.2
Change from Baseline in the TWSTRS-Pain Subscale
The TWSTRS pain subscale consists of three patient-rated items that assess the participants usual, worst and best pain from the preceding week, and the subscore has a maximum of 20.
Time frame
At all timepoints post injection until Week 36.3
Change from baseline in the daily Numerical Rating Scale (NRS) score
The Numeric Rating Scale (NRS) is a unidimensional measure of pain intensity in adults. The NRS is a segmented numeric version of the visual analogue scale in which a respondent selects a whole number (0 to 10 integers) that best reflects the intensity of his/her pain. The 11-point numeric scale ranges from '0' representing one pain extreme (e.g. "no pain") to '10' representing the other pain extreme (e.g. "pain as bad as you can imagine" or "worst pain imaginable").
Time frame
Averaged over every 7-day period until the Week 4 visit4
Time to onset of pain reduction
Defined as the duration from the administration of the study injection to the first recorded instance of a minimum 2-point decrease in the Numeric Rating Scale (NRS) score.
Time frame
From study injection to first timepoint at which at least 2-point reduction is observed in NRS score Source ClinicalTrials.gov