Clinical trial NCT06937931: A Study of IPN10200 for the Treatment of Cervical Dystonia in Adults
Official title: A Phase II, Multicentre, Randomised, Double-blind, Parallel-Group, Placebo Controlled Study to Evaluate the Efficacy and Safety of IPN10200 as a Treatment for Cervical Dystonia in Adult Participants
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Cervical Dystonia
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2025-04-22
Enrollment: 132
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the efficacy and safety of the study drug, 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. IPN10200 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 IPN10200 Dose A or Dose B (additional participants may receive IPN10200 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: Inclusion Criteria:

1. A clinical diagnosis of isolated Cervical Dystonia (CD) (idiopathic) characterized by dystonic symptoms localised to the head, neck, and shoulder areas with at least moderate severity at Screening and Baseline (Day 1) defined as:

   * (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
2. Treatment naïve or non-naïve to BoNT therapy for CD

Exclusion Criteria:

1. Participants presenting with a swallowing disorder of any origin which might be exacerbated by BoNT treatment, such as:

   * (a) Grade 3 or 4 on the Dysphagia Severity Scale (severe dysphagia) with swallowing difficulties and requiring a change in diet.
2. Predominant anterocollis.
3. Predominant retrocollis.
4. Traumatic torticollis or tardive torticollis.
5. Marked limitation on passive range of motion that suggests cervical contractures or structural abnormality.

Primary outcomes:
measure: Change from Baseline in the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score
timeFrame: At Week 4
Secondary outcomes:
measure: Change from Baseline in the TWSTRS total score at all other scheduled timepoints post injection until Week 36
timeFrame: At all timepoints post injection until Week 36.
measure: Change from Baseline in the TWSTRS-Pain Subscale
timeFrame: At all timepoints post injection until Week 36.
measure: Change from baseline in the daily Numerical Rating Scale (NRS) score
timeFrame: Averaged over every 7-day period until the Week 4 visit
measure: Time to onset of pain reduction
timeFrame: From study injection to first timepoint at which at least 2-point reduction is observed in NRS score
measure: Time to return of symptoms in responders (time from treatment to loss of 80% of peak treatment effect)
timeFrame: From randomization until Week 36
measure: Change from Baseline in the TWSTRS-Disability Subscale
timeFrame: At all timepoints post injection until Week 36.
measure: Change from Baseline in the TWSTRS-Severity Subscale
timeFrame: At all scheduled timepoints post injection until Week 36
measure: Change from baseline in Clinical Global Impression of Severity score
timeFrame: At all timepoints post injection until Week 36.
measure: Clinical Global Impression of Change score
timeFrame: At all timepoints post injection until Week 36.
measure: Change from baseline in Patients' Global Impression of Severity score
timeFrame: At all timepoints post injection until Week 36
measure: Patients' Global Impression of Change score
timeFrame: At all timepoints post injection until Week 36
measure: Change from Baseline in the CDIP-58 total score
timeFrame: At all timepoints post injection until Week 36
measure: Percentage of participants experiencing any Adverse Event (AEs) including treatment emergent adverse events (TEAEs), serious adverse events (SAEs), adverse event of special interest (AESI) and AE leading to treatment discontinuation
timeFrame: From baseline to Week 36.
measure: Percentage of participants with clinically significant changes from baseline in Laboratory Parameters
timeFrame: At all timepoints post injection until Week 36
measure: Percentage of Participants With Clinically Significant Changes from baseline in Vital Signs
timeFrame: At all timepoints post injection until Week 36
measure: Percentage of participants with clinically significant change from baseline in focused neurological/physical examinations.
timeFrame: At all timepoints post injection until Week 36
measure: Percentage of participants with clinically significant change from baseline in 12-lead Electrocardiogram (ECG) readings.
timeFrame: At all timepoints post injection until Week 36
measure: Treatment-emergence of suicidal ideation/suicidal behaviour
timeFrame: From baseline to Week 36.
measure: Percentage of participants with Binding antibodies to IPN10200
timeFrame: At all timepoints post injection until Week 36
measure: Percentage of participants with neutralising antibodies to IPN10200
timeFrame: At all timepoints post injection until Week 36
Other outcomes:
Locations:
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: