A study of IPN10200 for the treatment of cervical dystonia in adults

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ipsen Innovation

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

17 Jun 2025 → ongoing

Decision date (initial)

2025-05-20

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Ipsen Innovation

External identifiers

EU CT number

2024-519248-34-00

ClinicalTrials.gov

NCT06937931

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy

To assess the efficacy of IPN10200 treatment compared to placebo in reducing Cervical Dystonia (CD) symptoms at Week 4 in adult participants

Secondary objectives 11

1. To assess the efficacy of IPN10200 treatment compared to placebo over time in reducing Cervical Dystonia (CD) symptoms in adult participants with CD
2. To assess if the IPN10200 treatment compared to placebo reduces pain for the adult participants with CD
3. To evaluate the return of symptoms in adult participants with CD.
4. To assess if the IPN10200 treatment compared to placebo reduces disability for adult participants with CD
5. To assess if the IPN10200 treatment compared to placebo reduces severity of symptoms for adult participants with CD
6. To assess if the IPN10200 treatment compared to placebo reduces illness severity as assessed by the clinicians
7. To assess if the IPN10200 treatment compared to placebo improves illness symptoms as assessed by the clinicians
8. To assess if the IPN10200 treatment compared to placebo reduces illness severity as assessed by the participants
9. To assess if the IPN10200 treatment compared to placebo improves illness symptoms as assessed by the participants
10. To assess if the IPN10200 treatment compared to placebo reduces health impacts of CD
11. To assess the safety and tolerability of IPN10200 in participants with CD

Conditions and MedDRA coding

Cervical Dystonia

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

Yes

IPD plan description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board. Supporting information: Time Frame: Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and EU or after the primary manuscript describing the results has been accepted for publication, whichever is later. Access Criteria: Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/). URL: https://vivli.org/members/ourmembers/

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

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 5

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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from Baseline in the Toronto Western Spasmodic Torticollis Rating Scale (TWSTRS) total score. [Time frame: at week 4]

Secondary endpoints 20

1. Change from Baseline in the TWSTRS total score at all other scheduled timepoints post injection until Week 36. [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 [Time Frame: Averaged over every 7-day period until the Week 4 visit]
4. 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]
5. Time to return of symptoms in responders (time from treatment to loss of 80% of peak treatment effect) [Time Frame: From randomization until Week 36]
6. Change from Baseline in the TWSTRS-Disability Subscale. The TWSTRS disability subscale assesses the effect that Cervical Dystonia (CD) has on the participant’s daily activities and the subscore has a maximum of 30 and consists of six items. [Time Frame: At all timepoints post injection until Week 36.]
7. Change from Baseline in the TWSTRS-Severity Subscale. The TWSTRS severity subscale rates the maximal excursion of the torticollis (degree of tilt or rotation), elevation, range of motion, etc. This severity subscore has a maximum subtotal of 35 and consists of 11 items. [Time Frame: At all scheduled timepoints post injection until Week 36]
8. Change from baseline in Clinical Global Impression of Severity (CGI-S) score. [Time Frame: At all timepoints post injection until Week 36.]
9. Clinical Global Impression of Change (CGI-C) score [Time Frame: At all timepoints post injection until Week 36.]
10. Change from baseline in Patients' Global Impression of Severity (PGI-S) score [Time Frame: At all timepoints post injection until Week 36]
11. Patients' Global Impression of Change (PGI-S) score [Time Frame: At all timepoints post injection until Week 36]
12. Change from Baseline in the Cervical Dystonia Impact Profile (CDIP-58) total score. [Time Frame: At all timepoints post injection until Week 36]
13. 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. [Time Frame: From baseline to Week 36.]
14. Percentage of participants with clinically significant changes from baseline in Laboratory Parameters Clinically significant change in laboratory parameters will be reported. The clinical significance will graded by the investigator. [Time Frame: At all timepoints post injection until Week 36]
15. Percentage of Participants With Clinically Significant Changes from baseline in Vital Signs Clinically significant changes in vital signs will be reported. The clinical significance will be graded by the investigator. [Time Frame: At all timepoints post injection until Week 36]
16. Percentage of participants with clinically significant change from baseline in focused neurological/physical examinations. [Time Frame: At all timepoints post injection until Week 36]
17. Percentage of participants with clinically significant change from baseline in 12-lead Electrocardiogram (ECG) readings. [Time Frame: At all timepoints post injection until Week 36]
18. Treatment-emergence of suicidal ideation/suicidal behaviour. The Columbia Suicide Severity Rating Scale (C-SSRS) will be used in this study for the evaluation of suicidal ideation and behaviour. [Time Frame: From baseline to Week 36.]
19. Percentage of participants with Binding antibodies to IPN10200 [Time Frame: At all timepoints post injection until Week 36]
20. Percentage of participants with neutralising antibodies to IPN10200. [Time Frame: At all timepoints post injection until Week 36]

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ipsen Innovation

Sponsor organisation

Ipsen Innovation

Address

70 Rue Balard

City

Paris

Postcode

75015

Country

France

Scientific contact point

Organisation

Ipsen Innovation

Contact name

Ipsen Clinical Study Enquiries

Public contact point

Organisation

Ipsen Innovation

Contact name

Ipsen Clinical Study Enquiries

Third parties 6

Locations

6 EU/EEA countries · 30 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 122 files

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

Application history

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