A Study of IPN10200 for the Treatment of Cervical Dystonia in Adults

Phase 2RecruitingNCT06937931

Ipsen132 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

132

Conditions

Outcomes

Primary Outcomes

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 4

Secondary Outcomes

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.

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.

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 visit

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

Time to return of symptoms in responders (time from treatment to loss of 80% of peak treatment effect)

As assessed by TWSTRS total score. TWSTRS scale 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: From randomization until Week 36

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.

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

Change from baseline in Clinical Global Impression of Severity score

The Clinical Global Impression of Severity (CGI-S) is a clinician rated scale that measures the severity of an illness in a participant. It is rated on a 7-point scale ranging from 1 (normal) to 7 (severely ill) in answer to a question on the mental state of the participant at the time of the assessment.

Time frame: At all timepoints post injection until Week 36.

Clinical Global Impression of Change score

The Clinical Global Impression of Change (CGI-C) is a clinician rated scale that measures the clinical change in a participant. It is rated on a 7 point scale ranging from 1 (very much improved) to 7 (very much worse) in answer to a question on the mental state of the participant at the time of the assessment.

Time frame: At all timepoints post injection until Week 36.

Change from baseline in Patients' Global Impression of Severity score

The Patient Global Impression of Severity (PGI-S) is a global index that may be used to rate the severity of a specific condition (a single-state scale). The PGI-S is a single question asking the participant to rate how their condition is now on a scale of 1 (Normal) to 4 (Severe).