A Clinical Study to Investigate the Effectiveness and Safety of DaxibotulinumtoxinA for Injection in subjects with Isolated Cervical Dystonia (ASPEN-1)

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Center Trial to Evaluate the Efficacy and Safety of a Single Treatment of DaxibotulinumtoxinA for Injection in Adults with Isolated Cervical Dystonia (ASPEN-1) - ASPEN-1

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-000446-19-AT

Enrollment

300

Registered

2018-09-13

Start date

2018-11-30

Completion date

Unknown

Last updated

2020-08-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cervical Dystonia MedDRA version: 21.0 Level: LLT Classification code 10064124 Term: Cervical dystonia System Organ Class: 100000004859

Interventions

Product Name: DaxibotulinumtoxinA for injection Product Code: RT002 Pharmaceutical Form: Powder for solution for injection INN or Proposed INN: DaxibotulinumtoxinA CAS Number: 93384-43-1 Other descrip

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Adults, 18 to 80 years of age 2. Meets diagnostic criteria for isolated CD (idiopathic; dystonic symptoms localized to the head, neck, shoulder areas) with at least moderate severity at Baseline (Day 1), defined as a TWSTRS-total score of at least 20, with at least 15 on the TWSTRS-Severity subscale, at least 3 on the TWSTRS-Disability subscale, and at least 1 on the TWSTRS-Pain subscale 3. Written informed consent including authorization to release health information Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 210 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 90

Exclusion criteria

Exclusion criteria: 1. Cervical dystonia attributable to an underlying etiology, (e.g., traumatic torticollis or tardive torticollis) 2. Predominant retrocollis or anterocollis CD 3. Significant dystonia in other body areas, or is currently being treated with BoNT for dystonia in areas other than those associated with isolated CD 4. Severe dysphagia (Grade 3 or 4 on the Dysphagia Severity Scale) at Screening or Baseline (prior to study treatment) 5. Any neuromuscular neurological conditions that may place the subject at increased risk of morbidity with exposure to BoNT, including peripheral motor neuropathic diseases (e.g., amyotrophic lateral sclerosis and motor neuropathy, and neuromuscular junctional disorders such as Lambert-Eaton syndrome and myasthenia gravis) 6. Previous treatment with any BoNT product for any condition within the 14 weeks prior to Screening 7. Botulinum neurotoxin treatment-experienced subjects who have historically required 450 msec (males) or > 470 msec (females), heart block (i.e, second degree AV block Mobitz Type 2, third degree AV block or complete heart block), or ventricular tachycardia 18. History of severe (stage 3) chronic obstructive pulmonary disease, or unstable pulmonary disease within 30 days prior to Screening 19. History of chronic or recurrent hypokalemia, or serum potassium < 2.5 mEq/L or mmol/L on chemistry at Screening 20. History of congestive heart failure, (New York Heart Association Class III or IV), Torsade de Pointe (TdP), and/or Long QT Syndrome, 21. Participants in

Design outcomes

Primary

Measure	Time frame
Main Objective: To compare the efficacy of a high and low dose of DAXI for injection (250 U; 125 U) relative to placebo, and to each other, in adults with moderate to severe, isolated CD;Secondary Objective: - To establish duration of effect for DAXI for injection - To assess safety and immunogenicity of DAXI for injection;Primary end point(s): The average of the change from baseline in TWSTRS-total score at Weeks 4 and 6.;Timepoint(s) of evaluation of this end point: It will be assessed at weeks 4 and 6	—

Measure

Time frame

Main Objective: To compare the efficacy of a high and low dose of DAXI for injection (250 U; 125 U) relative to placebo, and to each other, in adults with moderate to severe, isolated CD;Secondary Objective: - To establish duration of effect for DAXI for injection - To assess safety and immunogenicity of DAXI for injection;Primary end point(s): The average of the change from baseline in TWSTRS-total score at Weeks 4 and 6.;Timepoint(s) of evaluation of this end point: It will be assessed at weeks 4 and 6

—

Secondary

Measure	Time frame
Secondary end point(s): • Change from baseline in TWSTRS-total score (all post-treatment timepoints) • Duration of effect, defined as time (number of weeks) from treatment to loss of at least 80% of the peak treatment effect achieved at Weeks 4 and 6 (i.e., the target TWSTRS score) • Percentage of subjects with at least “moderate” (a 2-point) improvement on CGIC at Week 4 or 6 • Percentage of subjects with at least “moderate” (a 2-point) improvement on PGIC at Week 4 or 6 • Safety, which includes adverse events (AEs), presence of serum neutralizing antibodies to BoNTA and novel excipient RTP004, hematology, serum chemistry, urinalysis, ECG, vital signs, physical and neurologic examinations, and clinically significant changes in pulmonary function by spirometry (FEV1 / FVC);Timepoint(s) of evaluation of this end point: It will be assessed at Week 6.	—

Measure

Time frame

Secondary end point(s): • Change from baseline in TWSTRS-total score (all post-treatment timepoints) • Duration of effect, defined as time (number of weeks) from treatment to loss of at least 80% of the peak treatment effect achieved at Weeks 4 and 6 (i.e., the target TWSTRS score) • Percentage of subjects with at least “moderate” (a 2-point) improvement on CGIC at Week 4 or 6 • Percentage of subjects with at least “moderate” (a 2-point) improvement on PGIC at Week 4 or 6 • Safety, which includes adverse events (AEs), presence of serum neutralizing antibodies to BoNTA and novel excipient RTP004, hematology, serum chemistry, urinalysis, ECG, vital signs, physical and neurologic examinations, and clinically significant changes in pulmonary function by spirometry (FEV1 / FVC);Timepoint(s) of evaluation of this end point: It will be assessed at Week 6.

—

Countries

Austria, Canada, Czech Republic, France, Germany, Hungary, Italy, Poland, Spain, United Kingdom, United States

Contacts

Public ContactRegulatory Affairs Manager

Revance Therapeutics Inc

sfors@revance.com+15107423666

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026