Efficacy and Safety of DaxibotulinumtoxinA for Injection for the Treatment of Adult Upper Limb Spasticity

NCT03821402 | Completed Phase 2 Results FDA Drug

• 1 other identifier: 1820203
• Study Type: interventional
• Target: 83
• Locations: 1 country
• Sites: 26

Brief Summary

This is a randomized, Double-Blind, Placebo-Controlled, Parallel Group, Dose-Ranging, trial to Evaluate the Efficacy and Safety of DaxibotulinumtoxinA for Injection for the Treatment of Upper Limb Spasticity in Adults After Stroke or Traumatic Brain Injury. The study will be conducted in the U.S.A., approximately 128 adult subjects from approximately 30 study centers will be randomly assigned (1:1:1:1) to one of four treatment groups. The study consists of a 21-day screening period, a treatment visit and follow-up visits. The protocol was amended and the study was completed with fewer subjects than described in the initial protocol due to impact of COVID-19 on enrollment.

Conditions

Upper Limb Spasticity

Keywords

ULS; JUNIPER; DAXI for injection; Botulinum toxin

Outcome Measures

Primary Outcomes (2)

• Change From Baseline at Week 6 on the Modified Ashworth Scale (MAS) in the Suprahypertonic Muscle Group (SMG) Score

  Mean change from baseline at Week 6 in muscle tone measured with the Modified Ashworth Scale (MAS) in the Suprahypertonic Muscle Group (SMG) in one of the following: elbow, wrist, or finger flexors. Score range: 0 (Normal tone, no in tone) to 4 (Affected part{s} rigid in flexion or extension).

  Week 6

• Physician Global Impression of Change (PGIC) Score

  Mean score on the Physician Global Impression of Change (PGIC) score at week 6. The PGIC is a single-item, 9-point scale that measures the physician's impression of improvement following treatment. Score range: -4 (Markedly worse) to +4 (Markedly improved).

  Week 6

Secondary Outcomes (4)

• Muscle Tone Improvement Responder Rate

  Weeks 6 and 12

• Physician Global Impression of Change (PGIC) Responder Rate

  Weeks 6 and 12

• Change From Baseline at Weeks 6 and 12 on the Disability Assessment Scale (DAS) Functional Impairment

  Weeks 6 and 12

• Duration of Effect

  Up to 36 weeks

Study Arms (4)

DAXI 250 U

EXPERIMENTAL

DAXI for injection for the treatment of Upper Limb Spasticity (ULS) in Adults with 250 U dose

Biological: DAXI for injection 250 U

DAXI 375 U

EXPERIMENTAL

DAXI for injection for the treatment of Upper Limb Spasticity (ULS) in Adults with 375 U

Biological: DAXI for injection 375 U

DAXI 500 U

EXPERIMENTAL

DAXI for injection for the treatment of Upper Limb Spasticity (ULS) in Adults with 500 U

Biological: DAXI for injection 500 U

Placebo

PLACEBO COMPARATOR

Placebo group

Other: Placebo

Interventions

DAXI for injection 250 U BIOLOGICAL

DaxibotulinumtoxinA for injection is a sterile, white to off-white lyophilized product containing the active ingredient, daxibotulinumtoxinA, and inactive ingredients to be reconstituted with sterile, non-preserved, 0.9% sodium chloride solution saline

DAXI 250 U

DAXI for injection 375 U BIOLOGICAL

DaxibotulinumtoxinA for injection is a sterile, white to off-white lyophilized product containing the active ingredient, daxibotulinumtoxinA, and inactive ingredients to be reconstituted with sterile, non-preserved, 0.9% sodium chloride solution saline.

DAXI 375 U

DAXI for injection 500 U BIOLOGICAL

DaxibotulinumtoxinA for injection is a sterile, white to off-white lyophilized product containing the active ingredient, daxibotulinumtoxinA, and inactive ingredients to be reconstituted with sterile, non-preserved, 0.9% sodium chloride solution saline

DAXI 500 U

Placebo OTHER

Placebo is a sterile lyophilized product consisting of inactive ingredients without the neurotoxin to be reconstituted with sterile, non-preserved 0.9% sodium chloride solution.

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• to 75 years of age
• Written informed consent including authorization to release health information
• Focal upper limb spasticity (ULS) after a stroke (as defined by WHO criteria) or traumatic brain injury (TBI), last stroke or TBI \> 24 weeks prior to Screening
• ULS with the primary aggregate posture
• Moderate to severe ULS with a MAS score ≥ 2 at the elbow, wrist, and finger flexors
• Moderate to severe functional disability (Disability Assessment Score \[DAS\] score ≥2) on the principal target of treatment
• Has sufficient cognitive and communication ability to be able to give informed consent

You may not qualify if:

• Upper limb spasticity attributable to an etiology other than stroke or TBI.
• Bilateral upper limb paresis or quadriplegia.
• Initiated in physiotherapy of the upper extremities ≤ 30 days prior to Screening or planned to start physiotherapy of the upper extremities during the course of the study.
• Previous or planned treatment of the spastic upper limb with phenol, alcohol injection, or surgery
• Profound muscular atrophy or fixed contracture leading to marked limitation on range of motion
• Prior treatment with intrathecal baclofen
• Any neuromuscular neurologic conditions (amyotrophic lateral sclerosis, Lambert- Eaton, myasthenia gravis)

Sponsors & Collaborators

• Revance Therapeutics, Inc.: lead

Study Sites (26)

Rancho Research Institute at Rancho Los Amigos National Rehab Center

Downey, California, 90242, United States

Location

The Parkinsons and Movement Disorder Institute

Fountain Valley, California, 92708-5153, United States

Location

Collaborative Neuroscience Network LLC

Long Beach, California, 90806, United States

Location

SC3 Research

Pasadena, California, 91105, United States

Location

Yale University

Fairfield, Connecticut, 06824-5340, United States

Location

Waterbury Neurologists

Middlebury, Connecticut, 06762, United States

Location

Ki Health Partners LLC DBA New England Institute for Clinical Research

Stamford, Connecticut, 06905-1206, United States

Location

MedStar National Rehabilitation Hospital

Washington D.C., District of Columbia, 20010, United States

Location

Parkinsons Disease and Movement Disorders Center

Boca Raton, Florida, 33486, United States

Location

NW FL Clinical Research Group, LLC

Gulf Breeze, Florida, 32561-4458, United States

Location

Infinity Clinical Research

Hollywood, Florida, 33024, United States

Location

Parkinsons Disease Treatment Center of Southwest Florida

Port Charlotte, Florida, 33980-8421, United States

Location

Shirley Ryan AbilityLab

Chicago, Illinois, 60611-2654, United States

Location

Kansas Institute of Research

Overland Park, Kansas, 66211-1358, United States

Location

William Beaumont Hospital

Royal Oak, Michigan, 48073, United States

Location

Rusk Rehabilitation Hospital

Columbia, Missouri, 65203, United States

Location

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Weill Cornell Medical College

New York, New York, 10065-4870, United States

Location

Carolinas Rehabilitation

Charlotte, North Carolina, 28203, United States

Location

Wake Forest University School of Medicine

Salem, North Carolina, 27157, United States

Location

MossRehab

Elkins Park, Pennsylvania, 19027-2220, United States

Location

University of Pittsburgh School of Medicine

Pittsburgh, Pennsylvania, 15213, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

North Texas Institute of Neurology and Headache

Frisco, Texas, 75034, United States

Location

University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

University of Utah

Salt Lake City, Utah, 84132, United States

Location

MeSH Terms

Interventions

Injections

Intervention Hierarchy (Ancestors)

Drug Administration Routes; Drug Therapy; Therapeutics

Results Point of Contact

• Title: Todd Gross, PhD, VP, Clinical Development & Data Science
• Organization: Revance Therapeutics, Inc.

tgross@revance.com

510-742-3400

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Double Blinded. The subject, Sponsor, and study site research personnel will be blinded to the identity of the subject's assigned treatment.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Eligible subjects will be randomized (1:1:1:1) to one of four treatment groups: Group 1: DAXI 250 U (N=32); Group 2: DAXI 375 U (N=32); Group 3: DAXI 500 U (N=32); Group 4: Placebo (N=32).

Study Record Dates

• First Submitted: January 15, 2019
• First Posted: January 29, 2019
• Study Start: December 12, 2018
• Primary Completion: November 23, 2020
• Study Completion: November 23, 2020
• Last Updated: September 21, 2023
• Results First Posted: September 21, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will not share

Locations

US (26)