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Dysport ® as an Adjunctive Treatment to Bracing in the Management of Adolescent Idiopathic Scoliosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03935295
Recruitment Status : Not yet recruiting
First Posted : May 2, 2019
Last Update Posted : February 5, 2020
Sponsor:
Information provided by (Responsible Party):
Johns Hopkins University

Tracking Information
First Submitted Date  ICMJE April 30, 2019
First Posted Date  ICMJE May 2, 2019
Last Update Posted Date February 5, 2020
Estimated Study Start Date  ICMJE April 2020
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 30, 2019)
Change in scoliotic curve as assessed by the Major Curve Cobb Angle [ Time Frame: Assessed at baseline (time 0), 6 weeks, 4 months, 4 months + 6 weeks, 8 months ]
Change in scoliotic curve will be assessed using by the Major Curve Cobb Angle with measurement of standing scoliosis radiographs. Cobb angle refers to a radiographic measurement of side to side spinal curvature, measured in angle degrees.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03935295 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 30, 2019)
Change in Patient Reported Outcomes [ Time Frame: Assessed at baseline (time 0), 6 weeks, 4 months, 4 months + 6 weeks, 8 months ]
This will be assessed by Scoliosis Research Society (SRS) 22 Survey; The SRS-22 questionnaire is a validated measure used to assesses quality of life in patients with idiopathic scoliosis. It consists of five domains that assess: function, pain, mental health, self-image, and satisfaction with treatment. Higher scores indicate increased satisfaction. Each of the 22 questions are scored on a scale of 1 to 5.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Dysport ® as an Adjunctive Treatment to Bracing in the Management of Adolescent Idiopathic Scoliosis
Official Title  ICMJE Dysport ® (abobotulinumtoxinA) as an Adjunctive Treatment to Bracing in the Management of Adolescent Idiopathic Scoliosis
Brief Summary This study evaluates the combined effect of botulinum toxin A (administered as Dysport® (Ipsen Pharmaceuticals)) and bracing in children with adolescent idiopathic scoliosis. Two thirds of patients will be treated with Dysport® and bracing, while the remaining patients will be treated with placebo and bracing.
Detailed Description

Adolescent idiopathic scoliosis (AIS) is lateral curvature of the spine with an unknown cause in children 10-16 years old. Without treatment, skeletally immature children with idiopathic curves of 20°-40° risk curve progression. Treatment of these patients is focused on either observation or bracing. Although bracing has been shown to be more effective than observation, success is contingent on patient adherence and has the potential for further optimization with adjunct therapies.

Little research is available concerning adjunct therapies that may be used during bracing of AIS patients. One therapy in particular, abobotulinumtoxinA (BTX) injection, has been poorly studied. Injection of BTX into the paraspinal musculature of the concave aspect of the major curve may result in a more malleable curve and thus optimize brace treatment. If BTX injections are found to improve outcomes in this way, a new standard of nonoperative care could be established for AIS patients, potentially reducing the number of patients who undergo surgery.

The aim of this study is to assess whether BTX injections (administered as Dysport® (Ipsen Pharmaceuticals)) in the paraspinal musculature at the site of the major scoliotic curve decrease curve progression in skeletally immature AIS patients who are concurrently treated with bracing. Dysport® will be evaluated primarily as an adjunct treatment to bracing.

Hypotheses

  1. Dysport® injections into the concave-side paraspinal muscles will decrease the rate of curve progression, with a lower rate of curve progression in patients treated with both Dysport® injections and bracing compared with those treated with only bracing.
  2. Quantitative physical and mental health scores will improve to a greater extent in patients treated with Dysport® and bracing compared with patients treated with only bracing.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Adolescent Idiopathic Scoliosis
Intervention  ICMJE
  • Drug: AbobotulinumtoxinA
    Paraspinal abobotulinumtoxinA injections (compared to placebo)
    Other Name: Dysport, Ipsen
  • Drug: Placebos
    Placebo prepared by Ipsen for use as control
  • Device: Custom Thoracolumbosacral Orthosis
    Non-operative external bracing for scoliosis curves
Study Arms  ICMJE
  • Experimental: Botulinum Toxin

    The investigators plan to administer approximately 1000 U Dysport ® in the concave-sided paraspinal musculature of the major curve, based on an estimated total dose of 1000 U, the maximum allowable dose. The total dose per treatment session will not exceed 15 units/kilogram or 1000 units, whichever is lower. If two curves are equivalent within 3˚, both will be treated, however, the dosing (described above) will be divided equally across both curves.

    There will be two cycles of injections. Patients will be treated at time 0 (baseline) and 4 months.

    Interventions:
    • Drug: AbobotulinumtoxinA
    • Device: Custom Thoracolumbosacral Orthosis
  • Placebo Comparator: Placebo

    Control patients will receive an injection of placebo specifically prepared as a control for this study. The same volumes as indicated in the "experimental" arm description will be injected.

    These will be administered during two cycles of injections. Patients will be treated at time 0 (baseline) and 4 months.

    Interventions:
    • Drug: Placebos
    • Device: Custom Thoracolumbosacral Orthosis
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: April 30, 2019)
90
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2022
Estimated Primary Completion Date December 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion criteria:

  • Clinically determined idiopathic nature of scoliosis
  • Age 10-16 years
  • Risser stage 0,1,or 2
  • major curve of 20°-40°
  • curve apex caudal to T7 vertebra
  • ability to adhere to bracing protocol
  • Botulinum toxin naïve or previously treated greater than 6 months prior to study entry

Exclusion criteria:

  • Diagnosis of congenital scoliosis, neuromuscular scoliosis, or other "reason" for scoliosis - Previous surgery at any segment of the spine
  • Current need for surgery at any level of the spine
  • Treatment with any drug known to interfere with neuromuscular function
  • Any other medical condition, laboratory or diagnostic procedure finding that might preclude administration of BTX
  • Ongoing infection at the injection sites
  • Diagnosis as either resistant or sensitive to botulinum toxin treatment of any type or to any components of the BTX formulation
  • Cow milk protein allergy
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 10 Years to 16 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Vivian Tran, BS 4109553136 vtran17@jhmi.edu
Contact: Varun Puvanesarajah, MD 9193605646 vpuvane1@jhmi.edu
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03935295
Other Study ID Numbers  ICMJE IRB00110399
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Johns Hopkins University
Study Sponsor  ICMJE Johns Hopkins University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Paul Sponseller, MD, MBA Johns Hopkins Hospital Department of Orthopaedic Surgery
Principal Investigator: Varun Puvanesarajah, MD Johns Hopkins Hospital Department of Orthopaedic Surgery
PRS Account Johns Hopkins University
Verification Date February 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP