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

April 13, 2023 updated by: Johns Hopkins University

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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

Interventional

Enrollment (Anticipated)

90

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Recruiting
        • Johns Hopkins University
        • Contact:
        • Contact:
        • Principal Investigator:
          • Paul D Sponseller, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years to 14 years (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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.
Drug: AbobotulinumtoxinA
Paraspinal abobotulinumtoxinA injections (compared to placebo)
Other Names:
  • Dysport, Ipsen
Device: Custom Thoracolumbosacral Orthosis
Non-operative external bracing for scoliosis curves
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.
Device: Custom Thoracolumbosacral Orthosis
Non-operative external bracing for scoliosis curves
Drug: Placebos
Placebo prepared by Ipsen for use as control

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Assessed at baseline (time 0), 6 weeks, 4 months, 4 months + 6 weeks, 8 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Assessed at baseline (time 0), 6 weeks, 4 months, 4 months + 6 weeks, 8 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Johns Hopkins University

Investigators

  • 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 3, 2020

Primary Completion (Anticipated)

June 1, 2024

Study Completion (Anticipated)

March 1, 2025

Study Registration Dates

First Submitted

April 30, 2019

First Submitted That Met QC Criteria

April 30, 2019

First Posted (Actual)

May 2, 2019

Study Record Updates

Last Update Posted (Actual)

April 18, 2023

Last Update Submitted That Met QC Criteria

April 13, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • IRB00110399

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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