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The Use of Botulinum Toxin A Injections As An Adjunct to Bracing to Treat Adolescent Idiopathic Scoliosis

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ClinicalTrials.gov Identifier: NCT03935295
Recruitment Status : Not yet recruiting
First Posted : May 2, 2019
Last Update Posted : January 17, 2020
Sponsor:
Information provided by (Responsible Party):
Johns Hopkins University

Brief Summary:
This study evaluates the combined effect of botulinum toxin A and bracing in children with adolescent idiopathic scoliosis. Two thirds of patients will be treated with botulinum toxin A and bracing, while the remaining patients will be treated with placebo and bracing.

Condition or disease Intervention/treatment Phase
Adolescent Idiopathic Scoliosis Drug: AbobotulinumtoxinA Drug: Placebos Device: Custom Thoracolumbosacral Orthosis Phase 4

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, botulinum toxin (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 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. BTX injections will be evaluated primarily as an adjunct treatment to bracing.

Hypotheses

  1. BTX 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 BTX 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 BTX and bracing compared with patients treated with only bracing.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 90 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Dysport ® (abobotulinumtoxinA) as an Adjunctive Treatment to Bracing in the Management of Adolescent Idiopathic Scoliosis
Estimated Study Start Date : April 2020
Estimated Primary Completion Date : December 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Botox Scoliosis

Arm Intervention/treatment
Experimental: Botulinum Toxin

We 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 abotulinumtoxinA injections (compared to placebo)
Other Name: Dysport, Ipsen

Device: Custom Thoracolumbosacral Orthosis
Non-operative external bracing for scoliosis curves

Placebo Comparator: Placebo

Control patients will receive an injection of saline 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.

Drug: Placebos
Placebo prepared by Ipsen for use as control

Device: Custom Thoracolumbosacral Orthosis
Non-operative external bracing for scoliosis curves




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   10 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03935295


Contacts
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Contact: Vivian Tran, BS 4109553136 vtran17@jhmi.edu
Contact: Varun Puvanesarajah, MD 9193605646 vpuvane1@jhmi.edu

Sponsors and Collaborators
Johns Hopkins University
Investigators
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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

Additional Information:
Publications:

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Responsible Party: Johns Hopkins University
ClinicalTrials.gov Identifier: NCT03935295    
Other Study ID Numbers: IRB00110399
First Posted: May 2, 2019    Key Record Dates
Last Update Posted: January 17, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Keywords provided by Johns Hopkins University:
AIS
adolescent idiopathic scoliosis
Additional relevant MeSH terms:
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Scoliosis
Spinal Curvatures
Spinal Diseases
Bone Diseases
Musculoskeletal Diseases
Botulinum Toxins
abobotulinumtoxinA
Botulinum Toxins, Type A
Acetylcholine Release Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Cholinergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Neuromuscular Agents
Peripheral Nervous System Agents