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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

 No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

 Interventional
Eligible Ages 10 Years - 16 Years
Gender All
More Inclusion & Exclusion 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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

 NCT03935295
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

 Phase 4
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

 Johns Hopkins University
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

 Paul Sponseller, MD, MBAVarun Puvanesarajah, MD
Principal Investigator Affiliation Johns Hopkins Hospital Department of Orthopaedic SurgeryJohns Hopkins Hospital Department of Orthopaedic Surgery
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

 Other
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

 Adolescent Idiopathic Scoliosis
Study Website: View Trial Website
Additional Details

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.

Arms & Interventions

Arms

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.

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: - AbobotulinumtoxinA

Paraspinal abobotulinumtoxinA injections (compared to placebo)

Drug: - Placebos

Placebo prepared by Ipsen for use as control

Device: - Custom Thoracolumbosacral Orthosis

Non-operative external bracing for scoliosis curves

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Johns Hopkins University, Baltimore, Maryland

Status

Recruiting

Address

Johns Hopkins University

Baltimore, Maryland, 21287

Site Contact

Varun Puvanesarajah, MD

[email protected]

410-955-3136

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