Dysport® + Bracing for Adolescent Idiopathic Scoliosis

Overseen By Gabrielle Reichard, MA

Age: < 18

Sex: Any

Trial Phase: Phase 4

Sponsor: Johns Hopkins University

Must not be taking: Neuromuscular drugs

Disqualifiers: Congenital scoliosis, Neuromuscular scoliosis, Surgery, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 10

Duration: 8 months

90 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment for adolescent idiopathic scoliosis, a condition where the spine curves in children for no known reason. Researchers are testing whether combining botulinum toxin A (Dysport®) injections with back bracing improves outcomes compared to bracing with a placebo. Participants will receive either Dysport® or a placebo along with their brace. The trial seeks children aged 10 to 16 with a specific type of scoliosis curve who have not recently received botulinum toxin treatment. As a Phase 4 trial, this research aims to understand how the already FDA-approved and effective treatment benefits more patients.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are taking drugs that interfere with neuromuscular function.

What is the safety track record for Dysport® and bracing in treating adolescent idiopathic scoliosis?

Research has shown that Dysport®, a type of botulinum toxin A, is generally safe for people. This treatment has been studied in various contexts and received FDA approval for conditions like muscle stiffness, indicating it has passed strict safety tests for those uses. In past studies with children, Dysport® was found to be safe, though it can have side effects like any treatment. These may include mild pain or swelling at the injection site, headache, or muscle weakness. Serious side effects are rare. This trial tests Dysport® along with bracing to assess its effectiveness for scoliosis in children. As a Phase 4 trial, the treatment is already approved, and the goal is to understand its effects better. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Researchers are excited about using Dysport® (a form of botulinum toxin) for treating adolescent idiopathic scoliosis because it offers a novel approach by targeting the muscles directly. Unlike the standard treatment options, such as bracing, which focus on external support, Dysport® works by relaxing the muscles on the concave side of the spinal curve, potentially reducing the curve's progression. This direct muscle action could complement existing methods, providing a more comprehensive approach to managing scoliosis. Additionally, Dysport® is administered via injection, which may offer a more targeted and less invasive alternative to surgical options.

What evidence suggests that Dysport® combined with bracing could be effective for adolescent idiopathic scoliosis?

Research has shown that botulinum toxin A, such as Dysport®, can reduce excessive muscle activity. This reduction may help manage spinal scoliosis by decreasing the spine's curvature. In this trial, one group of participants will receive Dysport® injections into specific back muscles. Previous studies found that these injections slowed the progression of the curve in adolescents with idiopathic scoliosis. This suggests that using Dysport® alongside bracing treatments could enhance the effectiveness of bracing. Overall, Dysport® supports existing scoliosis treatments by targeting the muscles that contribute to the curve. ^{1 5 6 7 8}

Who Is on the Research Team?

Varun Puvanesarajah, MD

Principal Investigator

Johns Hopkins Hospital Department of Orthopaedic Surgery

Paul Sponseller, MD, MBA

Principal Investigator

Johns Hopkins Hospital Department of Orthopaedic Surgery

Are You a Good Fit for This Trial?

This trial is for children aged 10-16 with adolescent idiopathic scoliosis, where the curve is below the T7 vertebra and measures 20°-40°. They should have no prior botulinum toxin treatment in the last 6 months, be able to follow a bracing protocol, and not have had spine surgery or need it currently. Kids with congenital or neuromuscular scoliosis, ongoing infections at potential injection sites, cow milk protein allergy, or on drugs affecting muscle function can't join.

Inclusion Criteria

I have never had botulinum toxin or it's been over 6 months since my last treatment.

My spine's major curve is between 20° and 40°.

The curve in your spine is below the T7 vertebra.

Exclusion Criteria

I need surgery on my spine.

I have a history of reacting to botulinum toxin treatments.

I have no health issues that would prevent me from taking BTX.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Dysport® or placebo injections in the paraspinal musculature and are treated with bracing

8 months

Injections at baseline and 4 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 months

Assessments at baseline, 6 weeks, 4 months, 4 months + 6 weeks, 8 months

What Are the Treatments Tested in This Trial?

Interventions

• AbobotulinumtoxinA
• Custom Thoracolumbosacral Orthosis
• Placebos

Trial Overview The study tests if Dysport® (botulinum toxin A) combined with wearing a custom back brace helps manage scoliosis better than just wearing the brace alone. Two-thirds of participants will receive Dysport® injections along with bracing; the rest will get placebo injections plus bracing.

How Is the Trial Designed?

Group I: Botulinum Toxin Experimental Treatment 2 Interventions

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.

Group II: Placebo Placebo Group 2 Interventions

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.

Who Is Running the Clinical Trial?

Johns Hopkins University

Lead Sponsor

Trials

2,366

Recruited

15,160,000+

Published Research Related to This Trial

In a study of 50 adolescents with idiopathic scoliosis treated with the Boston brace, those who wore the brace for 18 or more hours per day showed a significant curve correction of 49%, compared to 45% and 33% for those with less compliance.

Long-term follow-up (mean of 9.7 years) revealed that higher compliance with the brace significantly reduced the risk of curve progression beyond 45 degrees and the need for spinal fusion surgery, confirming the effectiveness of the Boston brace in managing larger scoliosis curves.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of the boston brace in treatment of large curves in adolescent idiopathic scoliosis. Wiley, JW., Thomson, JD., Mitchell, TM., et al.[2022]

The study evaluated the effectiveness of the thoracolumbosacral orthosis (TLSO) in treating idiopathic scoliosis in a well-defined group of 54 compliant patients compared to a control group of 47 patients who did not use the brace, over a mean follow-up period of 3.3 years.

Using multivariate analysis, the study aimed to provide clearer insights into TLSO's effectiveness, addressing previous criticisms regarding the lack of properly matched control studies and mixed patient types in earlier research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of TLSO bracing in the conservative treatment of idiopathic scoliosis. Fernandez-Feliberti, R., Flynn, J., Ramirez, N., et al.[2004]

In a study of 51 patients with adolescent idiopathic scoliosis treated with a Boston brace, 61% were successful in treatment, but 31% eventually required surgery, highlighting the challenges of managing larger curves.

Key predictors of treatment success included the degree of in-brace correction (at least 25% for double curves) and consistent brace wear (over 18 hours/day), particularly for patients with double curves and specific radiographic measurements.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Factors that influence outcome in bracing large curves in patients with adolescent idiopathic scoliosis. Katz, DE., Durrani, AA.[2022]

In a retrospective study of patients with adolescent idiopathic scoliosis (AIS) using the Boston Brace 3D, 84% of those with a single curve and 69% with a double curve experienced improvement or stabilization of their spinal curvature after treatment.

The effectiveness of the Boston Brace 3D in managing AIS was linked to the duration of brace wear, with patients who wore the brace for more hours each day showing better outcomes in curve reduction.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Short-Term Outcomes of the Boston Brace 3D Program Based on SRS and SOSORT Criteria: A Retrospective Study. Wynne, JH., Houle, LR.[2022]

The Charleston bending thoracolumbosacral orthosis (TLSO) was well tolerated by 32 patients with idiopathic scoliosis, showing excellent compliance and low psychological stress during nighttime use.

Out of the patients treated, 11 successfully completed treatment with minimal curve progression, while only 2 experienced significant curve worsening, suggesting that the TLSO may effectively control scoliosis progression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Results of treatment of idiopathic scoliosis with the Charleston bending orthosis. Federico, DJ., Renshaw, TS.[2019]

In a study of 108 adolescent idiopathic scoliosis patients, those treated with Rigo Chêneau orthoses (RCOs) showed significantly lower rates of progression to spinal surgery compared to those using Boston-style TLSOs (0% vs. 34%).

Patients using RCOs experienced less mean and percent progression of their spinal curves compared to those using TLSOs, indicating that RCOs may be more effective in managing scoliosis in adolescents.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of the Rigo Chêneau versus Boston-style orthoses for adolescent idiopathic scoliosis: a retrospective study. Minsk, MK., Venuti, KD., Daumit, GL., et al.[2020]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03935295

Dysport ® as an Adjunctive Treatment to Bracing in the ... Two thirds of patients will be treated with Dysport® and bracing, while the remaining patients will be treated with placebo and bracing.

2. centerwatch.com centerwatch.com/clinical-trials/listings/NCT03935295/dysport-r-as-an-adjunctive-treatment-to-bracing-in-the-management-of-adolescent-idiopathic-scoliosis

Dysport ® as an Adjunctive Treatment to Bracing in the ... Two thirds of patients will be treated with Dysport® and bracing, while the remaining patients will be treated with placebo and bracing.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9601180/

Effectiveness of Botulinum Neurotoxin in Treatment of ... Botulinum neurotoxin (BoNT) can reduce muscle overactivity and may have the potential to ameliorate spinal scoliosis. This study investigated ...

4. clinicaltrial.be clinicaltrial.be/en/details/63921?per_page=100&only_recruiting=0&only_eligible=0&only_active=0

Dysport ® as an Adjunctive Treatment to Bracing in the Ma... Dysport® injections into the concave-side paraspinal muscles will decrease the rate of curve progression, with a lower rate of curve progression ...

5. withpower.com withpower.com/trial/dysport-bracing-for-adolescent-idiopathic-scoliosis-896b1

Dysport® + Bracing for Adolescent Idiopathic Scoliosis In a study of 51 patients with adolescent idiopathic scoliosis treated with a Boston brace, 61% were successful in treatment, but 31% eventually required ...

6. trials.arthritis.org trials.arthritis.org/trials/NCT03935295

Dysport ® as an Adjunctive Treatment to Bracing in the ... This study evaluates the combined effect of botulinum toxin A (administered as Dysport® (Ipsen Pharmaceuticals)) and bracing in children with adolescent ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12225525/

Incobotulinumtoxin A and Yoga-like Isometric Exercise in ... Effect of Schroth exercises on curve characteristics and clinical outcomes in adolescent idiopathic scoliosis: Protocol for a multicentre randomised controlled ...

8. researchgate.net researchgate.net/publication/395618513_Yoga_and_Botulinum_Toxin_Reduce_Adolescent_Idiopathic_Lumbar_Scoliosis_-_A_Control_Randomized_Study

(PDF) Yoga and Botulinum Toxin Reduce Adolescent ... Background: Approximately 90% of scoliosis is adolescentidiopathic (AIS). From 10–14 years of age until 20, the spine is most vulnerable. An ...