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

Trial Summary

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 data supports the effectiveness of the treatment for adolescent idiopathic scoliosis?

Research shows that using a thoracolumbosacral orthosis (TLSO), a type of back brace, can help control the curve progression in adolescent idiopathic scoliosis. In one study, patients who wore the TLSO showed a decrease in curve progression, with some achieving skeletal maturity with minimal curve changes. ^{1 2 3 4 5}

How is the Dysport® + Bracing treatment for adolescent idiopathic scoliosis different from other treatments?

Dysport® + Bracing is unique because it combines a drug, Dysport® (a type of botulinum toxin used to relax muscles), with a custom thoracolumbosacral orthosis (a back brace) to treat adolescent idiopathic scoliosis. This approach is novel as it integrates muscle relaxation with physical support to potentially enhance the effectiveness of scoliosis management. ^{1 2 3 5 6}

What is the purpose of this trial?

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.

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

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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 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 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 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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Results of treatment of idiopathic scoliosis with the Charleston bending orthosis. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Factors that influence outcome in bracing large curves in patients with adolescent idiopathic scoliosis. [2022]

3. United States pubmed.ncbi.nlm.nih.gov

Effectiveness of the boston brace in treatment of large curves in adolescent idiopathic scoliosis. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Effectiveness of TLSO bracing in the conservative treatment of idiopathic scoliosis. [2004]

5. England pubmed.ncbi.nlm.nih.gov

Effectiveness of the Rigo Chêneau versus Boston-style orthoses for adolescent idiopathic scoliosis: a retrospective study. [2020]

6. Switzerland pubmed.ncbi.nlm.nih.gov

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