Dysport® + Bracing for Adolescent Idiopathic Scoliosis

Recruiting in Palo Alto (17 mi)

Overseen by Varun Puvanesarajah, MD

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Recruiting

Sponsor: Johns Hopkins University

Must not be taking: Neuromuscular drugs

Disqualifiers: Congenital scoliosis, Neuromuscular scoliosis, Surgery, others

Prior Safety Data

Trial Summary

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.

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.

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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.

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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.

Trial 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

Participant Groups

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.

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.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Johns Hopkins University Baltimore, MD

Who Is Running the Clinical Trial?

Johns Hopkins University Lead Sponsor

References

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

Results of treatment of idiopathic scoliosis with the Charleston bending orthosis. [2019] The authors present a preliminary retrospective review of the treatment of 32 patients with idiopathic scoliosis with the Charleston bending thoracolumbosacral orthosis (TLSO), a new, low-profile spinal orthosis. At the onset of treatment, the patients' mean age was 12.5 years and the mean Risser stage was 0.4. Females achieved menarche at an mean of 1.8 months after starting orthotic treatment. Single structural curves were treated in all patients. At this time, 2 patients have failed treatment, their curves progressing 12 degrees and 8 degrees, respectively. An additional 11 patients have successfully completed treatment, having reached skeletal maturity with no more than 5 degrees of curve progression. Their mean curve change was a 2.2 degrees decrease. The other 19 patients remain under treatment. The Charleston bending TLSO is worn only during nighttime sleeping hours. It is well tolerated, with excellent patient compliance and low psychological stress, and it may be as successful at curve control as other orthoses. Experience with more patients and longer follow-up is needed.

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

Factors that influence outcome in bracing large curves in patients with adolescent idiopathic scoliosis. [2022] A retrospective review of 51 patients with adolescent idiopathic scoliosis (AIS) treated with a Boston brace for curves ranging from 36 degrees to 45 degrees.

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

Effectiveness of the boston brace in treatment of large curves in adolescent idiopathic scoliosis. [2022] This is a retrospective study of 50 patients with adolescent idiopathic scoliosis with curves measuring 35 degrees to 45 degrees who were treated with a Boston brace.

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

Effectiveness of TLSO bracing in the conservative treatment of idiopathic scoliosis. [2004] A clear understanding of the effectiveness of the thoracolumbosacral orthosis (TLSO) as a conservative treatment for idiopathic scoliosis is still necessary. In the past few years, the review of pertinent literature has emphasized the lack of properly matched control studies and erroneous interpretations of results due to the use of univariate analysis. Also, in a previous controlled study evaluating the bracing of idiopathic scoliosis, the researchers mixed different types of braces and patients. Therefore, their findings were not specific to any kind of orthosis. In our study, we responded to these criticisms by providing a homogeneous group of patients with a control group and by conducting a multivariate analysis to assess the effectiveness of the TLSO. All the patients at the University Pediatric Hospital Scoliosis Clinic aged 8 through 15 with initial Cobb's angle between 20 and 40 degrees and evidence of progression were assessed. All the patients who used the TLSO and showed full compliance with treatment (n = 54) were compared with a control group. The control group consisted of the patients who needed the treatment with the brace but did not use it for several reasons (n = 47). Neither group showed significant differences in sex, initial age, initial Cobb's angle, menarche, Risser sign, or curve pattern. The mean follow-up period was 3.3 years after skeletal maturity. The results were analyzed using a multivariate analysis because the natural history of scoliosis is determined by multiple factors.(ABSTRACT TRUNCATED AT 250 WORDS)

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

Effectiveness of the Rigo Chêneau versus Boston-style orthoses for adolescent idiopathic scoliosis: a retrospective study. [2020] Bracing can effectively treat adolescent idiopathic scoliosis (AIS), but patient outcomes have not been compared by brace type. We compared outcomes of AIS patients treated with Rigo Chêneau orthoses (RCOs) or custom-molded Boston-style thoracolumbosacral orthoses (TLSOs).

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] Adolescent idiopathic scoliosis (AIS) is characterized by a lateral curvature of the spine with a Cobb angle greater than 10 degrees, accompanied by rotation of the vertebral body. Bracing has been shown to be effective in halting the progression of at-risk curves, and, in some cases, even improving the Cobb angle by 6° or more. The Boston Brace 3D is part of the Boston Orthotics and Prosthetics standardized scoliosis program. The orthosis is custom-fabricated from scans, computer-aided design (CAD), and computer-aided manufactured (CAM) thoracolumbosacral orthosis used in the non-operative management of AIS.