Dysport ® as an Adjunctive Treatment to Bracing in the Management of Adolescent Idiopathic Scoliosis
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| ClinicalTrials.gov Identifier: NCT03935295 |
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Recruitment Status :
Recruiting
First Posted : May 2, 2019
Last Update Posted : April 11, 2024
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Adolescent Idiopathic Scoliosis | Drug: AbobotulinumtoxinA Drug: Placebos Device: Custom Thoracolumbosacral Orthosis | Phase 4 |
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
- 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.
- 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.
| 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 |
| Actual Study Start Date : | September 3, 2020 |
| Estimated Primary Completion Date : | December 31, 2024 |
| Estimated Study Completion Date : | March 31, 2026 |
| Arm | Intervention/treatment |
|---|---|
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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. |
Drug: AbobotulinumtoxinA
Paraspinal abobotulinumtoxinA injections (compared to placebo)
Other Name: Dysport, Ipsen Device: Custom Thoracolumbosacral Orthosis Non-operative external bracing for scoliosis curves |
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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. |
Drug: Placebos
Placebo prepared by Ipsen for use as control Device: Custom Thoracolumbosacral Orthosis Non-operative external bracing for scoliosis curves |
- 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.
- 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 10 Years to 16 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
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
| Contact: Gabrielle Reichard, MA | 4105023626 | greicha1@jhmi.edu | |
| Contact: Varun Puvanesarajah, MD | 9193605646 | vpuvane1@jhmi.edu |
| United States, Maryland | |
| Johns Hopkins University | Recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Contact: Varun Puvanesarajah, MD 410-955-3136 vpuvane1@jhmi.edu | |
| Contact: Gabrielle Reichard, MA 4105023626 greicha1@jh.edu | |
| Principal Investigator: Paul D Sponseller, MD | |
| 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 |
Publications:
| 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: | April 11, 2024 |
| Last Verified: | April 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| 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 |
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AIS adolescent idiopathic scoliosis |
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Scoliosis Spinal Curvatures Spinal Diseases Bone Diseases Musculoskeletal Diseases 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 |