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

NCT03935295 | Recruiting Phase 4 FDA Drug

• 1 other identifier: IRB00110399
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Adolescent Idiopathic Scoliosis

Keywords

AIS; adolescent idiopathic scoliosis

Outcome Measures

Primary Outcomes (1)

• Change in scoliotic curve as assessed by the Major Curve Cobb Angle

  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.

  Assessed at baseline (time 0), 6 weeks, 4 months, 4 months + 6 weeks, 8 months

Secondary Outcomes (1)

• Change in Patient Reported Outcomes

  Assessed at baseline (time 0), 6 weeks, 4 months, 4 months + 6 weeks, 8 months

Study Arms (2)

Botulinum Toxin

EXPERIMENTAL

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; Device: Custom Thoracolumbosacral Orthosis

Placebo

PLACEBO COMPARATOR

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; Device: Custom Thoracolumbosacral Orthosis

Interventions

AbobotulinumtoxinA DRUG

Paraspinal abobotulinumtoxinA injections (compared to placebo)

Also known as: Dysport, Ipsen

Botulinum Toxin

Placebos DRUG

Placebo prepared by Ipsen for use as control

Placebo

Custom Thoracolumbosacral Orthosis DEVICE

Non-operative external bracing for scoliosis curves

Botulinum Toxin; Placebo

Eligibility Criteria

• Age: 10 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Johns Hopkins University: lead

Study Sites (1)

Johns Hopkins University

Baltimore, Maryland, 21287, United States

RECRUITING

Related Publications (15)

• Hresko MT. Clinical practice. Idiopathic scoliosis in adolescents. N Engl J Med. 2013 Feb 28;368(9):834-41. doi: 10.1056/NEJMcp1209063. No abstract available.

  PMID: 23445094 BACKGROUND

• Asher MA, Burton DC. Adolescent idiopathic scoliosis: natural history and long term treatment effects. Scoliosis. 2006 Mar 31;1(1):2. doi: 10.1186/1748-7161-1-2.

  PMID: 16759428 BACKGROUND

• Weinstein SL. Idiopathic scoliosis. Natural history. Spine (Phila Pa 1976). 1986 Oct;11(8):780-3. doi: 10.1097/00007632-198610000-00006.

  PMID: 3810292 BACKGROUND

• Weinstein SL, Dolan LA. The Evidence Base for the Prognosis and Treatment of Adolescent Idiopathic Scoliosis: The 2015 Orthopaedic Research and Education Foundation Clinical Research Award. J Bone Joint Surg Am. 2015 Nov 18;97(22):1899-903. doi: 10.2106/JBJS.O.00330. No abstract available.

  PMID: 26582623 BACKGROUND

• Weinstein SL, Dolan LA, Wright JG, Dobbs MB. Effects of bracing in adolescents with idiopathic scoliosis. N Engl J Med. 2013 Oct 17;369(16):1512-21. doi: 10.1056/NEJMoa1307337. Epub 2013 Sep 19.

  PMID: 24047455 BACKGROUND

• Nuzzo RM, Walsh S, Boucherit T, Massood S. Counterparalysis for treatment of paralytic scoliosis with botulinum toxin type A. Am J Orthop (Belle Mead NJ). 1997 Mar;26(3):201-7.

  PMID: 9240788 BACKGROUND

• Wong C, Gosvig K, Sonne-Holm S. The role of the paravertebral muscles in adolescent idiopathic scoliosis evaluated by temporary paralysis. Scoliosis Spinal Disord. 2017 Oct 10;12:33. doi: 10.1186/s13013-017-0138-7. eCollection 2017.

  PMID: 29046898 BACKGROUND

• Kouwenhoven JW, Castelein RM. The pathogenesis of adolescent idiopathic scoliosis: review of the literature. Spine (Phila Pa 1976). 2008 Dec 15;33(26):2898-908. doi: 10.1097/BRS.0b013e3181891751.

  PMID: 19092622 BACKGROUND

• Wong C. Mechanism of right thoracic adolescent idiopathic scoliosis at risk for progression; a unifying pathway of development by normal growth and imbalance. Scoliosis. 2015 Jan 27;10:2. doi: 10.1186/s13013-015-0030-2. eCollection 2015.

  PMID: 25657814 BACKGROUND

• RIDDLE HF, ROAF R. Muscle imbalance in the causation of scoliosis. Lancet. 1955 Jun 18;268(6877):1245-7. doi: 10.1016/s0140-6736(55)91020-5. No abstract available.

  PMID: 14382561 BACKGROUND

• Fidler MW, Jowett RL. Muscle imbalance in the aetiology of scoliosis. J Bone Joint Surg Br. 1976 May;58(2):200-1. doi: 10.1302/0301-620X.58B2.932082.

  PMID: 932082 BACKGROUND

• Asher M, Min Lai S, Burton D, Manna B. The reliability and concurrent validity of the scoliosis research society-22 patient questionnaire for idiopathic scoliosis. Spine (Phila Pa 1976). 2003 Jan 1;28(1):63-9. doi: 10.1097/00007632-200301010-00015.

  PMID: 12544958 BACKGROUND

• Cheung KM, Cheng EY, Chan SC, Yeung KW, Luk KD. Outcome assessment of bracing in adolescent idiopathic scoliosis by the use of the SRS-22 questionnaire. Int Orthop. 2007 Aug;31(4):507-11. doi: 10.1007/s00264-006-0209-5. Epub 2006 Aug 1.

  PMID: 16896864 BACKGROUND

• Schwieger T, Campo S, Weinstein SL, Dolan LA, Ashida S, Steuber KR. Body Image and Quality-of-Life in Untreated Versus Brace-Treated Females With Adolescent Idiopathic Scoliosis. Spine (Phila Pa 1976). 2016 Feb;41(4):311-9. doi: 10.1097/BRS.0000000000001210.

  PMID: 26555827 BACKGROUND

• Wong C, Pedersen SA, Kristensen BB, Gosvig K, Sonne-Holm S. The Effect of Botulinum Toxin A Injections in the Spine Muscles for Cerebral Palsy Scoliosis, Examined in a Prospective, Randomized Triple-blinded Study. Spine (Phila Pa 1976). 2015 Dec;40(23):E1205-11. doi: 10.1097/BRS.0000000000001049.

  PMID: 26165216 BACKGROUND

MeSH Terms

Interventions

abobotulinumtoxinA

Study Officials

• Paul Sponseller, MD, MBA

  Johns Hopkins Hospital Department of Orthopaedic Surgery

  PRINCIPAL INVESTIGATOR

• Varun Puvanesarajah, MD

  Johns Hopkins Hospital Department of Orthopaedic Surgery

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gabrielle Reichard, MA

CONTACT

4105023626; greicha1@jhmi.edu

Varun Puvanesarajah, MD

CONTACT

9193605646; vpuvane1@jhmi.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 30, 2019
• First Posted: May 2, 2019
• Study Start: September 3, 2020
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: December 9, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)