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

Официальное название

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

Обзор

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.

Подробное описание

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

1. 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. 2. 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.

Вмешательства

• Препарат AbobotulinumtoxinA
  Paraspinal abobotulinumtoxinA injections (compared to placebo)
• Препарат Placebos
  Placebo prepared by Ipsen for use as control
• Устройство Custom Thoracolumbosacral Orthosis
  Non-operative external bracing for scoliosis curves

Первичные конечные точки

• Change in scoliotic curve as assessed by the Major Curve Cobb Angle [Срок оценки: Assessed at baseline (time 0), 6 weeks, 4 months, 4 months + 6 weeks, 8 months]

Вторичные конечные точки (1)

• Change in Patient Reported Outcomes [Срок оценки: Assessed at baseline (time 0), 6 weeks, 4 months, 4 months + 6 weeks, 8 months]

Критерии участия

Критерии включения

• 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

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 1 центр

• Johns Hopkins University — Baltimore

Публикации

• 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
• 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
• Weinstein SL. Idiopathic scoliosis. Natural history. Spine (Phila Pa 1976). 1986 Oct;11(8):780-3. doi: 10.1097/00007632-198610000-00006. PMID 3810292
• 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
• 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
• 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
• 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
• 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

Идентификаторы

NCT: NCT03935295 · IRB00110399

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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