Rett Syndrome Clinical Trial
Official title:
An Intensive Anti-Scoliosis Postural Intervention Supported by an Application for Individuals With Rett Syndrome
Verified date | January 2024 |
Source | Ariel University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Background: Scoliosis is the most common orthopedic comorbidity in Rett syndrome (RTT), with a prevalence of 94% and a mean curve progression of 14-21° Cobb annually. A scoliosis prevention intervention based on daily activity programs was proposed for people with RTT within uncontrolled study designs. Aim: The current study aims to evaluate the effectiveness of a home-based activity program carried out during daily life to slow the progression of scoliosis in girls with RTT. Ethics: The proposal was approved by Ariel university IRB. All participants' parents will sign informed consent forms. Participants: Twenty Italian girls aged between six and 16 years with a genetically confirmed classic RTT and scoliosis at a severity level between 10° and 40° Cobb will be recruited and randomly divided into two groups (immediate intervention - Group 1; wait-list-intervention - Group 2). Both groups will follow the same 10-month intervention program, 10 months apart. Outcome measures: Participants' scoliosis Cobb's angle, motor functioning, and behavioral characteristics will be assessed three times. Procedure: Each participant will be evaluated three times: at T1, T2, and T3. Participants in the Group 1 will conduct the intervention for 10 months between T0 and T1. Group 2 will perform the intervention between T1 and T2. The interventions will comprise daily home-based activity programs carried out by participants' caregivers within everyday living environments. An expert therapist will remotely supervise each program through an ad hoc developed smartphone application. Specific strategies that will be implemented during the intervention will include the maintenance of asymmetrical postures that oppose the scoliosis curve during activities and exercises in sitting, standing, and walking positions (according to each participant's functional abilities). These strategies refer to a hypercorrective postural positioning of scoliosis. In addition, activities involving weight bearing on the lower limbs, such as walking and standing for at least two hours a day, will be encouraged, and passive stretching and spinal mobilization exercises will be conducted.
Status | Completed |
Enrollment | 20 |
Est. completion date | September 30, 2023 |
Est. primary completion date | September 30, 2023 |
Accepts healthy volunteers | No |
Gender | Female |
Age group | 6 Years to 16 Years |
Eligibility | Inclusion Criteria: - Diagnosis of RTT with confirmed MECP2 gene mutation; - Diagnosis of unstructured flexible scoliosis (Cobb angle between 10° and 40°) measured radiologically with an x-ray performed no more than six months before the recruitment; - Age between 6 and 16 years. Exclusion Criteria: - Presence of psychomotor developmental deficit evidenced in the first six months of life or diseases of neurometabolic, infectious, or secondary brain damage trauma origin; - Previous surgical intervention to the spine or its planning within the study period; - Use of a corset during most waking hours; - Clinical judgment of the evaluating specialist doctor who certifies unstable health conditions that are not compatible with the performance of the rehabilitation program (e.g., ongoing or recurrent infections, severe gastrointestinal disorders, drug-resistant epilepsy with multi-day seizures). - Clinical judgment of the research team suggesting an inability of the home environment to perform the intervention program (change in residence, a planned pregnancy, a pre-planned complex surgical/medical procedure within the period of the planned intervention program for the child or family member) within the intervention period. |
Country | Name | City | State |
---|---|---|---|
Italy | Centro AIRETT Ricerca e Innovazione (CARI) | Verona |
Lead Sponsor | Collaborator |
---|---|
Ariel University | ASST Santi Paolo e Carlo |
Italy,
Ager S, Downs J, Fyfe S, Leonard H. Parental experiences of scoliosis management in Rett syndrome. Disabil Rehabil. 2009;31(23):1917-24. doi: 10.1080/09638280902846392. — View Citation
Ager S, Fyfe S, Christodoulou J, Jacoby P, Schmitt L, Leonard H. Predictors of scoliosis in Rett syndrome. J Child Neurol. 2006 Sep;21(9):809-13. doi: 10.1177/08830738060210091501. — View Citation
Amir RE, Van den Veyver IB, Wan M, Tran CQ, Francke U, Zoghbi HY. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat Genet. 1999 Oct;23(2):185-8. doi: 10.1038/13810. — View Citation
Armstrong RA. When to use the Bonferroni correction. Ophthalmic Physiol Opt. 2014 Sep;34(5):502-8. doi: 10.1111/opo.12131. Epub 2014 Apr 2. — View Citation
Barney CC, Merbler AM, Quest K, Byiers BJ, Wilcox GL, Schwantes S, Roiko SA, Feyma T, Beisang A, Symons FJ. A case-controlled comparison of postoperative analgesic dosing between girls with Rett syndrome and girls with and without developmental disability undergoing spinal fusion surgery. Paediatr Anaesth. 2017 Mar;27(3):290-299. doi: 10.1111/pan.13066. Epub 2017 Feb 8. — View Citation
Bassett GS, Tolo VT. The incidence and natural history of scoliosis in Rett syndrome. Dev Med Child Neurol. 1990 Nov;32(11):963-6. doi: 10.1111/j.1469-8749.1990.tb08118.x. — View Citation
Downs J, Bergman A, Carter P, Anderson A, Palmer GM, Roye D, van Bosse H, Bebbington A, Larsson EL, Smith BG, Baikie G, Fyfe S, Leonard H. Guidelines for management of scoliosis in Rett syndrome patients based on expert consensus and clinical evidence. Spine (Phila Pa 1976). 2009 Aug 1;34(17):E607-17. doi: 10.1097/BRS.0b013e3181a95ca4. — View Citation
Downs J, Torode I, Ellaway C, Jacoby P, Bunting C, Wong K, Christodoulou J, Leonard H. Family satisfaction following spinal fusion in Rett syndrome. Dev Neurorehabil. 2016;19(1):31-7. doi: 10.3109/17518423.2014.898107. Epub 2014 Apr 11. — View Citation
Downs J, Torode I, Wong K, Ellaway C, Elliott EJ, Christodoulou J, Jacoby P, Thomson MR, Izatt MT, Askin GN, McPhee BI, Bridge C, Cundy P, Leonard H. The Natural History of Scoliosis in Females With Rett Syndrome. Spine (Phila Pa 1976). 2016 May;41(10):856-63. doi: 10.1097/BRS.0000000000001399. — View Citation
Downs J, Torode I, Wong K, Ellaway C, Elliott EJ, Izatt MT, Askin GN, Mcphee BI, Cundy P, Leonard H; Rett syndrome spinal fusion group. Surgical fusion of early onset severe scoliosis increases survival in Rett syndrome: a cohort study. Dev Med Child Neurol. 2016 Jun;58(6):632-8. doi: 10.1111/dmcn.12984. Epub 2015 Dec 11. — View Citation
Downs J, Young D, de Klerk N, Bebbington A, Baikie G, Leonard H. Impact of scoliosis surgery on activities of daily living in females with Rett syndrome. J Pediatr Orthop. 2009 Jun;29(4):369-74. doi: 10.1097/BPO.0b013e3181a53b41. — View Citation
Fabio RA, Martinazzoli C, Antonietti A. Development and standardization of the "RARS"(Rett Assessment Rating Scale). Life Span Disabil 2005; 8(2): 257-81.
Ferrari A, Ferrara C, Balugani M, Sassi S. Severe scoliosis in neurodevelopmental disabilities: clinical signs and therapeutic proposals. Eur J Phys Rehabil Med. 2010 Dec;46(4):563-80. — View Citation
Fombonne E, Simmons H, Ford T, Meltzer H, Goodman R. Prevalence of pervasive developmental disorders in the British nationwide survey of child mental health. Int Rev Psychiatry. 2003 Feb-May;15(1-2):158-65. doi: 10.1080/0954026021000046119. — View Citation
Hagberg B, Witt-Engerstrom I. Rett syndrome: a suggested staging system for describing impairment profile with increasing age towards adolescence. Am J Med Genet Suppl. 1986;1:47-59. doi: 10.1002/ajmg.1320250506. — View Citation
Haleem S, Nnadi C. Scoliosis: a review. Paediatr Child Health (Oxford) 2018; 28(5): 209-17. doi:10.1016/j.paed.2018.03.007.
Harrison DJ, Webb PJ. Scoliosis in the Rett syndrome: natural history and treatment. Brain Dev. 1990;12(1):154-6. doi: 10.1016/s0387-7604(12)80200-2. — View Citation
Huang TJ, Lubicky JP, Hammerberg KW. Scoliosis in Rett syndrome. Orthop Rev. 1994 Dec;23(12):931-7. — View Citation
Karimi MT, Rabczuk T. Scoliosis conservative treatment: A review of literature. J Craniovertebr Junction Spine. 2018 Jan-Mar;9(1):3-8. doi: 10.4103/jcvjs.JCVJS_39_17. — View Citation
Kerr AM, Webb P, Prescott RJ, Milne Y. Results of surgery for scoliosis in Rett syndrome. J Child Neurol. 2003 Oct;18(10):703-8. doi: 10.1177/08830738030180101201. — View Citation
Killian JT, Lane JB, Lee HS, Skinner SA, Kaufmann WE, Glaze DG, Neul JL, Percy AK. Scoliosis in Rett Syndrome: Progression, Comorbidities, and Predictors. Pediatr Neurol. 2017 May;70:20-25. doi: 10.1016/j.pediatrneurol.2017.01.032. Epub 2017 Feb 7. — View Citation
Kotwicki T, Jozwiak M. Conservative management of neuromuscular scoliosis: personal experience and review of literature. Disabil Rehabil. 2008;30(10):792-8. doi: 10.1080/09638280801889584. — View Citation
Larsson EL, Aaro S, Ahlinder P, Normelli H, Tropp H, Oberg B. Long-term follow-up of functioning after spinal surgery in patients with Rett syndrome. Eur Spine J. 2009 Apr;18(4):506-11. doi: 10.1007/s00586-008-0876-6. Epub 2009 Jan 23. — View Citation
Lidstrom J, Stokland E, Hagberg B. Scoliosis in Rett syndrome. Clinical and biological aspects. Spine (Phila Pa 1976). 1994 Jul 15;19(14):1632-5. doi: 10.1097/00007632-199407001-00013. — View Citation
Logan SW, Huang HH, Stahlin K, Galloway JC. Modified ride-on car for mobility and socialization: single-case study of an infant with Down syndrome. Pediatr Phys Ther. 2014 Winter;26(4):418-26. doi: 10.1097/PEP.0000000000000070. — View Citation
Lotan M, Downs J, Elefant C. A Pilot Study Delivering Physiotherapy Support for Rett Syndrome Using a Telehealth Framework Suitable for COVID-19 Lockdown. Dev Neurorehabil. 2021 Aug;24(6):429-434. doi: 10.1080/17518423.2021.1914762. Epub 2021 Apr 15. — View Citation
Lotan M, Ippolito E, Favetta M, Romano A. Skype Supervised, Individualized, Home-Based Rehabilitation Programs for Individuals With Rett Syndrome and Their Families - Parental Satisfaction and Point of View. Front Psychol. 2021 Sep 16;12:720927. doi: 10.3389/fpsyg.2021.720927. eCollection 2021. — View Citation
Lotan M, Merrick J, Carmeli E. Managing scoliosis in a young child with Rett syndrome: a case study. ScientificWorldJournal. 2005 Mar 29;5:264-73. doi: 10.1100/tsw.2005.33. — View Citation
Mehta J, Gibson M. The treatment of neuromuscular scoliosis. Current Orthopaedics. 2003; 17(4): 313-21. doi:10.1016/S0268-0890(03)00002-1.
Mount RH, Charman T, Hastings RP, Reilly S, Cass H. The Rett Syndrome Behaviour Questionnaire (RSBQ): refining the behavioural phenotype of Rett syndrome. J Child Psychol Psychiatry. 2002 Nov;43(8):1099-110. doi: 10.1111/1469-7610.00236. — View Citation
Neul JL, Fang P, Barrish J, Lane J, Caeg EB, Smith EO, Zoghbi H, Percy A, Glaze DG. Specific mutations in methyl-CpG-binding protein 2 confer different severity in Rett syndrome. Neurology. 2008 Apr 15;70(16):1313-21. doi: 10.1212/01.wnl.0000291011.54508.aa. Epub 2008 Mar 12. — View Citation
Neul JL, Kaufmann WE, Glaze DG, Christodoulou J, Clarke AJ, Bahi-Buisson N, Leonard H, Bailey ME, Schanen NC, Zappella M, Renieri A, Huppke P, Percy AK; RettSearch Consortium. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010 Dec;68(6):944-50. doi: 10.1002/ana.22124. — View Citation
Olafsson Y, Saraste H, Al-Dabbagh Z. Brace treatment in neuromuscular spine deformity. J Pediatr Orthop. 1999 May-Jun;19(3):376-9. — View Citation
Percy AK, Lee HS, Neul JL, Lane JB, Skinner SA, Geerts SP, Annese F, Graham J, McNair L, Motil KJ, Barrish JO, Glaze DG. Profiling scoliosis in Rett syndrome. Pediatr Res. 2010 Apr;67(4):435-9. doi: 10.1203/PDR.0b013e3181d0187f. — View Citation
Pini G, Milan M, Zappella M. Rett syndrome in northern Tuscany (Italy): family tree studies. Clin Genet. 1996 Dec;50(6):486-90. doi: 10.1111/j.1399-0004.1996.tb02718.x. — View Citation
Riise R, Brox JI, Sorensen R, Skjeldal OH. Spinal deformity and disability in patients with Rett syndrome. Dev Med Child Neurol. 2011 Jul;53(7):653-7. doi: 10.1111/j.1469-8749.2011.03935.x. Epub 2011 Apr 18. — View Citation
Roberts SB, Tsirikos AI. Factors influencing the evaluation and management of neuromuscular scoliosis: A review of the literature. J Back Musculoskelet Rehabil. 2016 Nov 21;29(4):613-623. doi: 10.3233/BMR-160675. — View Citation
Rocos B, Zeller R. Correcting Scoliosis in Rett Syndrome. Cureus. 2021 Jun 3;13(6):e15411. doi: 10.7759/cureus.15411. eCollection 2021 Jun. — View Citation
Rodocanachi Roidi ML, Isaias IU, Cozzi F, Grange F, Scotti FM, Gestra VF, Gandini A, Ripamonti E. A New Scale to Evaluate Motor Function in Rett Syndrome: Validation and Psychometric Properties. Pediatr Neurol. 2019 Nov;100:80-86. doi: 10.1016/j.pediatrneurol.2019.03.005. Epub 2019 Mar 16. — View Citation
Rodocanachi Roidi ML, Isaias IU, Cozzi F, Grange F, Scotti FM, Gestra VF, Gandini A, Ripamonti E. Motor function in Rett syndrome: comparing clinical and parental assessments. Dev Med Child Neurol. 2019 Aug;61(8):957-963. doi: 10.1111/dmcn.14109. Epub 2018 Nov 26. — View Citation
Romano A, Capri T, Semino M, Bizzego I, Di Rosa G, Fabio RA. Gross Motor, Physical Activity and Musculoskeletal Disorder Evaluation Tools for Rett Syndrome: A Systematic Review. Dev Neurorehabil. 2020 Nov;23(8):485-501. doi: 10.1080/17518423.2019.1680761. Epub 2019 Oct 31. — View Citation
Romano A, Di Rosa G, Tisano A, Fabio RA, Lotan M. Effects of a remotely supervised motor rehabilitation program for individuals with Rett syndrome at home. Disabil Rehabil. 2022 Oct;44(20):5898-5908. doi: 10.1080/09638288.2021.1949398. Epub 2021 Jul 20. — View Citation
Romano A, Ippolito E, Risoli C, Malerba E, Favetta M, Sancesario A, Lotan M, Moran DS. Intensive Postural and Motor Activity Program Reduces Scoliosis Progression in People with Rett Syndrome. J Clin Med. 2022 Jan 22;11(3):559. doi: 10.3390/jcm11030559. — View Citation
Skjeldal OH, von Tetzchner S, Aspelund F, Herder GA, Lofterld B. Rett syndrome: geographic variation in prevalence in Norway. Brain Dev. 1997 Jun;19(4):258-61. doi: 10.1016/s0387-7604(97)00572-x. — View Citation
SOSORT guideline committee; Weiss HR, Negrini S, Rigo M, Kotwicki T, Hawes MC, Grivas TB, Maruyama T, Landauer F. Indications for conservative management of scoliosis (guidelines). Scoliosis. 2006 May 8;1:5. doi: 10.1186/1748-7161-1-5. — View Citation
Westerlund LE, Gill SS, Jarosz TS, Abel MF, Blanco JS. Posterior-only unit rod instrumentation and fusion for neuromuscular scoliosis. Spine (Phila Pa 1976). 2001 Sep 15;26(18):1984-9. doi: 10.1097/00007632-200109150-00008. — View Citation
* Note: There are 46 references in all — Click here to view all references
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Scoliosis progression | The progression of scoliosis will be assessed for each participant by measuring the differences between Cobb's angle measured on three anteroposterior X-rays of the spine collected before and after the intervention of each group. The Cobb's angle will be obtained from the average value of three measurements carried out by independent blinded (to the treatment status of each subject) medical specialists with clinical experience concerning scoliosis in Rett syndrome. | Three days 10 months apart from each other, before the start of the Group 1 intervention (T1), before the start of the Group 2 intervention (T2), and at the end of the Group 2 intervention (T3). | |
Primary | Adherence to the Program | Adherence to the Program refers to the amount of treatment conducted by the participants compared to the total amount of treatment established in their program. It will be measured by the ad hoc developed smartphone application which will record the percentage of the performance of each activity foreseen in the programs. | 10 months during the Group 1 intervention phase. | |
Primary | Adherence to the Program | Adherence to the Program refers to the amount of treatment conducted by the participants compared to the total amount of treatment established in their program. It will be measured by the ad hoc developed smartphone application which will record the percentage of the performance of each activity foreseen in the programs. | 10 months during the Group 2 intervention phase. | |
Secondary | Gross Motor Function Level | The gross motor function level, including the ability to walk, will be measured for each participant during the rehabilitation assessments using the Rett Syndrome Motor Evaluation Scale. This scale includes 25 items that examine motor function divided into six sections: standing, sitting, postural transfers, walking, running, and climbing / descending stairs. Sixteen items are rated on a discrete scale, ranging from 0 (indicates no or very mild impairment) and 4 (severe impairment). Nine items related to walking skills are evaluated on a discrete scale with a score from 0 to 2 where 0 indicates that the activity could always be completed by the subject and 2 that is assigned if the item cannot be carried out. The items' scores are added together, thus giving a total RESMES score (range: 0 to 82). Moreover, a score for each section can be calculated. | 30 minutes, before the beginning of the Group 1 intervention. | |
Secondary | Gross Motor Function Level | The gross motor function level, including the ability to walk, will be measured for each participant during the rehabilitation assessments using the Rett Syndrome Motor Evaluation Scale. This scale includes 25 items that examine motor function divided into six sections: standing, sitting, postural transfers, walking, running, and climbing / descending stairs. Sixteen items are rated on a discrete scale, ranging from 0 (indicates no or very mild impairment) and 4 (severe impairment). Nine items related to walking skills are evaluated on a discrete scale with a score from 0 to 2 where 0 indicates that the activity could always be completed by the subject and 2 that is assigned if the item cannot be carried out. The items' scores are added together, thus giving a total RESMES score (range: 0 to 82). Moreover, a score for each section can be calculated. | 30 minutes, before the beginning of the Group 2 intervention. | |
Secondary | Gross Motor Function Level | The gross motor function level, including the ability to walk, will be measured for each participant during the rehabilitation assessments using the Rett Syndrome Motor Evaluation Scale. This scale includes 25 items that examine motor function divided into six sections: standing, sitting, postural transfers, walking, running, and climbing / descending stairs. Sixteen items are rated on a discrete scale, ranging from 0 (indicates no or very mild impairment) and 4 (severe impairment). Nine items related to walking skills are evaluated on a discrete scale with a score from 0 to 2 where 0 indicates that the activity could always be completed by the subject and 2 that is assigned if the item cannot be carried out. The items' scores are added together, thus giving a total RESMES score (range: 0 to 82). Moreover, a score for each section can be calculated. | 30 minutes, at the end of the Group 2 intervention. | |
Secondary | Behavioral Characteristics | The behavioral characteristics of the participants will be evaluated through the Rett Syndrome Behavior Questionnaire (RSBQ) administration. The RSBQ is a questionnaire created by comparing the behavioral aspects of people with Rett syndrome with those of individuals with severe intellectual disability. The questionnaire is filled in by parents who are asked to provide an evaluation of the behavioral characteristics shown by their child at the time of completion. The RSBQ includes elements describing behavioral characteristics and assessing physical capabilities, including hand function, sitting, and walking skills. Other items evaluated by the questionnaire include sleep disturbances, respiratory difficulties, mood disturbances, self-harm, social skills, and stereotyped hand movements. The RSBQ comprises 45 items scored on a three-point Likert scale (0 - Not true; 2 - Very or often true), providing a total score between 0 and 90 points. | 15 minutes, before the beginning of the Group 1 intervention. | |
Secondary | Behavioral Characteristics | The behavioral characteristics of the participants will be evaluated through the Rett Syndrome Behavior Questionnaire (RSBQ) administration. The RSBQ is a questionnaire created by comparing the behavioral aspects of people with Rett syndrome with those of individuals with severe intellectual disability. The questionnaire is filled in by parents who are asked to provide an evaluation of the behavioral characteristics shown by their child at the time of completion. The RSBQ includes elements describing behavioral characteristics and assessing physical capabilities, including hand function, sitting, and walking skills. Other items evaluated by the questionnaire include sleep disturbances, respiratory difficulties, mood disturbances, self-harm, social skills, and stereotyped hand movements. The RSBQ comprises 45 items scored on a three-point Likert scale (0 - Not true; 2 - Very or often true), providing a total score between 0 and 90 points. | 15 minutes, before the beginning of the Group 2 intervention. | |
Secondary | Behavioral Characteristics | The behavioral characteristics of the participants will be evaluated through the Rett Syndrome Behavior Questionnaire (RSBQ) administration. The RSBQ is a questionnaire created by comparing the behavioral aspects of people with Rett syndrome with those of individuals with severe intellectual disability. The questionnaire is filled in by parents who are asked to provide an evaluation of the behavioral characteristics shown by their child at the time of completion. The RSBQ includes elements describing behavioral characteristics and assessing physical capabilities, including hand function, sitting, and walking skills. Other items evaluated by the questionnaire include sleep disturbances, respiratory difficulties, mood disturbances, self-harm, social skills, and stereotyped hand movements. The RSBQ comprises 45 items scored on a three-point Likert scale (0 - Not true; 2 - Very or often true), providing a total score between 0 and 90 points. | 15 minutes, at the end of the Group 2 intervention. | |
Secondary | Caregivers' Burden | The burden related to the implementation of the treatment will be assessed through the administration, at the of each intervention phase, of an ad hoc developed questionnaire to all parents, therapists, teachers, and other caregivers involved in the intervention. The items included in this questionnaire will address the burden derived from the treatment implementation, the parental satisfaction, and the intervention's perceived efficacy concerning functional skills and the trunk and spine posture. The questionnaire comprises 10 items scored on a five-point Likert scale (0 - Completely disagree; 4 - Completely agree), providing a total score between 0 and 50 points. | 15 minutes, at the end of the Group 1 intervention. | |
Secondary | Caregivers' Burden | The burden related to the implementation of the treatment will be assessed through the administration, at the of each intervention phase, of an ad hoc developed questionnaire to all parents, therapists, teachers, and other caregivers involved in the intervention. The items included in this questionnaire will address the burden derived from the treatment implementation, the parental satisfaction, and the intervention's perceived efficacy concerning functional skills and the trunk and spine posture. The questionnaire comprises 10 items scored on a five-point Likert scale (0 - Completely disagree; 4 - Completely agree), providing a total score between 0 and 50 points. | 15 minutes, at the end of the Group 2 intervention. |
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