E-monitoring of PULMonary Function in Patients With Duchenne Muscular Dystrophy at Home"

NCT05516745 | Unknown

• 1 other identifier: EPULMoDMD 001/2021
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 1

Brief Summary

Duchenne muscular dystrophy (DMD) is the most common, progressive, irreversible muscular dystrophy. The pulmonary function is crucial for the duration of life in this disease. The European Respiratory Society is currently focused on digital health, seeking to define the realistic innovations for digital respiratory medicine to support professionals and patients during the COVID-19 pandemic. This study aimed to investigate whether it is possible to monitor pulmonary function at home by using an individual electronical spirometry system in children with Duchenne muscular dystrophy DMD. The second aim of the study is the implementation of respiratory telerehabilitation and the assessment of its impact on pulmonary function (FVC).

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

AioCare spirometer; home spirometry; spirometry e-monitoring; pulmonary rehabilitation; digital medicine; e-health

Outcome Measures

Primary Outcomes (2)

• Change from Baseline of the mean Forced Vital Capacity in Liters measured by home and hospital spirometry in DMD participants with vs without respiratory telerehabilitation

  Some studies showed that respiratory function declines at a rate of 6-11% annually in patients with DMD. A major component of respiratory dysfunction seems to be a decline of inspiratory muscle weakness. Methods of improving the functioning of the muscles of the respiratory system are constantly sought. One of the key factors that can improve the function of respiratory muscles is proper rehabilitation. The proposition is implementation of telerehabilitation of respiratory muscle together with e-monitoring pulmonary function at home.

  12 months

• Change from Baseline of the mean Forced Vital Capacity in %predicted value measured by home and hospital spirometry in DMD participants with vs without respiratory telerehabilitation

  Some studies showed that respiratory function declines at a rate of 6-11% annually in patients with DMD. A major component of respiratory dysfunction seems to be a decline of inspiratory muscle weakness. Methods of improving the functioning of the muscles of the respiratory system are constantly sought. One of the key factors that can improve the function of respiratory muscles is proper rehabilitation. The proposition is implementation of telerehabilitation of respiratory muscle together with e-monitoring pulmonary function at home.

  12 months

Secondary Outcomes (3)

• Possibility of home e-monitoring of pulmonary function in patients with Duchenne Muscular Dystrophy in the Covid-19 pandemic

  4 weeks

• The number of the participants who performed at least one correct spirometry examination

  4 weeks

• the difference in the value of spirometry results (FVC %pv, L) between home spirometry and spirometry in the hospital

  4 weeks

Study Arms (4)

arm with intervention (AioCare spirometry)

EXPERIMENTAL

50 participants with DMD aged 7-17 years, subjected to home electronic monitoring

Device: AioCare spirometer

arm with intervention (AioCare spirometry with telerehabilitation)

EXPERIMENTAL

50 participants with DMD aged 7-17 years, subjected to home electronic monitoring with the AioCare device and pulmonary rehabilitation exercises

Device: AioCare spirometer; Other: telerehabiliation of the respiratory system

arm with intervention (telerehabilitation)

EXPERIMENTAL

50 participants with DMD aged 7-17 years, subjected to pulmonary rehabilitation exercises

Other: telerehabiliation of the respiratory system

control arm (no intervention)

NO INTERVENTION

50 participants with DMD aged 7-17 years, subject to a standard of care

Interventions

AioCare spirometer DEVICE

In the first part of the trial, all participants from arms 1 and 2 will receive the AioCare spirometer (Healtup, Poland) to home-based monitoring pulmonary function. Hospital spirometry examinations (Jaeger, Germany) evaluating disease progression will be performed periodically. There are to be follow-up visits after 3 months and after 6 months. The end-point visit is planned for 12 months.

arm with intervention (AioCare spirometry with telerehabilitation); arm with intervention (AioCare spirometry)

telerehabiliation of the respiratory system OTHER

A major component of respiratory dysfunction seems to be a decline of respiratory muscle weakness. The proposition is respiratory telerehabilitation to improve the function of respiratory muscles. Telerehabilitation includes breathing exercises for use at home. The participants will be trained in the hospital and will receive video presentations to support the exercises at home.

arm with intervention (AioCare spirometry with telerehabilitation); arm with intervention (telerehabilitation)

Eligibility Criteria

• Age: 7 Years - 17 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• male, ≥7 years and \<18 years of age at the time of enrollment in the study;
• ability to perform spirometry;
• stated willingness to comply with all study procedures and availability for the duration of the study.

You may not qualify if:

• no consent to participate in the study;
• patients under 7 years of age or above 18 years of age;
• inability to perform spirometry

Sponsors & Collaborators

• Medical University of Gdansk: lead

Study Sites (1)

Medical University

Gdansk, 80-292, Poland

RECRUITING

Related Publications (7)

• Birnkrant DJ, Bushby K, Bann CM, Apkon SD, Blackwell A, Brumbaugh D, Case LE, Clemens PR, Hadjiyannakis S, Pandya S, Street N, Tomezsko J, Wagner KR, Ward LM, Weber DR; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018 Mar;17(3):251-267. doi: 10.1016/S1474-4422(18)30024-3. Epub 2018 Feb 3.

  PMID: 29395989 RESULT

• Birnkrant DJ, Bushby K, Bann CM, Alman BA, Apkon SD, Blackwell A, Case LE, Cripe L, Hadjiyannakis S, Olson AK, Sheehan DW, Bolen J, Weber DR, Ward LM; DMD Care Considerations Working Group. Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management. Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. Epub 2018 Feb 3.

  PMID: 29395990 RESULT

• Finder JD, Birnkrant D, Carl J, Farber HJ, Gozal D, Iannaccone ST, Kovesi T, Kravitz RM, Panitch H, Schramm C, Schroth M, Sharma G, Sievers L, Silvestri JM, Sterni L; American Thoracic Society. Respiratory care of the patient with Duchenne muscular dystrophy: ATS consensus statement. Am J Respir Crit Care Med. 2004 Aug 15;170(4):456-65. doi: 10.1164/rccm.200307-885ST. No abstract available.

  PMID: 15302625 RESULT

• Phillips MF, Quinlivan RC, Edwards RH, Calverley PM. Changes in spirometry over time as a prognostic marker in patients with Duchenne muscular dystrophy. Am J Respir Crit Care Med. 2001 Dec 15;164(12):2191-4. doi: 10.1164/ajrccm.164.12.2103052.

  PMID: 11751186 RESULT

• Toussaint M, Chatwin M, Soudon P. Mechanical ventilation in Duchenne patients with chronic respiratory insufficiency: clinical implications of 20 years published experience. Chron Respir Dis. 2007;4(3):167-77. doi: 10.1177/1479972307080697.

  PMID: 17711917 RESULT

• LoMauro A, D'Angelo MG, Aliverti A. Sleep Disordered Breathing in Duchenne Muscular Dystrophy. Curr Neurol Neurosci Rep. 2017 May;17(5):44. doi: 10.1007/s11910-017-0750-1.

  PMID: 28397169 RESULT

• Miller MR, Hankinson J, Brusasco V, Burgos F, Casaburi R, Coates A, Crapo R, Enright P, van der Grinten CP, Gustafsson P, Jensen R, Johnson DC, MacIntyre N, McKay R, Navajas D, Pedersen OF, Pellegrino R, Viegi G, Wanger J; ATS/ERS Task Force. Standardisation of spirometry. Eur Respir J. 2005 Aug;26(2):319-38. doi: 10.1183/09031936.05.00034805. No abstract available.

  PMID: 16055882 RESULT

Related Links

• AioCare description

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Eliza Wasilewska, MD,PhD

  Medical University Gdansk

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Eliza Wasilewska, MD,PhD

CONTACT

+48 56 349 2625; ewasilewska@gumed.edu.pl

Eliza Wasilewska, MD,PhD

CONTACT

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 4 arms - 1. arm - AioCare device spirometry 2. arm - AioCare device spirometry+ telerehabilitation of the respiratory system 3. arm - telerehabilitation of the respiratory system 4. arm - control arm - standard of care (no monitoring, no telerehabilitation)

Study Record Dates

• First Submitted: September 22, 2021
• First Posted: August 26, 2022
• Study Start: March 20, 2021
• Primary Completion: February 1, 2025
• Study Completion: March 1, 2025
• Last Updated: August 26, 2022

Record last verified: 2022-08

Locations

PL (1)