Neuromuscular DiseasesIAPVNCT05346263
Efficacy of Intermittent Abdominal Pressure Ventilation in Neuromuscular Patients
A study testing Intermittent Abdominal Pressure Ventilation and Usual ventilation in people with Neuromuscular Diseases, run by Fondazione Don Carlo Gnocchi ETS at 1 site in Italy.
Trial summary
Non-Invasive Ventilation (NIV) is an established treatment to manage respiratory muscles dysfunction in neuromuscular disease, preventing the progression of respiratory failure to intubation and/or a tracheotomy. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Neuromuscular Diseases
- What is being tested?
- Intermittent Abdominal Pressure Ventilation and Usual ventilation
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 1 siteItaly
- Study dates
- 1 Mar 2022to 1 Mar 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of Neuromuscular Disease (Amyotrophic Lateral Sclerosis, Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Pompe Disease)
- Non Invasive Ventilation > 16 hours/day
- Informed consent signed
Who cannot join
- Diagnosis of kyphoscoliosis
Trial locations and contacts
1 site in 1 country.
Valeria Volpi, PT contact
Italy1 site, 1 recruiting
- Recruiting
IRCCS S. Maria Nascente - Fondazione Don Carlo Gnocchi
Milan, 20146
Paolo Banfi, MD, +390240308812, pabanfi@dongnocchi.it
About this study
Official title: Efficacy of Intermittent Abdominal Pressure Ventilation in Neuromuscular Patients: Multicentre, Randomised Controlled Trial
Non-Invasive Ventilation (NIV) is an established treatment to manage respiratory muscles dysfunction in neuromuscular disease, preventing the progression of respiratory failure to intubation and/or a tracheotomy. NIV is commonly needed at first during the night, but when the disease worsens, it is required during the day. It is provided via nasal or oronasal masks, causing discomfort and/or aesthetic issues that result in poor compliance.
Intermittent Abdominal Pressure Ventilation (IAPV) is a valid, though unconventional, alternative to daytime NIV: it consists of a portable ventilator with an internal battery and a corset as interface. The IAPV corset is lightweight, comfortable and, thanks to velcro fasteners, easier and better fitting than a face mask. Cyclical inflation of a rubber bladder inside the corset moves the diaphragm upwards like a pneumobelt causing air to enter in the lungs via the upper airways as gravity draws the diaphragm back to its resting position.
IAPV is indicated in neuromuscular disease and has already been tested in few preliminary studies and case reports. This study wants to verify the hypothesis of its application in population of neuromuscular patients.
- Conditions
- Neuromuscular Diseases
- Being tested
- Intermittent Abdominal Pressure Ventilation, Usual ventilation
- Keywords
- Neuromuscular Diseases;, Intermittent Abdominal Pressure Ventilation;, IAPV;
- Medical subject headings
- Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intermittent Abdominal Pressure VentilationDevice
Also called IAPV
Patients of experimental group will use IAPV in daytime ventilation. Intermittent abdominal pressure ventilation (IAPV) is a portable ventilator with an internal battery and PneumoBelt corset as an interface.
Usual ventilationDevice
NIV
| Study group | Role | What they receive |
|---|---|---|
| Intermittent Abdominal Pressure Ventilation | Experimental | Patients in experimental group will be adapted to Intermittent Abdominal Pressure Ventilation (IAPV). Abdominal ventilation replaces part of usual ventilation (non invasive ventilation with mouthpiece or nasal-pillow) Receives: Device: Intermittent Abdominal Pressure Ventilation |
| Usual ventilation | Active comparator | Patients in control group continue with usual ventilation (NIV through mouthpiece or nasal-pillow) Receives: Device: Usual ventilation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Blood Gas Analysis
Changes of respiratory parameters in terms of normalization of oxyemia (Arterial Pressure of oxygen 80-100 mmHg) and normalization of capnia (Arterial Pressure of carbon dioxide 35-45 mmHg), assessed by blood gas analysis
Measured: 12 months
Secondary outcomes (4)
Therapy adherence
Adherence to IAPV tested with Beliefs about Ventilation Questionnaire (BVQ). BVQ is a qualitative questionnaire: it consist of 25 items, where higher scores mean a better outcome.
Measured: 12 months
Patients satisfaction to IAPV
Satisfaction to IAPV tested with Visual Analog Scale (VAS), with a score that ranges between 0 and 10, where a higher score means a worse outcome.
Measured: 12 months
Caregivers satisfaction to IAPV
Satisfaction to IAPV tested with Visual Analog Scale (VAS), with a score that ranges between 0 and 10, where a higher score means a worse outcome.
Measured: 12 months
Amelioration of Quality of life
Improvement of quality of life tested with World Health Organization Quality of Life-Brief (WHOQOL-Brief), with a score ranging from 0 to 100 for each of the four domain and where a higher score means a better outcome
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Apr 2022
- Study start1 Mar 2022
- Primary completion1 Oct 2023 (estimated)
- Study completion1 Mar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseasesStudies of the same condition, open and under way.
- More trials run by Fondazione Don Carlo Gnocchi ETSThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 78 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione Don Carlo Gnocchi ETS
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05346263
- Sponsor's protocol ID
- 19/2021/CE_FdG/FC/SA
- Acronym
- IAPV
- Status last verified
- Mar 2022
- First submitted
- 13 Apr 2022
- Last updated
- 19 Dec 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy of Intermittent Abdominal Pressure Ventilation in Neuromuscular Patients: Multicentre, Randomised Controlled Trial
- ClinicalTrials.gov ID
- NCT05346263
- Acronym / sponsor ID
- IAPV / 19/2021/CE_FdG/FC/SA
- Conditions
- Neuromuscular Diseases
- Keywords
- Neuromuscular Diseases;, Intermittent Abdominal Pressure Ventilation;, IAPV;
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- Fondazione Don Carlo Gnocchi ETSUniversity, hospital or other
- Study start
- 1 Mar 2022 (actual)
- Primary completion
- 1 Oct 2023 (estimated)
- Study completion
- 1 Mar 2025 (estimated)
- First posted
- 26 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Non-Invasive Ventilation (NIV) is an established treatment to manage respiratory muscles dysfunction in neuromuscular disease, preventing the progression of respiratory failure to intubation and/or a tracheotomy. NIV is commonly needed at first during the night, but when the disease worsens, it is required during the day. It is provided via nasal or oronasal masks, causing discomfort and/or aesthetic issues that result in poor compliance.
Intermittent Abdominal Pressure Ventilation (IAPV) is a valid, though unconventional, alternative to daytime NIV: it consists of a portable ventilator with an internal battery and a corset as interface. The IAPV corset is lightweight, comfortable and, thanks to velcro fasteners, easier and better fitting than a face mask. Cyclical inflation of a rubber bladder inside the corset moves the diaphragm upwards like a pneumobelt causing air to enter in the lungs via the upper airways as gravity draws the diaphragm back to its resting position.
IAPV is indicated in neuromuscular disease and has already been tested in few preliminary studies and case reports. This study wants to verify the hypothesis of its application in population of neuromuscular patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intermittent Abdominal Pressure Ventilation | Experimental | Device: Intermittent Abdominal Pressure VentilationPatients in experimental group will be adapted to Intermittent Abdominal Pressure Ventilation (IAPV). Abdominal ventilation replaces part of usual ventilation (non invasive ventilation with mouthpiece or nasal-pillow) |
| Usual ventilation | Active comparator | Device: Usual ventilationPatients in control group continue with usual ventilation (NIV through mouthpiece or nasal-pillow) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosis of Neuromuscular Disease (Amyotrophic Lateral Sclerosis, Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Pompe Disease)
- Non Invasive Ventilation > 16 hours/day
- Informed consent signed
Key exclusion criteria (1 listed)
- Diagnosis of kyphoscoliosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intermittent Abdominal Pressure VentilationAlso: IAPVPatients of experimental group will use IAPV in daytime ventilation. Intermittent abdominal pressure ventilation (IAPV) is a portable ventilator with an internal battery and PneumoBelt corset as an interface. | Device | Intermittent Abdominal Pressure Ventilation |
| Usual ventilationNIV | Device | Usual ventilation |
Primary outcome
Blood Gas Analysis
Time frame: 12 months
Changes of respiratory parameters in terms of normalization of oxyemia (Arterial Pressure of oxygen 80-100 mmHg) and normalization of capnia (Arterial Pressure of carbon dioxide 35-45 mmHg), assessed by blood gas analysis
4 secondary outcomes
Therapy adherence
Time frame: 12 months
Patients satisfaction to IAPV
Time frame: 12 months
Caregivers satisfaction to IAPV
Time frame: 12 months
Amelioration of Quality of life
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Dec 2022.
- Individual participant dataSharing planned: No
Related trials for Neuromuscular Diseases
Active, not recruitingPhase 1/2
Selumetinib in Treating Young Patients With Recurrent or Refractory Low Grade Glioma
Low Grade Glioma, Recurrent Childhood Pilocytic Astrocytoma
Enrolling by invitation
Online Exercise Program for Transplant Patients: A Pilot Study
Kidney Transplantation, Physical Function
Active, not recruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
RecruitingObservational study
Pain Phenotypes in Individuals With Multiple Sclerosis
Multiple Sclerosis, Chronic Pain
Not yet recruitingPhase 2
A Study of Bimagrumab in Participants With HFpEF
Heart Failure, Diastolic, Diastolic Heart Failure
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Valeria Volpi, PT contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Intermittent Abdominal Pressure Ventilation and Usual ventilation in people with Neuromuscular Diseases, run by Fondazione Don Carlo Gnocchi ETS at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Paolo Banfi, MD (00390240308812, pabanfi@dongnocchi.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Blood Gas Analysis (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Dec 2022.