Inspiratory Muscle Training (IMT) in Adult People With Pompe Disease

Summary

The goal of this multicentre, randomized and controlled cross-over trial is to evaluate the efficacy of a programme of Inspiratory Muscle Training in subjects with Late On-set Pompe Disease (LOPD). The main question is to:

* verify changes in Forced Vital Capacity, Postural Drop, Maximal Inspiratory Pressure, Maximal Expiratory Pressure, Peak expiratory cough pressure, Maximal Inspiratory Capacity, six- minute walk test and or 6-minute pegboard ring test.-
* measure changes in some questionnaries investigating dispnoea and quality of life (Short-Form 36, Individualized-Neuromuscular-Quality-of-Life, Maugeri-Respiratory-Failure 28, Borg scale, Dispnoea 12, Mulditimensional Dispnea Profile, modified Medical Research Council, Fatigue Severity Scale, Epsworth Scale, Visual Analogue Scale).

Measurement will take place at baseline and after one, three, four, six and twelve months.

Participants will undergo a specific treatment consisting of aerobic exercise and Inspiratory Muscle Training with Powerbreathe device or Air-Stacking.

Researchers will study if Powerbreathe device is more effective than Air-stacking maneuvres

Conditions

• Glycogen Storage Disease Type II
• Pompe Disease (Late-onset)

Interventions

DEVICE

Inspiratory Muscle Training (IMT)

Inspiratory muscle training (IMT) involves breathing exercises using a pressure threshold device to strengthen the muscles involved in breathing in

DEVICE

Inspiratory Muscle Training (IMT) + Air Stacking

Inspiratory muscle training (IMT) involves breathing exercises using a pressure threshold device to strengthen the muscles involved in breathing in, along with Air Stacking (AS). AS is a lung insufflation method that requires the use of a manual insufflator to provide air volumes higher than inspiratory capacity

Sponsors & Collaborators

• Associazione Italiana Glicogenosi (AIG)

  collaborator UNKNOWN

• Associazione Riabilitatori Insufficienza Respiratoria (ArIR)

  collaborator UNKNOWN

• U.O.C. AO Ospedali Riuniti Villa Sofia-Cervello, Palermo

  collaborator UNKNOWN

• S.C. di Pneumologia Azienda Sanitaria Universitaria Integrata di Trieste

  collaborator UNKNOWN

• Fondazione Don Carlo Gnocchi Onlus

  lead OTHER

Principal Investigators

• Paolo Banfi, MD · IRCCS Fondazione Don Carlo Gnocchi

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

CROSSOVER

Eligibility

Min Age

18 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2023-03-01

Primary Completion

2023-06-01

Completion

2024-04-01

Countries

• Italy

Study Locations