Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments
Overview
- Phase
- Early Phase 1
- Intervention
- Nusinersen Treatments
- Conditions
- Spinal Muscular Atrophy
- Sponsor
- NYU Langone Health
- Locations
- 1
- Primary Endpoint
- percent improvement Maximum inspiratory pressure (MIP)
- Status
- Withdrawn
- Last Updated
- 4 years ago
Overview
Brief Summary
It is well known that patients with spinal muscular atrophy (SMA) have progressive decline of respiratory muscle function. Therapy traditionally involved supportive means to ensure optimal nutrition and airway clearance. Nusinersen (spinraza) is a disease-modifying medication approved for treatment of SMA in pediatric and adult patients. The goal of this study is to observe pulmonary function test (PFT) changes and respiratory muscle strength trends throughout the first year of treatment. A prospective, longitudinal study measuring pulmonary function testing (PFTs) changes in spinal muscular atrophy (SMA) patients. Patients will be patients with SMA who are approved and maintained on nusinersen. Patient will have a baseline PFT. Investigators will repeat PFT at 3, 6, and 12 months while on nusinersen treatment.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Patients diagnosed with SMA of any type who are eligible to start nusinersen treatments at NYU Winthrop Hospital.
Exclusion Criteria
- •Patients unable to comply with nusinersen treatments according to recommended schedule (first 3 doses every 2 weeks, then the 4th dose is administered 1 month after the 3rd dose, and then maintenance dose administered every 4 months).
Arms & Interventions
SMA patients receiving nusinersen treatments
Intervention: Nusinersen Treatments
Outcomes
Primary Outcomes
percent improvement Maximum inspiratory pressure (MIP)
Time Frame: 3, 6, and 12 months
is the pressure developed during forceful inspiration against an occlusion. It is measured at end expiratory phase (near residual volume) and assesses inspiratory muscle strength (diaphragm, external intercostal muscles, and accessory muscles). Normal values for healthy adult females -50 cmH2O and healthy males -75 cmH2O.
percent improvement Maximum expiratory pressure (/MEP)
Time Frame: 3, 6, and 12 months
the pressure developed during forceful expiration against an occlusion. It is measured at end inspiratory phase (near TLC) and assesses expiratory muscle strength (abdominal muscles, internal intercostal muscles, and accessory muscles). Normal values for healthy adult females +80 cmH2O and healthy males +100 cmH2O.