Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments

NYU Langone Health

Status and phase

Withdrawn

Early Phase 1

Conditions

Spinal Muscular Atrophy

Treatments

Drug: Nusinersen Treatments

Study type

Interventional

Funder types

Other

Identifiers

NCT04050852

19-00242

Details and patient eligibility

About

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.

Sex

All

Ages

5 to 21 years old

Volunteers

No Healthy Volunteers

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).