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

Withdrawn before enrolling · Early Phase 1

Conditions studied: Spinal Muscular Atrophy

In brief

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.

Key facts

Study ID

NCT04050852

Run by

NYU Langone Health

People needed

0

Starts

2019-07-17

Expected to finish

2021-04-01

Last updated by the study team

2021-06-29

Who can join

Age: 5 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Patients diagnosed with SMA of any type who are eligible to start nusinersen treatments at NYU Winthrop Hospital.

You may not qualify if…

• 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).

Where it is running

• NYU Langone Health — New York, New York, United States

Full record on ClinicalTrials.gov

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