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

Early Phase 1

Withdrawn

• Conditions: Spinal Muscular Atrophy
• Interventions: Drug: Nusinersen Treatments

• Registration Number: NCT04050852
• Lead Sponsor: NYU Langone Health

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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2019-07-17
• Study First Submitted: 2019-08-07
• Study First Submitted QC: 2019-08-07
• Study First Posted: 2019-08-08
• Primary Completion: 2021-04-01
• Study Completion: 2021-04-01
• Status Verified: 2021-06
• Last Update Submitted: 2021-06-25
• Last Update Posted: 2021-06-29

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

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

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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).

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
SMA patients receiving nusinersen treatments	Nusinersen Treatments	-

Primary Outcome Measures

Name	Time	Method
percent improvement Maximum inspiratory pressure (MIP)	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)	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.

Trial Locations

Locations (1)

NYU Langone Health; 🇺🇸 New York, New York, United States