Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments
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|ClinicalTrials.gov Identifier: NCT04050852|
Recruitment Status : Recruiting
First Posted : August 8, 2019
Last Update Posted : August 8, 2019
|Condition or disease||Intervention/treatment||Phase|
|Spinal Muscular Atrophy||Drug: Nusinersen Treatments||Early Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||5-10 patients with spinal muscular atrophy (SMA), ages 5 years old and above, who have already consented to nusinersen treatment, or have already initiated nusinersen (spinraza) treatments at NYU Winthrop Hospital.|
|Masking:||None (Open Label)|
|Official Title:||Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments|
|Actual Study Start Date :||July 17, 2019|
|Estimated Primary Completion Date :||July 17, 2020|
|Estimated Study Completion Date :||July 17, 2020|
|Experimental: SMA patients receiving nusinersen treatments||
Drug: Nusinersen Treatments
Antisense oligonucleotide, which targets the SMN2 pre-mRNA, particularly exon 7.
Other Name: Spinraza
- 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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04050852
|Contact: Ann Mockemail@example.com|
|United States, New York|
|NYU Langone Health||Recruiting|
|New York, New York, United States, 10016|
|Contact: Ann Mock 516-663-9619 firstname.lastname@example.org|
|Principal Investigator: Melodi Pirzada, MD|
|Principal Investigator:||Melodi Pirzada, MD||New York Langone Medical Center|