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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
Sponsor:
Collaborator:
Winthrop University Hospital
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Drug: Nusinersen Treatments Early Phase 1

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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)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Experimental: SMA patients receiving nusinersen treatments Drug: Nusinersen Treatments
Antisense oligonucleotide, which targets the SMN2 pre-mRNA, particularly exon 7.
Other Name: Spinraza




Primary Outcome Measures :
  1. 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.

  2. 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.



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Ages Eligible for Study:   5 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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).

Information from the National Library of Medicine

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


Contacts
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Contact: Ann Mock 5166639619 ann.mock@nyulangone.org

Locations
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United States, New York
NYU Langone Health Recruiting
New York, New York, United States, 10016
Contact: Ann Mock    516-663-9619    ann.mock@nyulangone.org   
Principal Investigator: Melodi Pirzada, MD         
Sponsors and Collaborators
NYU Langone Health
Winthrop University Hospital
Investigators
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Principal Investigator: Melodi Pirzada, MD New York Langone Medical Center

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Responsible Party: NYU Langone Health
ClinicalTrials.gov Identifier: NCT04050852     History of Changes
Other Study ID Numbers: 19-00242
First Posted: August 8, 2019    Key Record Dates
Last Update Posted: August 8, 2019
Last Verified: August 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases