NCT04050852 is a EARLY_PHASE1 clinical trial of Nusinersen Treatments in Spinal Muscular Atrophy, sponsored by NYU Langone Health.

Who is sponsoring NCT04050852?

NCT04050852 is sponsored by NYU Langone Health.

What drugs are being tested in NCT04050852?

Interventions in NCT04050852: Nusinersen Treatments.

What condition does NCT04050852 study?

NCT04050852 studies Spinal Muscular Atrophy.

Is NCT04050852 still recruiting?

NCT04050852 is currently withdrawn. Last updated 29 June 2021.

Last reviewed 2021-06-29 · How we verify

NCT04050852

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

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

Withdrawn EARLY_PHASE1 Last updated 29 June 2021

What this trial tests

EARLY_PHASE1 trial testing Nusinersen Treatments in Spinal Muscular Atrophy. Withdrawn.

Timeline

17 July 2019

Primary endpoint
1 April 2021

1 April 2021

Quick facts

Lead sponsor	NYU Langone Health
Phase	EARLY_PHASE1
Status	Withdrawn
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Start date	17 July 2019
Primary completion	1 April 2021
Estimated completion	1 April 2021
Sites	1 location across United States

Drugs / interventions tested

Nusinersen Treatments — full drug profile →

Conditions studied

Spinal Muscular Atrophy — all drugs for Spinal Muscular Atrophy →

Sponsor

NYU Langone Health — full company profile →

Who can join

Adults 5 to 21, any sex, with Spinal Muscular Atrophy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

4 peer-reviewed publications reference this trial (live from Europe PMC):

Gene-based therapies for neurodegenerative diseases.
Sun J, Roy S. · · 2021 · cited 126× · PMID 33526943 · DOI 10.1038/s41593-020-00778-1
Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders.
Gomez Limia C, Baird M, Schwartz M, Saxena S, et al · · 2022 · cited 13× · PMID 36556200 · DOI 10.3390/jpm12121979
Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical-Experimental, and Real-World Evidence.
Crisafulli S, Boccanegra B, Vitturi G, Trifirò G, et al · · 2023 · cited 11× · PMID 37891814 · DOI 10.3390/brainsci13101446
Significance of gene therapy in neurodegenerative diseases.
Wang L, Ma L, Gao Z, Wang Y, et al · · 2025 · cited 1× · PMID 40406043 · DOI 10.3389/fnins.2025.1515255

Verify or expand the search:

Other recruiting trials for Spinal Muscular Atrophy

Currently open trials in the same condition.

NCT07478172 — Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease · NA · recruiting
NCT06955897 — Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA · recruiting
NCT06396325 — A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolescents With · NA · recruiting
NCT06288230 — An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy · Phase 1, PHASE2 · recruiting
NCT06321965 — Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy · NA · recruiting

Other NYU Langone Health trials

Trials by the same sponsor.

NCT07214519 — Permanent Supportive Housing Overdose Prevention-2 Study · NA · not yet recruiting
NCT05558267 — Potassium Containing Salt-Substitute in Hemodialysis-Dependent End Stage Kidney Disease · NA · not yet recruiting
NCT06637852 — Sexual and Urinary Function Improvement for Cancer Survivors · NA · not yet recruiting
NCT06236087 — Overdose Prevention Centers and Behavioral Health · not yet recruiting
NCT06462027 — Packed Red Blood Cell Transfusion During Cardiac Arrest · Phase 1 · suspended

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04050852 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by NYU Langone Health
Last refreshed: 29 June 2021

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT04050852.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing