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A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | Clinical Trials | Clareo Health

COMPLETEDPHASE2

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

NCT02386553•Biogen

View on ClinicalTrials.gov

Summary

The primary objective of the study is to examine the efficacy of multiple doses of Nusinersen administered intrathecally in preventing or delaying the need for respiratory intervention or death in infants with genetically diagnosed and presymptomatic spinal muscular atrophy (SMA). Secondary objectives of this study are to examine the effects of Nusinersen in infants with genetically diagnosed and presymptomatic SMA.

Eligibility

Age

0 - 0 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Age ≤ 6 weeks at first dose.
•Genetic documentation of 5q SMA homozygous gene deletion or mutation or compound heterozygous mutation.
•Genetic documentation of 2 or 3 copies of survival motor neuron 2 (SMN2).
•Ulnar compound muscle action potential (CMAP) ≥ 1 mV at Baseline.
•Gestational age of 37 to 42 weeks for singleton births; gestational age of 34 to 42 weeks for twins.
•Meet additional study related criteria.

Exclusion Criteria

•Hypoxemia (oxygen saturation \<96% awake or asleep without any supplemental oxygen or respiratory support).
•Any clinical signs or symptoms at Screening or immediately prior to the first dosing (Day 1) that are, in the opinion of the Investigator, strongly suggestive of SMA.
•Clinically significant abnormalities in hematology or clinical chemistry parameters.
•Treatment with an investigational drug given for the treatment of SMA biological agent, or device. Any history of gene therapy, prior antisense oligonucleotide (ASO) treatment, or cell transplantation.
•Meet additional study related criteria.

Locations (21)

David Geffen School of Medicine

Los Angeles, California, United States

University of California Davis Health System

Sacramento, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nemours Children's Hospital, Orlando

Orlando, Florida, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

The Johns Hopkins Hospital

Baltimore, Maryland, United States

Massachusetts General Hospital

Boston, Massachusetts, United States

Columbia University

New York, New York, United States

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Utah

Salt Lake City, Utah, United States

Key Dates

Start Date

May 18, 2015

Primary Completion Date

December 17, 2024

Completion Date

December 17, 2024

Last Updated

October 20, 2025

Enrollment

ACTUAL participants

Conditions

Spinal Muscular Atrophy

Interventions

Nusinersen

DRUG

Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

NCT06321965

Spinal Muscular Atrophy

View study

RECRUITINGNA

Long Read Analysis in Spinal Muscular Atrophy - LOREASI

NCT07332702

Spinal Muscular Atrophy (SMA)

View study

RECRUITINGPHASE3

Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: October 20, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Inclusion Criteria

Exclusion Criteria

Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

Long Read Analysis in Spinal Muscular Atrophy - LOREASI

Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases

Outpatient Rehabilitation Intervention for Young Children With SMA