A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
| Status: | Active, not recruiting |
|---|---|
| Conditions: | Neurology, Neurology, Orthopedic |
| Therapuetic Areas: | Neurology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 8/29/2018 |
| Start Date: | May 20, 2015 |
| End Date: | January 25, 2022 |
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.
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.
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.
Key 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.
Key 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.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
We found this trial at
10
sites
1800 Orleans St.
Baltimore, Maryland 21287
Baltimore, Maryland 21287
410-955-5000
Principal Investigator: Tom Crawford, MD
Phone: 443-287-6294
Johns Hopkins Hospital Patients are the focus of everything we do at The Johns Hopkins...
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185 Cambridge Street
Boston, Massachusetts 02114
Boston, Massachusetts 02114
617-724-5200
Principal Investigator: Kathryn Swoboda, MD
Phone: 617-312-8318
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100 N Mario Capecchi Dr
Salt Lake City, Utah 84132
Salt Lake City, Utah 84132
(801) 662-1000
Principal Investigator: Ai Sakonju, MD
Phone: 801-585-9055
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13123 E 16th Ave
Aurora, Colorado 80045
Aurora, Colorado 80045
(720) 777-1234
Principal Investigator: Julie Parsons, MD
Phone: 720-777-1319
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Johns Hopkins Hospital Patients are the focus of everything we do at The Johns Hopkins...
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225 E Chicago Ave
Chicago, Illinois 60611
Chicago, Illinois 60611
(312) 227-4000
Principal Investigator: Nancy Kuntz, MD
Phone: 312-227-4449
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
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New York, New York 10032
Principal Investigator: Darryl DeVivo, MD
Phone: 212-304-5205
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Orlando, Florida 32806
Principal Investigator: Richard Finkel, MD
Phone: 407-567-6201
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