Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02052791
Other study ID # ISIS 396443-CS12
Secondary ID
Status Completed
Phase Phase 1
First received
Last updated
Start date January 31, 2014
Est. completion date January 31, 2017

Study information

Verified date February 2021
Source Biogen
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The primary objective of this study is to examine the safety and tolerability of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA) who previously participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246). The secondary objective is to examine the plasma and cerebrospinal fluid (CSF) pharmacokinetic(s) (PK) of nusinersen administered intrathecally to participants with SMA who previously participated in ISIS 396443-CS2 or ISIS 396443-CS10.


Description:

This study was conducted and the protocol was registered by Ionis Pharmaceuticals, Inc. In August 2016, sponsorship of the trial was transferred to Biogen.


Recruitment information / eligibility

Status Completed
Enrollment 47
Est. completion date January 31, 2017
Est. primary completion date January 31, 2017
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Key Inclusion Criteria: - Clinical signs attributable to Spinal Muscular Atrophy - Satisfactory completion of dosing and all study visits in ISIS 396443-CS2 (NCT01703988) or ISIS 396443 CS10 (NCT01780246) with an acceptable safety profile, per Investigator judgement. - Able to complete all study procedures, measurements and visits and parent/participant has adequately supportive psychosocial circumstances, in the opinion of the investigator - Estimated life expectancy > 2 years from Screening - Meets age-appropriate institutional criteria for use of anesthesia/sedation, if use is planned for study procedure Key Exclusion Criteria: - Have any new or worsening of existing condition which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the participant participating in or completing the study. - Dosing in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) within 180 days (6 months) of screening, or longer ago than 396 days (13 months) from screening - Hospitalization for surgery (i.e. scoliosis surgery) or pulmonary event within 2 months of screening or planned during the duration of the study - Presence of an untreated or inadequately treated active infection requiring systemic antiviral or antimicrobial therapy - Clinically significant abnormalities in hematology or clinical chemistry parameters - Treatment with another investigational drug, biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Any history of gene therapy or cell transplantation. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design


Intervention

Drug:
nusinersen
Administered by intrathecal (IT) injection

Locations

Country Name City State
United States Boston Children's Hospital Boston Massachusetts
United States UT Southwestern Medical Center - Children's Medical Center Dallas Dallas Texas
United States Columbia University Medical Center New York New York
United States University of Utah School of Medicine Salt Lake City Utah

Sponsors (1)

Lead Sponsor Collaborator
Biogen

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Number of participants that experience Adverse Events (AEs) and Serious Adverse Events Up to 24 Months
Primary Number of participants with clinically significant neurological examination abnormalities Up to 24 Months
Primary Number of participants with clinically significant vital sign abnormalities Up to 24 Months
Primary Number of participants with clinically significant physical examination abnormalities Up to 24 Months
Primary Number of participants with clinically significant weight abnormalities Up to 24 Months
Primary Number of participants with clinically significant laboratory parameters Up to 24 Months
Primary Number or participants with clinically significant cerbrospinal fluid (CSF) laboratory parameters Up to Day 176
Primary Number of participants with clinically significant electrocardiograms (ECGs) abnormalities Up to 24 Months
Primary Change from Baseline in concomitant medications Up to 24 Months
Secondary PK parameters of nusinersen (ISIS 396443) in CSF levels: Maximum observed plasma drug concentration (Cmax) Pre-dose Day 176, Day 358 and Day 540
Secondary PK parameters of nusinersen in CSF levels: Time to reach maximum observed concentration (Tmax) Pre-Dose Day 176, Day 358 and Day 540
Secondary PK parameters of nusinersen in CSF levels: Area under the plasma concentrations time curve from the time of the intrathecal (IT) dose to the last collected sample (AUCinf) Pre-Dose Day 176, Day 358 and Day 540
See also
  Status Clinical Trial Phase
Completed NCT04851873 - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) Phase 3
Completed NCT03223051 - Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy N/A
Completed NCT04335942 - Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor N/A
Recruiting NCT05794139 - Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy Phase 2
Not yet recruiting NCT06300996 - Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb N/A
Not yet recruiting NCT00961103 - Motor Development and Orthoses in Spinal Muscular Atrophy (SMA) N/A
Completed NCT02003937 - Aerobic Training in Patients With Spinal Muscular Atrophy Type III N/A
Completed NCT00227266 - Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy Phase 2
Completed NCT00374075 - Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy Phase 1
Enrolling by invitation NCT05539456 - Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old
Recruiting NCT05779956 - Personalized Medicine for SMA: a Translational Project
Recruiting NCT03300869 - Natural History of Types 2 and 3 SMA in Taiwan
Recruiting NCT03217578 - Neonatal Spinal Muscular Atrophy (SMA) Screening
Completed NCT01703988 - An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Phase 1/Phase 2
Withdrawn NCT02235090 - Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting N/A
Completed NCT02123186 - Newborn Screening for Spinal Muscular Atrophy N/A
Completed NCT00756821 - A Pilot Study of Biomarkers for Spinal Muscular Atrophy N/A
Completed NCT00004771 - Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease Phase 2
Recruiting NCT05366465 - Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France
Recruiting NCT06310421 - Spinal Muscular Atrophy Neonatal Screening Program