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A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

NCT02462759 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2021-02-17

Study results available
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Summary

The primary objective of Part 1 of this study is to assess the safety and tolerability of Nusinersen in participants with SMA who are not eligible to participate in the clinical studies ISIS 396443-CS3B (NCT02193074) or ISIS 396443-CS4 (NCT02292537). The secondary objective of Part 1 of this study is to examine the pharmacokinetics (PK) of Nusinersen in participants with SMA. The primary objective of Part 2 of this study is to assess the long-term safety and tolerability of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments. The secondary objective of Part 2 of this study is to examine the PK of Nusinersen in participants with SMA who participated in Part 1 and completed their End of Part 1 Evaluation assessments.

Conditions

Interventions

DRUG

Nusinersen

Administered by intrathecal injection.

PROCEDURE

Sham Procedure

Small needle prick on the lower back at the location where the IT injection is normally made.

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-08-19
Primary Completion
2018-09-24
Completion
2018-09-24
FDA Drug
Yes

Countries

  • United States
  • Germany

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02462759 on ClinicalTrials.gov