A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies (SHINE)

Biogen

Status and phase: Completed; Phase 3

Conditions: Spinal Muscular Atrophy

Treatments: Drug: nusinersen

Study type: Interventional

Funder types: Industry

Identifiers

NCT02594124

ISIS 396443-CS11

2015-001870-16 (EudraCT Number)

Details and patient eligibility

About

The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.

Full description

This study was initiated and the protocol was registered by Ionis Pharmaceuticals, Inc.

In August 2016, Biogen assumed responsibility for this study.

Enrollment: 292 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Signed informed consent of parent or guardian and signed informed assent of participant, if indicated per participant's age and institutional guidelines.
• Completion of the index study in accordance with the study protocol or as a result of Sponsor decision (e.g., early termination of the index study) within the preceding 16 weeks

Key Exclusion Criteria:

• Have any condition or worsening condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study
• Clinically significant abnormalities in hematology or clinical chemistry parameters or electrocardiogram (ECG), as assessed by the Site Investigator, at the Screening visit that would render the participant unsuitable for participation in the study
• Participant's parent or legal guardian is not willing or able to meet standard of care guidelines (including vaccinations and respiratory syncytial virus prophylaxis if available), nor provide nutritional and respiratory support throughout the study
• Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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292 participants in 5 patient groups

Group 1

Experimental group

Description:

Participants transitioned from ISIS 396443-CS3B (NCT02193074)

Treatment:

Drug: nusinersen

Group 2

Experimental group

Description:

Participants transitioned from ISIS 396443-CS4 (NCT02292537)

Treatment:

Drug: nusinersen

Group 3

Experimental group

Description:

Participants transitioned from ISIS 396443-CS12 (NCT02052791)

Treatment:

Drug: nusinersen

Group 4

Experimental group

Description:

Participants transitioned from ISIS 396443-CS3A (NCT01839656)

Treatment:

Drug: nusinersen

Group 5

Experimental group

Description:

Participants transitioned from 232SM202 (NCT02462759)

Treatment:

Drug: nusinersen