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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2024
• Main ID: NCT02594124
• Date of registration: 30/10/2015
• Prospective Registration: Yes
• Primary sponsor: Biogen
• Public title: A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies SHINE
• Scientific title: An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443
• Date of first enrolment: November 4, 2015
• Target sample size: 292
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02594124
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, France, Germany, Hong Kong, Italy, Japan, Korea, Republic of, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Biogen

Key inclusion & 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.



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Spinal Muscular Atrophy

Intervention(s)

Drug: nusinersen

Primary Outcome(s)

Number of participants experiencing Adverse events (AEs) and/or Serious Adverse Events (SAEs) [Time Frame: Up to Day 1814]

Number of participants with clinically significant vital sign abnormalities [Time Frame: Up to Day 1814]

Number of participants with clinically significant 12-lead electrocardiograms (ECGs) abnormalities [Time Frame: Up to Day 1814]

Number of participants with clinically significant laboratory assessment abnormalities [Time Frame: Up to Day 1814]

Number of participants with clinically significant neurological examination abnormalities [Time Frame: Up to Day 1814]

Number of participants with clinically significant weight abnormalities [Time Frame: Up to Day 1814]

Change from Baseline in concomitant medications [Time Frame: Up to Day 1814]

Number of participants with clinically significant coagulation parameter abnormalities [Time Frame: Up to Day 1814]

Secondary Outcome(s)

Number of Disease-related hospitalizations and AEs [Time Frame: Up to Day 1814]

Number of participants hospitalized [Time Frame: Up to Day 1814]

Percentage of participants not requiring permanent ventilation [Time Frame: Up to Day 1814]

Proportion of participants who achieved standing alone [Time Frame: Up to Day 1814]

Number of participants with motor milestones achieved [Time Frame: Up to Day 1814]

Change from Baseline in 6-Minute Walk Test (6MWT) [Time Frame: Up to Day 1814]

Change from Baseline in Cobb-Angle on X-Ray of the thoracolumbar spine [Time Frame: Up to Day 1814]

Change from Baseline in arm circumference (for participants up to 36 months of age) [Time Frame: Up to Day 1814]

Change from Baseline in body length and/or height (for all participants) [Time Frame: Up to Day 1814]

Change from Baseline in Compound Muscular Action Potential (CMAP) [Time Frame: Up to Day 1814]

Change from Baseline in Hammersmith Functional Motor Scale [Time Frame: Up to Day 1814]

Change from Baseline in Quality of Life (QOL) Questionnaires [Time Frame: Up to Day 1814]

Change from Baseline in Revised Upper Limb Module (RULM) [Time Frame: Up to Day 1814]

Change from Baseline in head circumference (for participants up to 36 months of age) [Time Frame: Up to Day 1814]

Duration of hospitalizations [Time Frame: Up to Day 1814]

Overall survival rate [Time Frame: Up to Day 1814]

Percentage of participants who attained motor milestones as assessed by World Health Organization (WHO) criteria [Time Frame: Up to Day 1814]

Percentage of participants who attained motor milestones as assessed by Section 2 of Hammersmith Infant Neurological Examination (HINE) [Time Frame: Up to Day 1814]

Change from Baseline in the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) motor function scale [Time Frame: Up to Day 1814]

Proportion of participants who achieved walking with assistance [Time Frame: Up to Day 1814]

Number of participants with serious respiratory events [Time Frame: Up to Day 1814]

Change from Baseline in chest circumference (for participants up to 36 months of age) [Time Frame: Up to Day 1814]

Time to death or permanent ventilation [Time Frame: Up to Day 1814]

Proportion of CMAP responders [Time Frame: Up to Day 1814]

Secondary ID(s)

2015-001870-16

ISIS 396443-CS11

Source(s) of Monetary Support

Please refer to primary and secondary sponsors