An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies

Conditions: Spinal Muscular Atrophy (SMA)
MedDRA version: 18.1Level: PTClassification code 10041582Term: Spinal muscular atrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Registration Number: EUCTR2015-001870-16-ES

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 274

Inclusion Criteria

Subjects must meet all of the following criteria at Screening to be eligible:
1.Signed informed consent of parent or guardian. Signed informed assent of subject, if indicated per subject?s age and institutional guidelines
2.Completion of the index study in accordance with the study protocol within preceding 12 weeks
Are the trial subjects under 18? yes
Number of subjects for this age range: 274
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

Subjects meeting any of the following criteria are not eligible for the study:
1.Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the subject unsuitable for enrollment, or could interfere with the subject participating in or completing the study
2.Clinically significant abnormalities in hematology or clinical chemistry parameters or ECG, as assessed by the Site Investigator, at the Screening visit that would render the subject unsuitable for participation in the study
3.Subject?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
4.Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer