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An Open label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443
Study ID: STU 112015-059
Summary
This is an open-label extension study in subjects with SMa who previously participated in investigational studies of iSiS 396443. For the purposes of this protocol, investigational studies of iSiS 396443, include Studies CS3a, CS3B, CS4, and CS12. Subjects from other studies of iSiS 396443 may be included into the current long-term extension study with future amendments of the protocol. upon approval of this amendment at study sites, regardless of their original dosing visit schedule (i.e., 6-month dosing schedule), all subjects will begin receiving maintenance doses of nusinersen every 4 months upon entering the modified maintenance dosing regimen (MMDR) period of the study. This study will consist of Screening, Treatment, and Post-Treatment Follow-up Periods, and an end-of-Study (eoS) evaluation Visit. The total duration of participation in the study is 5 years from MMDR Day 1 (eoS evaluation Visit to occur at approximately MMDR Day 1800) or as determined by the Sponsor (via early termination or amendment to extend). Group 1a subjects will receive 4 loading doses by iT injection on Days 1, 15, 29, and 64 ((+-)1 day). Group 1B will receive 3 sham procedures on Days 1, 15, and 64 and 1 loading dose by iT injection on Day 29 ((+-)1 ). Both Group1a and 1B loading doses will be followed by maintenance doses approximately every 4 months according to the MMDR dosing schedule. Subjects currently participating in the loading dose treatment cycle in iSiS 396443-CS11 will complete the cycle and then transition to MMDR Day 1. Group 2a subjects will receive 3 loading doses by iT injection on Days 1, 29, and 85 ((+-)1 day). Group 2B will receive 2 loading doses by iT injection on Days 1 and 85 and 1 sham procedure on Day 29 ((+-)1 day). Both Group 2 a and 2B loading doses will be followed by maintenance doses approximately every 4 months according to the MMDR dosing schedule. Subjects currently participating in the loading dose treatment cycle in iSiS 396443- CS11 will complete the cycle and then transition to MMDR Day 1. Group 3 subjects will receive maintenance doses every 4 months according to the MMDR dosing schedule. Subjects currently receiving open-label maintenance doses of nusinersen in iSiS 396443-CS11 will be transitioned to the MMDR dosing period at their next study visit, which should be scheduled as close as possible to 120 days from the date of the last visit (i.e., date of last visit + 120 days [?] MMDR Day 1). addition of dosing for iSiS 396443-CS3a Group 4 subjects will receive maintenance doses every 4 months according to the MMDR dosing schedule. Subjects currently receiving open-label maintenance doses of nusinersen in iSiS 396443-CS11 will be transitioned to the MMDR dosing period at their next study visit, which should be scheduled as close as possible to 120 days from the date of the last visit (i.e., date of last visit + 120 days [?] MMDR Day 1). all subjects will remain at the Study Center for at least 1 hour post-procedure for safety monitoring and can be discharged at the discretion of the site investigator and in compliance with the institutional requirements once the subjects adequately recover from the dosing procedure. Safety monitoring by telephone contact will occur to monitor the subjects' condition 1 to 7 days post dose and every other month throughout the duration of the study, except for the months during which a study visit occurs. Subjects who discontinue treatment will continue follow-up assessments through scheduled study visits unless consent is withdrawn. Subjects who terminate early from the study for any reason will be encouraged to complete safety assessments per the protocol. Subjects who discontinue from the study early will not be replaced.
Participant Eligibility
1. Signed informed consent of parent or guardian and signed informed assent of subject, if indicated per subject[Single Quote]s age and institutional guidelines. 2. 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 preceding16 weeks. 3. Ability to complete all study procedures, measurements, and visits, and parent/subject has adequately supportive psychosocial circumstances, in the opinion of the Investigator. 4. Subjects who, in the opinion of the Investigator, have reached reproductive maturity, must satisfy one of the following: * * Females must have a negative pregnancy test at Screening and must agree to employ adequate contraceptive measures for the duration of the study. Acceptable contraception methods are restricted to abstinence*, barrier contraceptives, intrauterine contraceptive devices, or licensed hormonal products. * * Males must be abstinent* for the duration of the study or must be using an acceptable contraceptive method (i.e., use a condom together with spermicidal foam/gel/film/cream/suppository). *Abstinence is only acceptable as true abstinence (i.e., when this is representative of the preferred and usual lifestyle of the subject). Periodic abstinence (e.g., abstinence for the duration of the study) and withdrawal are not acceptable methods of contraception.
- Cancer Related
- No
- Healthy Volunteers
- No
- UT Southwestern Principal Investigator
- Diana Patricia Castro
IONIS PHARMACEUTICALS INC
Other