Isis CS11 SHINE

Principal Investigator: Nicholas Johnson
Keywords: Spinal Muscular Atrophy , SMA Department: Neurology
IRB Number: 00088164 Co Investigator: Russell Butterfield
Specialty: Neurology, Neurology, Neurology
Sub Specialties: Movement Disorders, Spinal Muscular Atrophy, Muscular Dystrophy
Recruitment Status: Not yet recruiting

Contact Information

Nicole Rausch
nicoler@genetics.utah.edu
801-585-9717

Brief Summary

Amendment Revisions are as follows: 

The objective of this open-label extension study is to gather additional information on the
long-term safety, tolerability, and efficacy of repeated 12-mg doses of ISIS 396443 (also known
as BIIB058 and nusinersen) administered as intrathecal (IT) injections by lumbar puncture (LP)
in subjects with spinal muscular atrophy (SMA) who previously participated in investigational
studies of ISIS 396443.


Note: For the purposes of this protocol, when referring to “study drug,” nusinersen will be used
in place of ISIS 396443. When referring to the Protocol Title or previous (“index”) studies,
ISIS 396443 will be used. The term “study treatment” refers to administration of nusinersen or
the sham procedure during the blinded portion of the study.

 

 

The primary purpose of this study is to gather additional information on the long-term safety, tolerability, and efficacy of repeated doses of ISIS 396443 (12 mg) administered as intrathecal (into the spinal fluid) injections by lumbar puncture (LP) in participants who previously participated in investigational studies of ISIS 396443. For the purposes of the current protocol, investigational studies of ISIS 396443, including studies ISIS 396443-CS3B, ISIS 396443-CS4 and ISIS 396443-CS12. Participants from other studies of ISIS 396443 may be included into the current long-term extension study with future amendments of the protocol.

 

The objectives of this study are to gather additional information on the long-term safety, tolerability, and efficacy of repeated doses of ISIS 396443 (12 mg) administered as intrathecal (IT) injections by lumbar puncture (LP) in participants who previously participated in investigational studies of ISIS 396443. Another objective of this study is to examine the cerebrospinal fluid (CSF) pharmacokinetics (PK) of ISIS 396443 administered intrathecally to patients with SMA who previously participated in investigational studies of ISIS 396443.

 

 

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
3. Ability to complete all study procedures, measurements and visits, and presence of 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: have a negative pregnancy test at Screening and 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: be abstinent* for the duration of the study or 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 trial) and withdrawal are not acceptable methods of contraception.

Exclusion Criteria

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. The subject's parent or legal guardian is unable to understand the nature, scope, and
possible consequences of the study, or does not agree to comply with the protocol's schedule of assessments
4. 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 5. Treatment with another investigational agent, biological agent, or device within one month of Screening, or 5 half-lives of study agent, whichever is longer