A Prospective, Multi-center, Observational Study of the Safety, Tolerability and Effectiveness of SPINRAZA® (Nusinersen) in Adult Patients With Spinal Muscular Atrophy

Status: Active_not_recruiting
Location: See all (11) locations...
Intervention Type: Other, Drug
Study Type: Observational
SUMMARY

This is a longitudinal, observational study of adult patients with genetically confirmed chromosome 5q SMA to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 70
Healthy Volunteers: f
View:

• Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.

• Males and females with SMA type II or type III, aged 18 to 70 years at the time of enrollment.

• Genetic documentation of 5Q SMA homozygous gene deletion, mutation, or compound heterozygote.

• Are treatment naïve for SPINRAZA® (nusinersen).

• Have been prescribed SPINRAZA® (nusinersen) by the treating physician as part of their clinical care for SMA following the FDA approved prescribing information guidelines as follows: dose level (12 mg), dosing schedule (3 loading doses administered at 14-day intervals, and the fourth loading dose administered 30 days after the third dose and subsequent maintenance doses administered every 4 months) and safety lab monitoring (CBC, PT, INR, PTT, UA) done prior to each dose administration.

• Believed to be able to complete all study procedures, measurements and visits.

• Estimated life expectancy at least 30 months from first dosing, in the opinion of the Investigator.

• Revised upper limb module (RULM) score ≥ 4 (more than marginal upper extremity function/strength.

• Must meet either Group 1 or Group 2 criteria.

⁃ For Group 1 subjects:

• May be ambulatory or non-ambulatory (defined as being wheelchair reliant at least 75% of time and unable to walk at least 10 meters without assistance).

• RULM score of 4-34, inclusive.

⁃ For Group 2 subjects:

• Ability to walk at least 10 meters without assistance (i.e., four point walking aid).

• Be free of major orthopedic deformities that limit ambulation.

• An ambulatory subject can qualify for both Group 1 and Group 2 if the RULM score is ≤ 34.

Locations
United States
Arizona
Barrow Neurological Institute
Phoenix
Washington, D.c.
Georgetown University
Washington D.c.
Massachusetts
Massachusetts General Hospital-Harvard University
Boston
Maryland
Johns Hopkins
Baltimore
Michigan
Memorial Healthcare
Owosso
Missouri
Washington University School of Medicine
St Louis
New York
New York University School of Medicine
New York
Texas
Houston Methodist Neurological Institute
Houston
Virginia
Children's Hospital of the King's Daughthers
Norfolk
Washington
University of Washington
Seattle
Other Locations
Canada
Montreal Neurological Institute and Hospital
Montreal
Time Frame
Start Date: 2018-08-16
Completion Date: 2025-01-01
Participants
Target number of participants: 148
Treatments
Cohort 1
This is a prospective, longitudinal, multi-center, observational study designed to evaluate the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) in ambulatory and non-ambulatory adult patients with SMA. Subjects with SMA II/III that are 18 years to 70 years of age who are planning to initiate treatment with SPINRAZA® (nusinersen) as part of their clinical care plan will be enrolled in this study. This study does not provide SPINRAZA® (nusinersen) or cover costs associated with standard clinical care.These patients will be treated by their respective physicians according to standard clinical practice. Study visits, some of which including standardized assessments of strength and function, will occur at baseline, day 15 after treatment initiation, day 30, day 60, and then 4-month intervals through month 30.
Cohort 2
The cohort 2 is a one time survey to gain a better understanding of this adult population and their treatment preferences.
Sponsors
Leads: Washington University School of Medicine

This content was sourced from clinicaltrials.gov