Spinraza in Adult Spinal Muscular Atrophy

Study Identifier:
201805187
ClinicalTrials.gov Identifier:
NCT03709784
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Washington University School of Medicine
Active, not recruiting

Study Details

Medical Condition
  • All Spinal Muscular Atrophies
  • Spinal Muscular Atrophy Type II
  • Spinal Muscular Atrophy Type III
Study Drug
  • Drug: Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care
Date
Aug 2018 - Dec 2024
Patient Requirements
Sex: Female & Male
Age: 18 - 70 Years
Requirements Information
Inclusion and Exclusion Criteria
Inclusion Criteria
  • 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.
Exclusion Criteria
  • Revised upper limb score ≤ 3.
  • Respiratory insufficiency, defined by the medical necessity for invasive or noninvasive ventilation for \>16 hours during a 24-hour period, at screening.
  • Hospitalization for major medical event including: surgery (i.e., scoliosis surgery, other surgery), cardiac event, pulmonary event, or other major medical problem within 2 months of screening or planned major surgical procedure likely to impact the clinical assessments during the duration of the study. Outpatient surgical procedure (i.e., placement of feeding tube) is not considered an exclusionary major medical event.
  • Presence of a symptomatic severe active infection or illness during the screening period that is likely to impact the performance on the clinical assessments.
  • Prior exposure to SPINRAZA® (nusinersen).
  • Prior disorder, injury (e.g., upper or lower limb fracture) or surgical procedure which impacts the subject's ability to perform any of the outcome measure testing required in the protocol and from which the subject has not fully recovered or achieved a stable baseline.
  • Treatment with an investigational drug (e.g., oral albuterol/salbutamol, riluzole, carnitine, creatine, sodium phenylbutyrate, etc.), biological agent, or device within 1-month of Screening or 5 half-lives of study agent, whichever is longer. Patients using intrathecal drug delivery devices, including investigational devices with an active IDE designation in the United States, may be eligible but require Study PI approval prior to enrollment.
  • Any history of exposure to gene therapy, antisense oligonucleotide therapy, or cell transplantation that was intended for the treatment of SMA.
  • Ongoing medical condition that according to the Clinical Center Investigator would interfere with the conduct and assessments of the study. Examples are medical disability (e.g., wasting or cachexia, severe anemia, etc.) that would interfere with the assessment of safety or would compromise the ability of the subject to undergo study procedures.
  • For Cohort 2 Inclusion criteria
  • 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.
  • Believed to be able to complete the structured interview.
  • Cohort 2 Exclusion Criteria
  • 1\. Ongoing medical condition that according to the Clinical Center Investigator would interfere with the conduct and assessments of the study.

Protocol 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.

Trial Locations

Location
Status
Location
Barrow Neurological Institute
Phoenix, Arizona, United States, 85013
Status
N/A
Location
Georgetown University
Washington D.C., District of Columbia, United States, 20007
Status
N/A
Location
Johns Hopkins
Baltimore, Maryland, United States, 21287
Status
N/A
Location
Massachusetts General Hospital-Harvard University
Boston, Massachusetts, United States, 02114
Status
N/A
Location
Memorial Healthcare
Owosso, Michigan, United States, 48867
Status
N/A
Location
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Status
N/A
Go to page

Contact Cure SMA

User Information

If you have general questions regarding clinical trials or would like to be added to the Cure SMA mailing list to receive communications regarding future registries, studies, or clinical trials in SMA, please fill out the form below. Fields marked with an asterisk (*) are mandatory.

  • Disability Advocate who does not have SMA
  • I am a healthcare provider
  • I am a researcher
  • I have no direct connection to SMA
  • I have SMA
  • I lost a child to SMA
  • My child has SMA
  • Someone close to me has/had SMA
  • Australia
  • Belgium
  • Brazil
  • Canada
  • Chile
  • China
  • Colombia
  • Croatia
  • Czech Republic
  • Denmark
  • Estonia
  • France
  • Germany
  • Greece
  • Hong Kong
  • Hungary
  • India
  • Ireland
  • Israel
  • Italy
  • Japan
  • Lebanon
  • Malaysia
  • Mexico
  • Netherlands
  • Norway
  • Poland
  • Portugal
  • Puerto Rico
  • Qatar
  • Republic of Korea
  • Romania
  • Russian Federation
  • Saudi Arabia
  • Serbia
  • Singapore
  • South Africa
  • Spain
  • Sweden
  • Switzerland
  • Taiwan, Province of China
  • Thailand
  • Ukraine
  • United Kingdom
  • United States
  • Vietnam
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.