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Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA® (SMAII)

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ClinicalTrials.gov Identifier: NCT04159987
Recruitment Status : Not yet recruiting
First Posted : November 12, 2019
Last Update Posted : November 12, 2019
Sponsor:
Information provided by (Responsible Party):
Centre Hospitalier Universitaire de Nice

Tracking Information
First Submitted Date  ICMJE October 4, 2019
First Posted Date  ICMJE November 12, 2019
Last Update Posted Date November 12, 2019
Estimated Study Start Date  ICMJE November 30, 2019
Estimated Primary Completion Date June 30, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 8, 2019)
  • To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at baseline ]
  • To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at 1 month ]
  • To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at 3 months ]
  • To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® from baseline [ Time Frame: at 7 months ]
  • To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at 15 months ]
  • To monitor the evolution of the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at 27 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®
Official Title  ICMJE A Multicenter, Interventional, Open-label Study to Monitor the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®
Brief Summary SPINRAZA® (Nusinersen) is the first intrathecal administered drug which was approved by the FDA to treat SMA children and adults (2016). The aim is to monitor the evolution of the Motor Function Measure-32 for SMA type II adult patients treated with SPINRAZA® (Nusinersen).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Condition  ICMJE Spinal Muscular Atrophy
Intervention  ICMJE Other: Spinraza intrathecal injection
The aim is to monitor the evolution of the Motor Function Measure-32 for SMA type II adult patients treated with SPINRAZA® (Nusinersen).
Study Arms  ICMJE Experimental: Spinal muscular atrophy patient
Intervention: Other: Spinraza intrathecal injection
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: November 8, 2019)
20
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE November 30, 2022
Estimated Primary Completion Date June 30, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Adult patients with SMA type II disease who are wheelchair bound,
  • Must be 18 years or older,
  • Men or Women with SMA type II disease (age at symptoms onset: >6 months old and who never acquired the capacity to walk but acquired the ability to sit without support) with genetic confirmed diagnosis of 5q SMA homozygous gene deletion (SMN1 exon 7/8) or mutation or compound heterozygous mutation - performed by PCR amplification and restriction digest of DNA using primers flanking SMN1 and SMN2 exon 7.
  • MFM 32 score ≥ 19/96
  • Lumbar CT scan showing the feasibility of intrathecal injection.
  • Written informed consent from the subject prior to initiation of any study-mandated procedures
  • Females of childbearing potential must have a negative pregnancy test at Screening and at Enrollment, must agree to use reliable method of contraception (if sexually active) from screening up to study drug discontinuation plus 180 days.

Exclusion Criteria:

  • Patients with a high risk for thrombocytopenia or hemorrhage or renal diseases: Urine protein, platelet count and coagulation tests will be done prior to intrathecal injection with SPINRAZA®
  • Patients with high risk of hydrocephalus
  • Adult patients under guardianship
  • Pregnant and/or breastfeeding females
  • Subject has received any investigational therapy or pharmacological treatment for SMA one month prior the beginning of the study
  • Subject has received gene therapy for SMA
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Listed Location Countries  ICMJE Not Provided
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04159987
Other Study ID Numbers  ICMJE 19-PP-09
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Centre Hospitalier Universitaire de Nice
Study Sponsor  ICMJE Centre Hospitalier Universitaire de Nice
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Centre Hospitalier Universitaire de Nice
Verification Date September 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP