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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 11, 2019
Last Update Posted : November 11, 2019
Sponsor:
Information provided by (Responsible Party):
Centre Hospitalier Universitaire de Nice

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).

Condition or disease Intervention/treatment Phase
Spinal Muscular Atrophy Other: Spinraza intrathecal injection Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Other
Official Title: A Multicenter, Interventional, Open-label Study to Monitor the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA®
Estimated Study Start Date : November 30, 2019
Estimated Primary Completion Date : June 30, 2022
Estimated Study Completion Date : November 30, 2022


Arm Intervention/treatment
Experimental: Spinal muscular atrophy patient 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).




Primary Outcome Measures :
  1. To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at baseline ]
  2. To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at 1 month ]
  3. To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at 3 months ]
  4. 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 ]
  5. To monitor the Motor Function Measure-32 in adult wheelchair bound SMA type II patients treated with SPINRAZA® [ Time Frame: at 15 months ]
  6. 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 ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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Responsible Party: Centre Hospitalier Universitaire de Nice
ClinicalTrials.gov Identifier: NCT04159987     History of Changes
Other Study ID Numbers: 19-PP-09
First Posted: November 11, 2019    Key Record Dates
Last Update Posted: November 11, 2019
Last Verified: September 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Motor Neuron Disease
Muscular Atrophy
Muscular Atrophy, Spinal
Atrophy
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases