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Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy (NatHis-SMA)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02391831
Recruitment Status : Completed
First Posted : March 18, 2015
Last Update Posted : July 19, 2018
Sponsor:
Collaborator:
Institut Roche
Information provided by (Responsible Party):
Institut de Myologie, France

Tracking Information
First Submitted Date  ICMJE March 2, 2015
First Posted Date  ICMJE March 18, 2015
Last Update Posted Date July 19, 2018
Study Start Date  ICMJE May 2015
Actual Primary Completion Date June 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 12, 2015)
  • Change from baseline of muscle strength [ Time Frame: Baseline and then every 6 months until end of the study, up to 24 months ]
    Study-specific assessments: Grip and pinch strength
  • Change from baseline of motor function [ Time Frame: Baseline and then every 6 months until end of the study, up to 24 months ]
    Study-specific assessments: Moviplate and MFM scores, upper extremity functional reaching volume, timed tests (time to rise from floor, time to walk 10 meters, time to climb and descend stairs, distance walked on the Six-Minute Walk Test)
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 12, 2015)
  • Change from baseline of respiratory function [ Time Frame: Baseline and then every 6 months until end of the study, up to 24 months ]
    Study-specific assessments: Pulmonary function tests
  • Change from baseline of physical activity of upper limbs movements [ Time Frame: Baseline and then every 6 months until end of the study, up to 24 months ]
    Quantity and duration of movements, time of inactivity during the day
  • Change from baseline of skeletal muscle nuclear magnetic resonance (NMR) imaging (MRI) [ Time Frame: Baseline and then every 12 months until the end of the study, up to 24 months ]
    Muscle volume changes, intramuscular fatty infiltration progression, indices of disease activity (only for Paris and Strasbourg sites and for patients older than 4 years)
  • Change from baseline of electrophysiology measurements [ Time Frame: Baseline and then every 6 months until end of the study, up to 24 months ]
    Compound Motor Action Potential (CMAP) Amplitude and Decrement search
  • Change from baseline of Biomarkers of SMA progression [ Time Frame: Baseline and then every 6 months until end of the study, up to 24 months ]
    SMN mRNA and protein analysis, SMA exploratory biomarkers (e.g. mRNA, DNA profiling, RNA profiling, proteomic profiling)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
Official Title  ICMJE Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy
Brief Summary NatHis-SMA is a prospective, longitudinal and interventional study of the natural history of patients with type 2 and 3 Spinal Muscular Atrophy (SMA). The purpose of this study is to characterize the disease course over 2 years and identify prognostic variables of the disease and biomarkers of SMA progression, as well as determine the best outcome measures for further therapeutics approaches.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Condition  ICMJE
  • Type 2 Spinal Muscular Atrophy
  • Type 3 Spinal Muscular Atrophy
Intervention  ICMJE
  • Other: Strength, function and activity measurements
  • Other: Muscle MRI
  • Other: Electrophysiology measurements
  • Other: Blood sampling for biomarker analysis
Study Arms  ICMJE Not Provided
Publications * Chabanon A, Seferian AM, Daron A, Péréon Y, Cances C, Vuillerot C, De Waele L, Cuisset JM, Laugel V, Schara U, Gidaro T, Gilabert S, Hogrel JY, Baudin PY, Carlier P, Fournier E, Lowes LP, Hellbach N, Seabrook T, Toledano E, Annoussamy M, Servais L; NatHis-SMA study group. Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study. PLoS One. 2018 Jul 26;13(7):e0201004. doi: 10.1371/journal.pone.0201004. eCollection 2018.

*   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 Completed
Actual Enrollment  ICMJE
 (submitted: May 11, 2016)
81
Original Estimated Enrollment  ICMJE
 (submitted: March 12, 2015)
70
Actual Study Completion Date  ICMJE June 2018
Actual Primary Completion Date June 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

INCLUSION CRITERIA

  • Type 2 or 3 spinal muscular atrophy genetically confirmed
  • Age superior or equal to 2 years old up to 30 years of age included
  • For patients older than 6 years old, willing and able to comply with all protocol requirements and procedures.
  • For non-ambulant patients, able to sit upright in a wheelchair for at least three hours
  • Patients over 18 years of age and parent(s)/legal guardian(s) of patients < 18 years of age must provide written informed consent prior to participating in the study and informed assent will be obtained from minors at least 7 years of age when required by regulation.
  • In France only: Affiliated to or a beneficiary of a social security category

EXCLUSION CRITERIA

  • Previously treated with an investigational drug within 6 months prior the recruitment in this study.
  • Other condition which may significantly interfere with the assessment of the SMA and is clearly not related to the disease
  • Current or anticipated participation in any therapeutic investigational clinical studies.
  • Patients with specific contraindication to MRI (i.e. metallic foreign body, claustrophobia, and others deemed to be prohibitive by the investigators) will be allowed to participate, but MRI will not be performed.
  • For women : pregnancy or current breastfeeding
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 30 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   France,   Germany
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02391831
Other Study ID Numbers  ICMJE NatHis-SMA
IDRCB-2014-A01263-44 ( Other Identifier: ANSM (French Regulatory Authority) )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Institut de Myologie, France
Study Sponsor  ICMJE Institut de Myologie, France
Collaborators  ICMJE Institut Roche
Investigators  ICMJE
Principal Investigator: Laurent Servais, MD Association Institut de Myologie
PRS Account Institut de Myologie, France
Verification Date July 2018

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