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Prospective Study of the Natural History of Patients With Type 2 and 3 Spinal Muscular Atrophy

NCT02391831 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 81

Last updated 2018-07-19

No results posted yet for this study

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.

Conditions

  • Type 2 Spinal Muscular Atrophy
  • Type 3 Spinal Muscular Atrophy

Interventions

OTHER

Strength, function and activity measurements

OTHER

Muscle MRI

OTHER

Electrophysiology measurements

OTHER

Blood sampling for biomarker analysis

Sponsors & Collaborators

  • Institut Roche

    collaborator UNKNOWN

  • Institut de Myologie, France

    lead OTHER

Principal Investigators

  • Laurent Servais, MD · Association Institut de Myologie

Study Design

Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
30 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-05-31
Primary Completion
2018-06-30
Completion
2018-06-30

Countries

  • Belgium
  • France
  • Germany

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02391831 on ClinicalTrials.gov