Overview

This trial is active, not recruiting.

Condition hibm
Treatments motor function and strength assessment, nmr assessment, 24h urine and serum collection
Sponsor Institut de Myologie, France
Start date April 2014
End date April 2018
Trial size 30 participants
Trial identifier NCT02196909, ClinBio-GNE

Summary

The objective of the study is to identify the best clinical and biological outcome measures for further therapeutics approaches.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation non-randomized
Intervention model parallel assignment
Masking open label
Primary purpose basic science
Arm
(Active Comparator)
motor function, muscle strength, NMR, 24h urine and serum collections at baseline, then annually
motor function and strength assessment
nmr assessment
24h urine and serum collection
(Active Comparator)
motor function, muscle strength, 24h urine and serum collections at baseline only
motor function and strength assessment
24h urine and serum collection

Primary Outcomes

Measure
Measure of changes in motor function and muscle strength assessment between 2 time points
time frame: baseline, 1 year, 2 years, 3 years
Measure of changes in NMR upper and lower limbs assessment between 2 timepoints(not for controls)
time frame: baseline, 1 year, 2 years, 3 years
24h urine and serum collection, measures at different time points
time frame: baseline, 1 year, 2 years, 3 years

Eligibility Criteria

Male or female participants from 18 years up to 65 years old.

Inclusion Criteria: - Must be at least 18 years of age. - Must be willing and able to provide consent. - Must have a genetic diagnosis of HIBM, GNE myopathy, Quadriceps Sparing Myopathy (QSM), Inclusion Body Myopathy Type 2, distal myopathy with rimmed vacuoles (DMRV), or Nonaka disease. - Must be willing and able to comply with all study requirements. - Affiliated to or a beneficiary of a social security category - Must take part in the HIBM-PMP UX001-CL401 study Exclusion Criteria: - Received ManNAc therapy or other similar substance - Any unrelated, comorbid disease or condition that, in the view of the investigator, would interfere with study participation or would affect safety. - Patients with specific contraindication to MRI (i.e. metallic foreign body, claustrophobia…) will be allowed to participate, but MRI will not be performed. - Pregnant women.

Trial information was received from ClinicalTrials.gov and was last updated in November 2015.
Information provided to ClinicalTrials.gov by Institut de Myologie, France.