NCT02196909

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jul 2014

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 8, 2014

Completed
3 months until next milestone

Study Start

First participant enrolled

July 1, 2014

Completed
21 days until next milestone

First Posted

Study publicly available on registry

July 22, 2014

Completed
3.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2018

Completed
Last Updated

August 8, 2018

Status Verified

August 1, 2018

Enrollment Period

3.8 years

First QC Date

April 8, 2014

Last Update Submit

August 7, 2018

Conditions

HIBM

Keywords

HIBMGNE

Outcome Measures

Primary Outcomes (3)

  • Measure of changes in motor function and muscle strength assessment between 2 time points

    baseline, 1 year, 2 years, 3 years

  • Measure of changes in NMR upper and lower limbs assessment between 2 timepoints(not for controls)

    baseline, 1 year, 2 years, 3 years

  • 24h urine and serum collection, measures at different time points

    baseline, 1 year, 2 years, 3 years

Study Arms (2)

HIBM patient

ACTIVE COMPARATOR

motor function, muscle strength, NMR, 24h urine and serum collections at baseline, then annually

Other: motor function and strength assessmentOther: NMR assessmentOther: 24h urine and serum collection

Controls

ACTIVE COMPARATOR

motor function, muscle strength, 24h urine and serum collections at baseline only

Other: motor function and strength assessmentOther: 24h urine and serum collection

Interventions

motor function and strength assessmentOTHER
ControlsHIBM patient
NMR assessmentOTHER
HIBM patient
24h urine and serum collectionOTHER
ControlsHIBM patient

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Must be at least 18 years of age.
  • Must be willing and able to provide consent.
  • 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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Institute of Myology

Paris, 75013, France

Location

MeSH Terms

Interventions

Urination

Intervention Hierarchy (Ancestors)

Urinary Tract Physiological PhenomenaReproductive and Urinary Physiological Phenomena

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 8, 2014

First Posted

July 22, 2014

Study Start

July 1, 2014

Primary Completion

May 1, 2018

Study Completion

May 1, 2018

Last Updated

August 8, 2018

Record last verified: 2018-08

Locations

FR(1)