Differential Study of Muscle Transcriptome | NCT01984957

Studies
Differential Study of Muscle Transcriptome

NCT01984957

Sponsor

Assistance Publique - Hôpitaux de Paris

Status

Completed

Last Update Posted

Mar 23, 2015Estimated

Enrollment

42Actual

Phase

Not Applicable

Conditions

ALS
SMA
SBMA

Interventions

Muscle biopsy

Countries

France

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jun 2014

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jan 2013

Primary Completion Date

Jan 2015Actual

Completion Date

Jan 2015Actual

First Submitted Date

Jun 27, 2013

First Submission Date that Met QC Criteria

Nov 7, 2013

First Posted Date

Nov 15, 2013Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 20, 2015

Last Update Posted Date

Mar 23, 2015Estimated

Sponsor/Collaborators Module

Responsible Party, by Official Title

Sponsor

Lead Sponsor

Assistance Publique - Hôpitaux de ParisOTHER

Collaborators

Name	Class
Direction Générale de l'Offre de Soins	OTHER_GOV
Institut National de la Santé Et de la Recherche Médicale, France	OTHER_GOV

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

To isolate biomarkers of ALS in muscle

Detailed Description

At present, there is no validated biological diagnostic marker for amyotrophic lateral sclerosis (ALS). The main objective of the study is to isolate a set of genes that are specifically dysregulated in muscle from ALS patients.

Gene profiling in muscle will be compared between ALS patients and patients with other motor neuron diseases (SMA and SBMA)

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

42Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
disease (ALS, SMA or SBMA)	Other	muscle biopsy	Procedure: Muscle biopsy

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Muscle biopsy	Procedure	open muscular biopsy of the deltoid under local anaesthesia	disease (ALS, SMA or SBMA)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Gene expression levels	Gene expression levels in muscle as determined By microarrays (transcriptome analysis)	day 1

Secondary Outcomes

Measure	Description	Time Frame
muscle denervation	Severity of muscle denervation as determined by histological analysis of muscle	day 1
Functional evaluation	ALSFRS-R, Manual Muscle Testing and Slow Vital Capacity

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion criteria :

- Patient with ALS, SMA, or SBMA

Exclusion criteria :

Indication against biopsy (allergy to anesthetics local haemostasis disorders)
Treatments that can affect muscle metabolism

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

75 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Pierre-Francois Pradat, MD, PhD	APHP	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Centre d'évaluation Thérapeutique, Dr LACOMBLEZ		Paris		75013	France

Condition Browse Module

MeSH Terms

ID	Term
D016472	Motor Neuron Disease

Ancestor Terms

ID	Term
D019636	Neurodegenerative Diseases
D009422	Nervous System Diseases
D009468	Neuromuscular Diseases

Browse Leaves

Not provided

Browse Branches

Not provided