NCT00847847

Brief Summary

Consistent data suggest that neuromuscular transmission is impaired in ALS patients. Neuromuscular junctions dysfunction may appear very early in the disease, as shown by data in animal models. The pathogenesis of this neuromuscular transmission impairment is unknown. Nogo A isoform, a possible marker of the disease over-expressed in skeletal muscle of ALS patients, can be involved. We will characterize the pathophysiological mechanisms implicated using a complete study of the structure and function of the NMJ on muscle biopsies, in a group of 20 ALS patients compared to 10 controls.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
14

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Mar 2009

Typical duration 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

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 11, 2009

Completed
8 days until next milestone

First Posted

Study publicly available on registry

February 19, 2009

Completed
10 days until next milestone

Study Start

First participant enrolled

March 1, 2009

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2012

Completed
Last Updated

November 19, 2012

Status Verified

December 1, 2009

Enrollment Period

3.1 years

First QC Date

February 11, 2009

Last Update Submit

November 16, 2012

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALSMotor neuronPathophysiologyNeuromuscular junctionNeuromuscular transmissionMicroelectrodes

Outcome Measures

Primary Outcomes (1)

  • Characterization of the neuromuscular transmission dysfunction in ALS by studying the structural and functional features of NMJs on muscle biopsies

    within the 15 days after inclusion (month 0 = M0)

Secondary Outcomes (1)

  • Search for correlations between the results of the structural and functional study of neuromuscular junctions on muscle biopsy and surface-EMG and clinical data

    at M0, M3, M6

Study Arms (2)

2

OTHER

control subjects with muscle biopsy

Procedure: Anconeus Muscle biopsy

1

OTHER

ALS patients with muscle biopsy

Procedure: Anconeus Muscle biopsy

Interventions

Anconeus Muscle biopsyPROCEDURE

Anconeus muscle specimens will be surgically removed. The biopsy will be performed under regional anaesthesia and will require an about 5 cm incision of the skin and muscle fascia from the lateral condyle to over the ridge of the proximal ulna, 3 or 4 cm distal to the tip of the olecranon. A triangular muscle flap will be removed, and then the fascia and skin will be closed with running dissolving suture.

12

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Aged 18 to 75 (inclusive)
  • Possible, probable (clinically or laboratory) or definite ALS according to the revised El Escorial criteria
  • Duration of the disease of less then 12 months
  • Willing and able to provide a written informed consent
  • With french social insurance affiliation

You may not qualify if:

  • Cognitive changes or psychiatric condition, inability to give informed consent
  • patient unable to contact or to be contacted by the investigator in case of emergency
  • women who are pregnant or nursing
  • concomitant medication contraindicating muscular biopsy (platelet suppressive agents if treatment can not medically be stopped 2 weeks before surgical procedure, oral anticoagulant therapy)
  • medical condition contraindicating muscular biopsy (hypo-coagulative disease, allergy to anaesthetic drugs)
  • medical condition susceptible to influence on EMG examination (concomitant neurological or rheumatological disease)
  • Controls:
  • adult patients (minimum 18y) without neuromuscular disease
  • undergoing elbow surgery for local joint or bone disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Bruneteau Gaelle

Paris, 75003, France

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Gaelle Bruneteau, MD

    Assistance Publique - Hôpitaux de Paris

    PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

First Submitted

February 11, 2009

First Posted

February 19, 2009

Study Start

March 1, 2009

Primary Completion

April 1, 2012

Study Completion

April 1, 2012

Last Updated

November 19, 2012

Record last verified: 2009-12

Locations

FR(1)