TRANE: Differential Study of Muscle Transcriptome

• Sponsor: Assistance Publique - Hôpitaux de Paris (Other)
• Overall Status: Completed
• CT.gov ID: NCT01984957
• Collaborator: Direction Générale de l'Offre de Soins (Other), Institut National de la Santé Et de la Recherche Médicale, France (Other)
• Enrollment: 42
• Location: 1
• Arm: 1
• Duration (Months): 24
• Patients Per Site Per Month: 1.8

Study Details

Study Description

Brief Summary

To isolate biomarkers of ALS in muscle

Condition or Disease	Intervention/Treatment	Phase
ALS; SMA; SBMA	Procedure: Muscle biopsy	N/A

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)

Study Design

• Study Type: Interventional
• Actual Enrollment: 42 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Diagnostic
• Official Title: Differential Study of Muscle Transcriptome in Patients With Neuromuscular Disease and Control Subjects
• Study Start Date: Jan 1, 2013
• Actual Primary Completion Date: Jan 1, 2015
• Actual Study Completion Date: Jan 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Other: disease (ALS, SMA or SBMA) muscle biopsy	Procedure: Muscle biopsy; open muscular biopsy of the deltoid under local anaesthesia

Outcome Measures

Primary Outcome Measures

1. Gene expression levels [day 1]

   Gene expression levels in muscle as determined By microarrays (transcriptome analysis)

Secondary Outcome Measures

1. muscle denervation [day 1]

   Severity of muscle denervation as determined by histological analysis of muscle

2. Functional evaluation [day 1]

   ALSFRS-R, Manual Muscle Testing and Slow Vital Capacity

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 75 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Centre d'évaluation Thérapeutique, Dr LACOMBLEZ	Paris		France	75013

Sponsors and Collaborators

• Assistance Publique - Hôpitaux de Paris
• Direction Générale de l'Offre de Soins
• Institut National de la Santé Et de la Recherche Médicale, France

Investigators

• Principal Investigator: Pierre-Francois Pradat, MD, PhD, APHP

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Assistance Publique - Hôpitaux de Paris
• ClinicalTrials.gov Identifier: NCT01984957
• Other Study ID Numbers: P110401
• First Posted: Nov 15, 2013
• Last Update Posted: Mar 23, 2015
• Last Verified: Jun 1, 2014

• Keywords provided by Assistance Publique - Hôpitaux de Paris: ALS; Motor neuron diseases; Transcriptome

Study Results

No Results Posted as of Mar 23, 2015