ALSTRANENCT01984957
Differential Study of Muscle Transcriptome
A study testing Muscle biopsy in people with ALS and SMA, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
To isolate biomarkers of ALS in muscle Read more about this study
- Who is it for?
- Ages 18 to 75 years
- What is being tested?
- Muscle biopsy
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteFrance
- Study dates
- Jan 2013to Jan 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient with ALS, SMA, or SBMA
Who cannot join
- Indication against biopsy (allergy to anesthetics local haemostasis disorders)
- Treatments that can affect muscle metabolism
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Differential Study of Muscle Transcriptome in Patients With Neuromuscular Disease and Control Subjects
To isolate biomarkers of ALS in muscle
Read the 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)
- Being tested
- Muscle biopsy
- Keywords
- ALS, Motor neuron diseases, Transcriptome
- Medical subject headings
- Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Muscle biopsyProcedure or surgery
open muscular biopsy of the deltoid under local anaesthesia
| Study group | Role | What they receive |
|---|---|---|
| disease (ALS, SMA or SBMA) | Other | muscle biopsy Receives: Procedure: Muscle biopsy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Gene expression levels
Gene expression levels in muscle as determined By microarrays (transcriptome analysis)
Measured: day 1
Secondary outcomes (2)
muscle denervation
Severity of muscle denervation as determined by histological analysis of muscle
Measured: day 1
Functional evaluation
ALSFRS-R, Manual Muscle Testing and Slow Vital Capacity
Measured: day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2013 (estimated)
- Study startJan 2013
- Primary completionJan 2015
- Study completionJan 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALSStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Collaborators
- Direction Générale de l'Offre de Soins, Institut National de la Santé Et de la Recherche Médicale, France
- Responsible party
- Sponsor
- Investigators
- Pierre-Francois Pradat, MD, PhD, Principal investigator, APHP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01984957
- Sponsor's protocol ID
- P110401
- Acronym
- TRANE
- Status last verified
- Jun 2014
- First submitted
- 27 Jun 2013
- Last updated
- 23 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Differential Study of Muscle Transcriptome in Patients With Neuromuscular Disease and Control Subjects
- ClinicalTrials.gov ID
- NCT01984957
- Acronym / sponsor ID
- TRANE / P110401
- Keywords
- ALS, Motor neuron diseases, Transcriptome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Collaborators
- Direction Générale de l'Offre de Soins, Institut National de la Santé Et de la Recherche Médicale, France
- Study start
- Jan 2013
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 15 Nov 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| disease (ALS, SMA or SBMA) | Other | Procedure: Muscle biopsymuscle biopsy |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Patient with ALS, SMA, or SBMA
Key exclusion criteria (2 listed)
- Indication against biopsy (allergy to anesthetics local haemostasis disorders)
- Treatments that can affect muscle metabolism
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Muscle biopsyopen muscular biopsy of the deltoid under local anaesthesia | Procedure or surgery | disease (ALS, SMA or SBMA) |
Primary outcome
Gene expression levels
Time frame: day 1
Gene expression levels in muscle as determined By microarrays (transcriptome analysis)
2 secondary outcomes
muscle denervation
Time frame: day 1
Functional evaluation
Time frame: day 1
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pierre-Francois Pradat, MD, PhD principal investigator
APHP
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Muscle biopsy in people with ALS and SMA, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Gene expression levels (time frame: day 1). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2015.