Amyotrophic Lateral SclerosisPhase 1NCT01759784
Intraventricular Transplantation of Mesenchymal Stem Cell in Patients With ALS
A Phase 1 study testing Intraventricular injection in people with Amyotrophic Lateral Sclerosis, run by Royan Institute at 1 site in Iran.
Trial summary
ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Intraventricular injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteIran
- Study dates
- Mar 2014to Mar 2017
- Phase
- Phase 1
- 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 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age:18-65
- both gender
- duration of disease<2 years
- FVC>40% ALS-FRS>26
Who cannot join
- neurological and psychiatric concomitant disease
- concomitant systemic disease
- treatment with corticosteroid,Ig,immunosuppressive during 12 months.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety of Intraventricular Injection of Bone Marrow Derived Mesenchymal Stem Cell in Patients With ALS
ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). ALS is the most common of the five motor neuron diseases.Riluzole (Rilutek) is the only treatment that has been found to improve survival but only to a modest extent. It lengthens survival by several months, and may have a greater survival benefit for those with a bulbar onset. It also extends the time before a person needs ventilation support.Stem cell transplantation is a new hopeful way to improve the patients conditions and reduce the period of disabilities.
Read the detailed description
In this study our purpose is to evaluate the safety of intraventricular injection of bone marrow derived mesenchymal stem cell.the patients who are eligible,underwent bone marrow aspiration.after cell separation and preparation,the patients underwent mesenchymal stem cell intraventricular injection by stereotaxis.after injection he will be under observed in ICU to monitor the adverse events(allergic and neurological side effects).patients are followed 1th,3th ,6th and 12 months after injection and each time these parameters are checked:ALS-FRS,EMG-NCV,FVC,side effect check list.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Intraventricular injection
- Keywords
- mesenchymal stem cell intraventricular injection ALS
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intraventricular injectionBiological
Also called intraventricular injection of mesenchymal stem cell by stereotaxis
intraventricular injection of mesenchymal stem cell by stereo taxi in patients with ALS
| Study group | Role | What they receive |
|---|---|---|
| stem cell recipient | Experimental | The patients who underwent mesenchymal stem cell transplantation. Receives: Biological: Intraventricular injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
fever
evaluation the rate of fever 48hours after stem cell injection
Measured: 48hours
unconsciousness
evaluation the rate of unconsciousness during 6months after stem cell injection.
Measured: 6months
brain hematoma
Evaluation the rate of hematoma 48hours after stem cell injection.
Measured: 48hours
vomiting
evaluation the rate of vomiting 48hours after stem cell injection.
Measured: 48hours
Secondary outcomes (2)
ALS-FRS
evaluation the improvement of ALS-FRS 6months after stem cell injection.
Measured: 6months
EMG-NCV
evaluation the improvement of EMG-NCV after stem cell injection.
Measured: 6months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Jan 2013 (estimated)
- Study startMar 2014
- Primary completionMar 2017 (estimated)
Why it stopped: The stereotaxis can be threatful for the patients because of respiratory insufficiency
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Royan Institute
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Hamid Gourabi, PhD, Study chair, Head of Royan InstituteNasser Aghdami, MD,PhD, Study director, Head of Royan department of degenerative medicine,Head of Royan celltherapy centerAli Reza Zali, MD, Study director, Head of Neurosurgery research center of Shahid Beheshti University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01759784
- Sponsor's protocol ID
- Royan-Nerve-004
- Status last verified
- May 2012
- First submitted
- 29 Dec 2012
- Last updated
- 1 Jan 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 1 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety of Intraventricular Injection of Bone Marrow Derived Mesenchymal Stem Cell in Patients With ALS
- ClinicalTrials.gov ID
- NCT01759784
- Acronym / sponsor ID
- Royan-Nerve-004
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- mesenchymal stem cell intraventricular injection ALS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The stereotaxis can be threatful for the patients because of respiratory insufficiency
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Royan InstituteOther government
- Study start
- Mar 2014
- Primary completion
- Mar 2017 (estimated)
- First posted
- 3 Jan 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.
ALS is a debilitating disease with varied etiology characterized by rapidly progressive weakness, muscle atrophy and fasciculations, muscle spasticity, difficulty speaking (dysarthria), difficulty swallowing (dysphagia), and difficulty breathing (dyspnea). ALS is the most common of the five motor neuron diseases.Riluzole (Rilutek) is the only treatment that has been found to improve survival but only to a modest extent. It lengthens survival by several months, and may have a greater survival benefit for those with a bulbar onset. It also extends the time before a person needs ventilation support.Stem cell transplantation is a new hopeful way to improve the patients conditions and reduce the period of disabilities.
Detailed description
In this study our purpose is to evaluate the safety of intraventricular injection of bone marrow derived mesenchymal stem cell.the patients who are eligible,underwent bone marrow aspiration.after cell separation and preparation,the patients underwent mesenchymal stem cell intraventricular injection by stereotaxis.after injection he will be under observed in ICU to monitor the adverse events(allergic and neurological side effects).patients are followed 1th,3th ,6th and 12 months after injection and each time these parameters are checked:ALS-FRS,EMG-NCV,FVC,side effect check list.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| stem cell recipient | Experimental | Biological: Intraventricular injectionThe patients who underwent mesenchymal stem cell transplantation. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Age:18-65
- both gender
- duration of disease<2 years
- FVC>40% ALS-FRS>26
Key exclusion criteria (3 listed)
- neurological and psychiatric concomitant disease
- concomitant systemic disease
- treatment with corticosteroid,Ig,immunosuppressive during 12 months.
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 |
|---|---|---|
| Intraventricular injectionAlso: intraventricular injection of mesenchymal stem cell by stereotaxisintraventricular injection of mesenchymal stem cell by stereo taxi in patients with ALS | Biological | stem cell recipient |
Primary outcome
fever
Time frame: 48hours
evaluation the rate of fever 48hours after stem cell injection
unconsciousness
Time frame: 6months
evaluation the rate of unconsciousness during 6months after stem cell injection.
brain hematoma
Time frame: 48hours
Evaluation the rate of hematoma 48hours after stem cell injection.
vomiting
Time frame: 48hours
evaluation the rate of vomiting 48hours after stem cell injection.
2 secondary outcomes
ALS-FRS
Time frame: 6months
EMG-NCV
Time frame: 6months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jan 2016.
- Related InfoLink provided by the sponsor.
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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.
Hamid Gourabi, PhD study chair
Head of Royan Institute
Nasser Aghdami, MD,PhD study director
Head of Royan department of degenerative medicine,Head of Royan celltherapy center
Ali Reza Zali, MD study director
Head of Neurosurgery research center of Shahid Beheshti University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intraventricular injection in people with Amyotrophic Lateral Sclerosis, run by Royan Institute at 1 site in Iran.
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 4 inclusion and 3 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: withdrawn, as of the sponsor's last update on 1 Jan 2016. 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 Iran. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
fever (time frame: 48hours). The study lists 4 primary outcomes in all. 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 1 Jan 2016.