Amyotrophic Lateral SclerosisPhase 1/2NCT01254539
Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
A Phase 1/2 study testing Laminectomy and bone marrow stem cells transplantation and Intrathecal infusion of autologous bone marrow stem cells in people with Amyotrophic Lateral Sclerosis, run by Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia at 1 site in Spain.
Trial summary
The purpose of this clinical trial is to assess the feasibility and the security of the intraspinal and intrathecal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients. 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?
- Laminectomy and bone marrow stem cells transplantation and Intrathecal infusion of autologous bone marrow stem cells
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 63 participants
- Where
- 1 siteSpain
- Study dates
- Oct 2010to 20 Nov 2015
- Phase
- Phase 1/2
- 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
- Diagnose established following the World Federation of Neurology criteria
- More than 6 and less than 36 months of evolution of the disease
- Medullar onset of the disease
- More than 18 and less than 70 years old
- Forced Vital Capacity ≥ 50%
- Total time of oxygen saturation <90% inferior to 5% of the sleeping time
- Signed informed consent
- Unability to understand the informed consent
Who cannot join
- Neurological or psychiatric concomitant disease
- Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube
- Concomitant systemic disease
- Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 12 months
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase I/II Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
The purpose of this clinical trial is to assess the feasibility and the security of the intraspinal and intrathecal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients.
Read the detailed description
Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intraspinal injections of bone marrow mononuclear cells (MNC) have been able to ameliorate the course of ALS in murine models, acting as pumps of trophic factors that keep the motoneurons functional. Moreover, the clinical trial (Study NCT00855400 on www.ClinicalTrials.gov) conducted by our research group to determine the safety and efficacy of Autologous Stem Cell transplantation in Amyotrophic Lateral Sclerosis in humans, found that this procedure is feasible and safe. Continuing with that study, we have designed a phase I/II clinical trial to check the feasibility of the intraspinal and intrathecal infusion of autologous bone marrow stem cells.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Laminectomy and bone marrow stem cells transplantation, Intrathecal infusion of autologous bone marrow stem cells, Intrathecal infusion of placebo (saline solution).
- Keywords
- Amyotrophic Lateral Sclerosis, Bone marrow, Stem cells, Autologous, Safety
- 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.
Laminectomy and bone marrow stem cells transplantationProcedure or surgery
Autologous bone marrow cells collection under sedation. Sixty ml are obtained and processed through a ficoll gradient.
T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation
Intrathecal infusion of autologous bone marrow stem cellsProcedure or surgery
Autologous bone marrow cells collection under sedation. Sixty ml are obtained and processed through a ficoll gradient.
Patients were drawn 2 ml of cerebrospinal fluid and infused 2 ml (two 1 ml syringes) of Autologous Stem Cells.
Intrathecal infusion of placebo (saline solution).Procedure or surgery
Patients were infused 2 ml of saline solution
| Study group | Role | What they receive |
|---|---|---|
| Autologous bone marrow stem cells intraspinal transplantation | Experimental | T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation Receives: Procedure: Laminectomy and bone marrow stem cells transplantation |
| Intrathecal infusion of autologous bone marrow stem cells | Experimental | Patients were drawn 2 ml of cerebrospinal fluid and infused 2 ml (two 1 ml syringes) of Autologous Stem Cells. Receives: Procedure: Intrathecal infusion of autologous bone marrow stem cells |
| Intrathecal infusion of placebo (saline solution). | Placebo comparator | Patients were infused 2 ml of saline solution Receives: Procedure: Intrathecal infusion of placebo (saline solution). |
What is being measured
The primary outcome is the main question the study is designed to answer.
Forced vital capacity
Measured: Every 3 months
Secondary outcomes (6)
Neurological variables: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS), Medical Research Council (MRC) and Norris scales
Measured: Every 3 months
Absence of adverse events
Measured: Every week / month depending on the study phase
Neurophysiological variables: Electromyography, polysomnography, evoked potentials
Measured: Every 3 months
Neuroradiological variables: Spinal Magnetic Resonance Imaging (MRI)
Measured: Every 3 months
Respiratory variables: Maximal inspiratory pressure (PIM), Maximal expiratory pressure (PEM), sniff nasal, oxymetry.
Measured: Every 3 months
Psychological variables: Health Questionnaire (EuroQol-5D), The Profile of Mood States (POMS)
Measured: Every 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2010 (estimated)
- Study startOct 2010
- Primary completion20 Nov 2015
- Study completion20 Nov 2015
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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 63 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
- Sponsor type
- University, hospital or other
- Collaborators
- Instituto de Salud Carlos III, Hospital Universitario Virgen de la Arrixaca, Hospital General Universitario Morales Meseguer, Diógenes Foundation
- Responsible party
- Sponsor
- Investigators
- Jose María Moraleda Jiménez, M.D. PhD., Study director, Hospital Universitario Virgen de la Arrixaca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01254539
- Sponsor's protocol ID
- Extension CMN/ELA
- Other IDs
- 2006-003096-12, EC07/90762
- Status last verified
- Nov 2015
- First submitted
- 3 Dec 2010
- Last updated
- 30 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
- ClinicalTrials.gov ID
- NCT01254539
- Acronym / sponsor ID
- Extension CMN/ELA
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Amyotrophic Lateral Sclerosis, Bone marrow, Stem cells, Autologous, Safety
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 63 participants (actual)
- Lead sponsor
- Fundacion para la Formacion e Investigacion Sanitarias de la Region de MurciaUniversity, hospital or other
- Collaborators
- Instituto de Salud Carlos III, Hospital Universitario Virgen de la Arrixaca, Hospital General Universitario Morales Meseguer, Diógenes Foundation
- Study start
- Oct 2010
- Primary completion
- 20 Nov 2015 (actual)
- Study completion
- 20 Nov 2015 (actual)
- First posted
- 6 Dec 2010 (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.
The purpose of this clinical trial is to assess the feasibility and the security of the intraspinal and intrathecal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients.
Detailed description
Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intraspinal injections of bone marrow mononuclear cells (MNC) have been able to ameliorate the course of ALS in murine models, acting as pumps of trophic factors that keep the motoneurons functional. Moreover, the clinical trial (Study NCT00855400 on www.ClinicalTrials.gov) conducted by our research group to determine the safety and efficacy of Autologous Stem Cell transplantation in Amyotrophic Lateral Sclerosis in humans, found that this procedure is feasible and safe. Continuing with that study, we have designed a phase I/II clinical trial to check the feasibility of the intraspinal and intrathecal infusion of autologous bone marrow stem cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Autologous bone marrow stem cells intraspinal transplantation | Experimental | Procedure: Laminectomy and bone marrow stem cells transplantationT3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation |
| Intrathecal infusion of autologous bone marrow stem cells | Experimental | Procedure: Intrathecal infusion of autologous bone marrow stem cellsPatients were drawn 2 ml of cerebrospinal fluid and infused 2 ml (two 1 ml syringes) of Autologous Stem Cells. |
| Intrathecal infusion of placebo (saline solution). | Placebo comparator | Procedure: Intrathecal infusion of placebo (saline solution).Patients were infused 2 ml of saline solution |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Diagnose established following the World Federation of Neurology criteria
- More than 6 and less than 36 months of evolution of the disease
- Medullar onset of the disease
- More than 18 and less than 70 years old
- Forced Vital Capacity ≥ 50%
Key exclusion criteria (4 listed)
- Neurological or psychiatric concomitant disease
- Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube
- Concomitant systemic disease
- Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 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 |
|---|---|---|
| Laminectomy and bone marrow stem cells transplantationAutologous bone marrow cells collection under sedation. Sixty ml are obtained and processed through a ficoll gradient. T3-T4 laminectomy and bone marrow ficoll separated mononuclear autologous cells intraspinal transplantation | Procedure or surgery | Autologous bone marrow stem cells intraspinal transplantation |
| Intrathecal infusion of autologous bone marrow stem cellsAutologous bone marrow cells collection under sedation. Sixty ml are obtained and processed through a ficoll gradient. Patients were drawn 2 ml of cerebrospinal fluid and infused 2 ml (two 1 ml syringes) of Autologous Stem Cells. | Procedure or surgery | Intrathecal infusion of autologous bone marrow stem cells |
| Intrathecal infusion of placebo (saline solution).Patients were infused 2 ml of saline solution | Procedure or surgery | Intrathecal infusion of placebo (saline solution). |
Primary outcome
Forced vital capacity
Time frame: Every 3 months
6 secondary outcomes
Neurological variables: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS), Medical Research Council (MRC) and Norris scales
Time frame: Every 3 months
Absence of adverse events
Time frame: Every week / month depending on the study phase
Neurophysiological variables: Electromyography, polysomnography, evoked potentials
Time frame: Every 3 months
Neuroradiological variables: Spinal Magnetic Resonance Imaging (MRI)
Time frame: Every 3 months
Respiratory variables: Maximal inspiratory pressure (PIM), Maximal expiratory pressure (PEM), sniff nasal, oxymetry.
Time frame: Every 3 months
Psychological variables: Health Questionnaire (EuroQol-5D), The Profile of Mood States (POMS)
Time frame: Every 3 months
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 30 Mar 2017.
- Amyotrophic Lateral SclerosisLink provided by the sponsor.
Related trials for Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)
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.
Jose María Moraleda Jiménez, M.D. PhD. study director
Hospital Universitario Virgen de la Arrixaca
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Laminectomy and bone marrow stem cells transplantation and Intrathecal infusion of autologous bone marrow stem cells in people with Amyotrophic Lateral Sclerosis, run by Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia at 1 site in Spain.
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 8 inclusion and 4 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 30 Mar 2017. 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 Spain. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 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?
Forced vital capacity (time frame: Every 3 months). 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 30 Mar 2017.