ALS (Amyotrophic Lateral Sclerosis)Phase 1TCIM/ELANCT02286011
Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis
A Phase 1 study testing MNC (Mononuclear cells) and Saline in people with ALS (Amyotrophic Lateral Sclerosis), run by Red de Terapia Celular at 1 site in Spain.
Trial summary
The purpose of this study is to evaluate the safety of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis by a prospective, single-center, randomized, parallel, double-blind, placebo-controlled phase I clinical trial. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- ALS (Amyotrophic Lateral Sclerosis)
- What is being tested?
- MNC (Mononuclear cells) and Saline
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteSpain
- Study dates
- Nov 2014to Dec 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
- Diagnosis of definite or probable ALS according to the criteria established by the World Federation of Neurology
- Patient that provides reasonable assurance of adherence to protocol.
- Neurophysiological data confirming affectation of lower motor neurons in the lumbar region.
- Assessment of motor deficits in dorsiflexion of both feet (4 or 5 points on the MRC scale)
- The patient must fulfill all inclusion criteria.
Who cannot join
- Diabetes Mellitus.
- Other diseases that may present with polyneuropathy.
- Previous history of stroke.
- Prior Pathology of the peripheral nervous system affecting one or both lower limbs with or without clinically evident neurological sequelae.
- Pregnant or breastfeeding patients active.
- Patients physiologically capable of becoming pregnant, unless they are using reliable contraception.
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- Patients with cardiac disease, renal, hepatic, systemic, immune that may influence patient survival during the test.
- Positive serology for hepatitis B, hepatitis C or HIV.
- Clinical and anesthesiologic Criteria, contraindicating either sedation or extraction of MO (Altered coagulation system or anticoagulated patient with inability to withdraw anticoagulation, hemodynamic instability, altered skin puncture site, etc.)
- Included in other clinical trials in the last 6 months.
Trial locations and contacts
1 site in 1 country.
Spain1 site
Clinical Universitary Hospital Virgen de la Arrixaca
El Palmar, Murcia, 30120
About this study
Official title: Phase I Clinical Trial on Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
The purpose of this study is to evaluate the safety of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis by a prospective, single-center, randomized, parallel, double-blind, placebo-controlled phase I clinical trial.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Being tested
- MNC (Mononuclear cells), Saline
- 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.
MNC (Mononuclear cells)Biological
The cells are infused into the TA muscle of the lower limb randomized as Group A (experimental) intramuscularly. The infusion is made with a needle, 26 gauge, at 4 points of the TA muscle a specific depth given by the neurophysiological study. The total volume infused will be 2 ml, 0.5 ml at each point.
For infusion is as painless as possible for the patient to comply exactly with stereotactic indications of neurophysiology, the infusion was made at a uniform controlled rate and for this, the syringe is placed on a Yesargil arm, equipped with a microinjector and controlled infusion device.
SalineOther
The placebo, 2ml of saline, will be infused like in the experimental arm.
| Study group | Role | What they receive |
|---|---|---|
| MNC (Mononuclear cells) | Experimental | All patients included in the clinical trial will receive an intramuscular infusion of autologous mononuclear cells (MNC) of Bone Marrow (BM) in TA muscle of one of the lower limb (experimental group). The lower limb on the CMN infuse autologous BM will be determined randomly.
The average dose is 550 millions of cells (100-1200 million) diluted in 2 ml. saline Receives: Biological: MNC (Mononuclear cells) |
| Saline | Placebo comparator | All patients included in the clinical trial will receive an intramuscular infusion of 2 mL of saline (placebo) in the TA muscle of the contralateral limb (group control). Receives: Other: Saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis.
Measured: 24 months from baseline
Secondary outcomes (6)
Estimated number of motor units (MUNE)
Several techniques for estimating the MUNE, all based on the relationship between the amplitude or area of compound muscle action potential (CMAP) and amplitude or area corresponding to a single motor unit response. The differences between the techniques are due to the different ways of estimating the amplitude of the responses for individual motor units. The study will use two techniques:
Incremental Technique: The unitary amplitude (or area) of individual motor units are calculated from the responses to increasing intensities stimuli near of intensity threshold (Dantes and McComas, 1991) Statistical technique: The unit amplitude (or area) of the individual motor units are calculated from the amplitude variations (or area) of the muscle action potential obtained in response to stimuli from a fixed intensity (Daube, 2006)
Measured: 24 months from baseline
Compound muscle action potential (CMAP)
CMAP is registered after supramaximal stimulation intensity (0.1-0.2 ms pulses at 1 Hz) of the common peroneal nerve at the level of the head of the fibula. The electrical stimulus is placed in a fixed position during the entire registration process. CMAP will be recorded simultaneously in 5 positions along longitudinally oriented TA muscle. To determine these 5 positions bony landmarks that are reproducible between members and between different patients will be used.
Measured: 24 months from baseline
Fiber density (FD)
Quantifies the average number of muscle fibers per motor unit. It is obtained from single fiber recordings made with electrodes. The average number of motor unit potentials is calculated in 20 different positions in the muscle.
Measured: 24 months from baseline
Muscle force MRC (Medical Research Council) score
Measured: 24 months from baseline
Maximum force developed in an isometric contraction of the tibialis anterior (TA) muscle.
The measurement will be done in Newtons, with a dynamometer during dorsiflexion of the foot (from certain angles).
Measured: 24 months from baseline
Maximum transversal area of the tibialis anterior (TA)
The area will be measured in cm2 by echography.
Measured: 24 months from baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Nov 2014 (estimated)
- Study startNov 2014
- Primary completionDec 2017 (estimated)
- Study completionDec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for ALS (Amyotrophic Lateral Sclerosis)Studies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Red de Terapia Celular
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Spanish National Health System, Hospital Universitario Virgen de la Arrixaca, Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia, Public Health Service, Murcia, Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca
- Responsible party
- Sponsor
- Investigators
- Joaquín A Gómez Espuch, MD, Principal investigator, Hospital Universitario Virgen de la Arrixaca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02286011
- Sponsor's protocol ID
- TCIM/ELA
- Other IDs
- 2011-004801-25
- Acronym
- TCIM/ELA
- Status last verified
- Mar 2017
- First submitted
- 24 Oct 2014
- Last updated
- 30 Mar 2017
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Blanquer M, Moraleda JM, Iniesta F, Gomez-Espuch J, Meca-Lallana J, Villaverde R, Perez-Espejo MA, Ruiz-Lopez FJ, Garcia Santos JM, Bleda P, Izura V, Saez M, De Mingo P, Vivancos L, Carles R, Jimenez J, Hernandez J, Guardiola J, Del Rio ST, Antunez C, De la Rosa P, Majado MJ, Sanchez-Salinas A, Lopez J, Martinez-Lage JF, Martinez S. Neurotrophic bone marrow cellular nests prevent spinal motoneuron degeneration in amyotrophic lateral sclerosis patients: a pilot safety study. Stem Cells. 2012 Jun;30(6):1277-85. doi: 10.1002/stem.1080.
PubMed 22415951Geijo-Barrientos E, Pastore-Olmedo C, De Mingo P, Blanquer M, Gomez Espuch J, Iniesta F, Iniesta NG, Garcia-Hernandez A, Martin-Estefania C, Barrios L, Moraleda JM, Martinez S. Intramuscular Injection of Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis Patients: A Randomized Clinical Trial. Front Neurosci. 2020 Mar 24;14:195. doi: 10.3389/fnins.2020.00195. eCollection 2020.
PubMed 32265627
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 Clinical Trial on Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
- ClinicalTrials.gov ID
- NCT02286011
- Acronym / sponsor ID
- TCIM/ELA / TCIM/ELA
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Red de Terapia CelularIndustry (pharma, biotech, devices)
- Collaborators
- Spanish National Health System, Hospital Universitario Virgen de la Arrixaca, Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia, Public Health Service, Murcia, Instituto Murciano de Investigación Biosanitaria Virgen de la Arrixaca
- Study start
- Nov 2014
- Primary completion
- Dec 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 7 Nov 2014 (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 study is to evaluate the safety of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis by a prospective, single-center, randomized, parallel, double-blind, placebo-controlled phase I clinical trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MNC (Mononuclear cells) | Experimental | Biological: MNC (Mononuclear cells)All patients included in the clinical trial will receive an intramuscular infusion of autologous mononuclear cells (MNC) of Bone Marrow (BM) in TA muscle of one of the lower limb (experimental group). The lower limb on the CMN infuse autologous BM will be determined randomly. The average dose is 550 millions of cells (100-1200 million) diluted in 2 ml. saline |
| Saline | Placebo comparator | Other: SalineAll patients included in the clinical trial will receive an intramuscular infusion of 2 mL of saline (placebo) in the TA muscle of the contralateral limb (group control). |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Diagnosis of definite or probable ALS according to the criteria established by the World Federation of Neurology
- Patient that provides reasonable assurance of adherence to protocol.
- Neurophysiological data confirming affectation of lower motor neurons in the lumbar region.
- Assessment of motor deficits in dorsiflexion of both feet (4 or 5 points on the MRC scale)
- The patient must fulfill all inclusion criteria.
Key exclusion criteria (10 listed)
- Diabetes Mellitus.
- Other diseases that may present with polyneuropathy.
- Previous history of stroke.
- Prior Pathology of the peripheral nervous system affecting one or both lower limbs with or without clinically evident neurological sequelae.
- Pregnant or breastfeeding patients active.
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 |
|---|---|---|
| MNC (Mononuclear cells)The cells are infused into the TA muscle of the lower limb randomized as Group A (experimental) intramuscularly. The infusion is made with a needle, 26 gauge, at 4 points of the TA muscle a specific depth given by the neurophysiological study. The total volume infused will be 2 ml, 0.5 ml at each point. For infusion is as painless as possible for the patient to comply exactly with stereotactic indications of neurophysiology, the infusion was made at a uniform controlled rate and for this, the syringe is placed on a Yesargil arm, equipped with a microinjector and controlled infusion device. | Biological | MNC (Mononuclear cells) |
| SalineThe placebo, 2ml of saline, will be infused like in the experimental arm. | Other | Saline |
Primary outcome
Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis.
Time frame: 24 months from baseline
6 secondary outcomes
Estimated number of motor units (MUNE)
Time frame: 24 months from baseline
Compound muscle action potential (CMAP)
Time frame: 24 months from baseline
Fiber density (FD)
Time frame: 24 months from baseline
Muscle force MRC (Medical Research Council) score
Time frame: 24 months from baseline
Maximum force developed in an isometric contraction of the tibialis anterior (TA) muscle.
Time frame: 24 months from baseline
Maximum transversal area of the tibialis anterior (TA)
Time frame: 24 months from baseline
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2017.
- Results publication: PubMed 22415951Blanquer M, Moraleda JM, Iniesta F, Gomez-Espuch J, Meca-Lallana J, Villaverde R, Perez-Espejo MA, Ruiz-Lopez FJ, Garcia Santos JM, Bleda P, Izura V, Saez M, De Mingo P, Vivancos L, Carles R, Jimenez J, Hernandez J, Guardiola J, Del Rio ST, Antunez C, De la Rosa P, Majado MJ, Sanchez-Salinas A, Lopez J, Martinez-Lage JF, Martinez S. Neurotrophic bone marrow cellular nests prevent spinal motoneuron degeneration in amyotrophic lateral sclerosis patients: a pilot safety study. Stem Cells. 2012 Jun;30(6):1277-85. doi: 10.1002/stem.1080.
- Derived from this study: PubMed 32265627Geijo-Barrientos E, Pastore-Olmedo C, De Mingo P, Blanquer M, Gomez Espuch J, Iniesta F, Iniesta NG, Garcia-Hernandez A, Martin-Estefania C, Barrios L, Moraleda JM, Martinez S. Intramuscular Injection of Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis Patients: A Randomized Clinical Trial. Front Neurosci. 2020 Mar 24;14:195. doi: 10.3389/fnins.2020.00195. eCollection 2020.
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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.
Joaquín A Gómez Espuch, MD principal investigator
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 study testing MNC (Mononuclear cells) and Saline in people with ALS (Amyotrophic Lateral Sclerosis), run by Red de Terapia Celular 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 5 inclusion and 10 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: status not confirmed, 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 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?
Results are not available in the source record.
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?
Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis. (time frame: 24 months from baseline). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02286011, which also lists 2 linked publications.
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.