ALS (Amyotrophic Lateral Sclerosis)Phase 2TCIM-ELAIINCT04849065
Clinical Trial on the Use of Cell Therapy in the Treatment of Patients With Amyotrophic Lateral Sclerosis
A Phase 2 study testing MNC (Mononuclear cells) in people with ALS (Amyotrophic Lateral Sclerosis), run by Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia at 1 site in Spain.
Trial summary
Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease 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)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 1 siteSpain
- Study dates
- 1 May 2021to 1 Sep 2023
- Phase
- Phase 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
- Diagnosis of ALS defined or probable according to the criteria established by the World Federation of Neurology
- Age between 18 and 70 years.
- Patient who offers sufficient guarantees of adherence to the protocol.
- Neurophysiological data confirming lower motor neuron involvement at the lumbar and cervical level.
- Assessment of the motor deficit in the dorsal flexion of both feet (between 3 and 5 points on the MRC scale).
- Inability to understand informed consent.
Who cannot join
- Mellitus diabetes.
- Other diseases that may be associated with polyneuropathies.
- Previous history of cerebral stroke.
- Previous pathology of the peripheral nervous system that affected one or both lower or upper limbs, with or without clinically evident neurological sequelae.
- Pregnant or actively breastfeeding patients
- Patients physiologically capable of becoming pregnant, unless they are using a reliable contraceptive method (Annex III)
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- Patients with cardiac, renal, hepatic, systemic, immune disease that may influence the survival of the patient during the test.
- Positive serology for hepatitis B, hepatitis C or HIV.
- Clinical and anesthesiological criteria that contraindicate either sedation or the extraction of BM itself (Alteration of the coagulation system or anticoagulated patient with inability to withdraw anticoagulation, hemodynamic instability, skin alteration in the puncture site, etc.)
Trial locations and contacts
1 site in 1 country.
Spain1 site
Hospital Clinico Universitario Virgen de la Arrixaca
Murcia, 30003
Miguel Blanquer Blanquer, 968359767
Miguel Blanquer Blanquer, MD
About this study
Official title: Clinical Trial in Phase II of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease
Read the detailed description
Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease.
This hypothesis is based on experimental work done in animal models of ALS and the results of our previous phase I clinical trial. In this clinical trial (TCIM / ALS, ClinicalTrials.gov Identifier: NCT02286011) we studied with electrophysiological techniques (in a small number of patients with ALS) the effects of intramuscular injection of a single dose of BMSC in the tibialis anterioris (TA) muscle. . The results indicate that this is a safe procedure and show the presence of a positive and apparently transitory effect on the size and number of motor units of the TA muscle.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Being tested
- MNC (Mononuclear cells), Placebo / 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)Drug
- Intramuscular infusion of autologous BM CMN into the TA muscle of one of the lower limbs (experimental side)
- Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)
Placebo / SalineDrug
- Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side)
| Study group | Role | What they receive |
|---|---|---|
| MNC (Mononuclear cells) | Experimental | MNC (Mononuclear cells) (patients in which stem cells will be injected into the two muscles on one side and placebo -vehicle- in the two contralateral muscles).
This group would consist of 74 patients. Receives: Drug: MNC (Mononuclear cells); Drug: Placebo / Saline |
| Saline | Placebo comparator | (patients in which placebo -vehicle- will be injected into both muscles on both sides). This group would consist of 26 patients. Receives: Drug: Placebo / 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: 48 months from baseline
D50 index obtained from stimulus intensity curves
This parameter quantifies the number and size of the functional motor units of a given muscle.
Measured: 48 months from baseline
Secondary outcomes (7)
Motor unit number index" (MUNIX)
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
Measured: 48 months from baseline
Motor unit size index" (MUSIX)
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
Measured: 48 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: 48 months from baseline
PAMC amplitude: mV
Compound muscle action potential (CMAP)
Measured: 48 months from baseline
PAMC area: mV / ms
Compound muscle action potential (CMAP)
Measured: 48 months from baseline
Maximum force developed in an isometric contraction of the muscles
The measurement will be made with a dynamometer during dorsiflexion of the foot (from certain angles) and during abduction of the index finger of the hand, respectively.
Measured: 48 months from baseline
Fuerza muscular (MRC Medical Research Council) score
Muscle strength data will be collected according to the Medical Research Council scale. Muscle strength is graded on a scale of 0 to 5, with 5 being normal muscle strength and 0 being the absence of muscle contraction.
Measured: 48 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 posted19 Apr 2021
- Study start1 May 2021 (estimated)
- Primary completion1 May 2023 (estimated)
- Study completion1 Sep 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Phase II, prospective, multicenter, randomized, parallel, double-blind, placebo-controlled clinical trial
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04849065
- Sponsor's protocol ID
- IMB-TCIM/ELAII-2019-01
- Acronym
- TCIM-ELAII
- Status last verified
- Apr 2021
- First submitted
- 15 Apr 2021
- Last updated
- 19 Apr 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Trial in Phase II of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
- ClinicalTrials.gov ID
- NCT04849065
- Acronym / sponsor ID
- TCIM-ELAII / IMB-TCIM/ELAII-2019-01
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Fundacion para la Formacion e Investigacion Sanitarias de la Region de MurciaUniversity, hospital or other
- Study start
- 1 May 2021 (estimated)
- Primary completion
- 1 May 2023 (estimated)
- Study completion
- 1 Sep 2023 (estimated)
- First posted
- 19 Apr 2021 (actual)
- 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.
Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease
Detailed description
Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease.
This hypothesis is based on experimental work done in animal models of ALS and the results of our previous phase I clinical trial. In this clinical trial (TCIM / ALS, ClinicalTrials.gov Identifier: NCT02286011) we studied with electrophysiological techniques (in a small number of patients with ALS) the effects of intramuscular injection of a single dose of BMSC in the tibialis anterioris (TA) muscle. . The results indicate that this is a safe procedure and show the presence of a positive and apparently transitory effect on the size and number of motor units of the TA muscle.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
- Model description
- Phase II, prospective, multicenter, randomized, parallel, double-blind, placebo-controlled clinical trial
| Arm | Type | Receives |
|---|---|---|
| MNC (Mononuclear cells) | Experimental | Drug: MNC (Mononuclear cells), Drug: Placebo / SalineMNC (Mononuclear cells) (patients in which stem cells will be injected into the two muscles on one side and placebo -vehicle- in the two contralateral muscles). This group would consist of 74 patients. |
| Saline | Placebo comparator | Drug: Placebo / Saline(patients in which placebo -vehicle- will be injected into both muscles on both sides). This group would consist of 26 patients. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosis of ALS defined or probable according to the criteria established by the World Federation of Neurology
- Age between 18 and 70 years.
- Patient who offers sufficient guarantees of adherence to the protocol.
- Neurophysiological data confirming lower motor neuron involvement at the lumbar and cervical level.
- Assessment of the motor deficit in the dorsal flexion of both feet (between 3 and 5 points on the MRC scale).
Key exclusion criteria (9 listed)
- Mellitus diabetes.
- Other diseases that may be associated with polyneuropathies.
- Previous history of cerebral stroke.
- Previous pathology of the peripheral nervous system that affected one or both lower or upper limbs, with or without clinically evident neurological sequelae.
- Pregnant or actively breastfeeding patients
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)* Intramuscular infusion of autologous BM CMN into the TA muscle of one of the lower limbs (experimental side) * Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side) | Drug | MNC (Mononuclear cells) |
| Placebo / Saline- Intramuscular infusion of saline solution (placebo) in the TA muscle of the contralateral lower limb (control side) | Drug | MNC (Mononuclear cells), 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: 48 months from baseline
D50 index obtained from stimulus intensity curves
Time frame: 48 months from baseline
This parameter quantifies the number and size of the functional motor units of a given muscle.
7 secondary outcomes
Motor unit number index" (MUNIX)
Time frame: 48 months from baseline
Motor unit size index" (MUSIX)
Time frame: 48 months from baseline
Fiber density (FD)
Time frame: 48 months from baseline
PAMC amplitude: mV
Time frame: 48 months from baseline
PAMC area: mV / ms
Time frame: 48 months from baseline
Maximum force developed in an isometric contraction of the muscles
Time frame: 48 months from baseline
Fuerza muscular (MRC Medical Research Council) score
Time frame: 48 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 19 Apr 2021.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing MNC (Mononuclear cells) in people with ALS (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 6 inclusion and 9 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 19 Apr 2021. 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Miguel Blanquer Blanquer, MD (968 36 95 00, miguelblanquer@um.es), or a site near the patient. 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: 48 months from baseline). The study lists 2 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 19 Apr 2021.