Amyotrophic Lateral SclerosisPhase 1/2NCT02290886
A Multicenter Phase I/II Clinical Trial to Evaluate Safety of Mesenchymal Stem Cell in Patients With Amyotrophic Sclerosis Lateral
A Phase 1/2 study testing Intravenous administration of 1 million of MSC, Intravenous administration of 2 million of MSC and Intravenous administration of 4 million of MSC in people with Amyotrophic Lateral Sclerosis, run by Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud at 4 sites in Spain.
Trial summary
A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Intravenous administration of 1 million of MSC, Intravenous administration of 2 million of MSC and Intravenous administration of 4 million of MSC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 52 participants
- Where
- 4 sitesSpain
- Study dates
- Jul 2014to 2 Mar 2022
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Women and males over 18-year-old.
- Good understanding of the protocol and aptitude to grant the informed assent.
- Diagnosis of sporadic ALS, with diagnosis of certainty, that is to say, definite or probable, in agreement with the criteria of "El Escorial", of the World Federation of Neurology.
- Forced vital capacity of at least 50 % of the one that would correspond to them for sex, height and age.
- More than 6 and less than 36 months of evolution of the disease (from the beginning of the symptoms).
- Possibility of obtaining, at least, 50gr of adipose tissue.
- Treatment with riluzole, for at least, a month before the inclusion.
Who cannot join
- Any concomitant disease that under investigator's criteria could concern the measures of the clinical variables of the trial (hepatic, renal or cardiac insufficiency, diabetes mellitus, etc).
- Previous therapy with stem cells.
- Participation in another clinical trial during 3 months previous to the entry in this trial.
- Any disease lymphoproliferative
- Tracheostomy and /or gastrostomy.
- Haemophilia, diathesis hemorrhagic or anticoagulative current therapy.
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- Hypersensitivity known to the bovine foetal whey or the gentamicin.
- Medical precedents of infection of the HIV or any serious condition of immunocompromised.
- Positive HBV or HCV serology
- Levels of creatinine in whey > 3.0 in subjects not submitted to haemodialysis.
Trial locations and contacts
4 sites in 1 country.
Spain4 sites
Hospital Regional Universitario Reina Sofía
Córdoba, 14004
Hospital Regional Universitario de Málaga
Málaga, 29010
Hospital Universitario Virgen Macarena, Servicio de Neurología
Seville, 41009
Hospital Universitario Virgen del Rocío
Seville, 41013
About this study
Official title: A Multicenter Phase I/II Clinical Trial, Randomized, Controlled With Placebo, Triple Blind to Evaluate Safety, and Indications of Efficiency of the Intravenous Administration of the Therapy With 3 Doses of MSC in Patients With ASL Moderated to Severe
A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).
Read the detailed description
A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).
40 patients will be enrolled and will be randomized into one of the following 4 arms:
- 10 patients in the control group (placebo)
- 10 patients received a dose of 1 million MSC / kg
- 10 patients received a dose of 2 million MSC / kg.
- 10 patients received a dose of 4 million MSC / kg
The follow-up phase of each patient from the cell infusion/placebo will be 6 months.
At the time that each patient completed the follow-up period (i.e., 6 months after the infusion of the cellular product or placebo), the blind will be open, and patients who have been assigned to the control group, will receive the cell product as secondary treatment. These patients will be randomized to receive each of the doses used in the first phase. From this point, they begin a second period of follow up of 6 months.
In addition, after 6 months of MSC infusion, every patient will continue in an open extension study for 36 months to assess the safety of MSC.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Intravenous administration of placebo, Intravenous administration of 1 million of MSC, Intravenous administration of 2 million of MSC, Intravenous administration of 4 million of MSC
- Keywords
- safety evaluation, intravenous administration, 3 doses of autologous MSC, 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.
Intravenous administration of placeboOther
Intravenous administration of 1 million of MSCDrug
Intravenous administration of 2 million of MSCDrug
Intravenous administration of 4 million of MSCDrug
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Intravenous administration of placebo Receives: Other: Intravenous administration of placebo |
| 1 million of MSC | Experimental | Intravenous administration of 1 million of MSC/ kg Receives: Drug: Intravenous administration of 1 million of MSC |
| 2 million of MSC | Experimental | Intravenous administration of 2 million of MSC/ kg Receives: Drug: Intravenous administration of 2 million of MSC |
| 4 million of MSC | Experimental | Intravenous administration of 4 million of MSC/ kg Receives: Drug: Intravenous administration of 4 million of MSC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)
To evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells (MSC) from adipose tissue in patients with Amyotrophic lateral Sclerosis (ALS) ABSENCE of: complications in the place of the infusion, appearance of a new neurological effect not attributable to the natural progression of this pathology and adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)
Measured: 6 months
Complications in the place of the infusion
To evaluate the safety of the intravenous administration of 3 doses of autologous
Measured: 6 months
Appearance of a new neurological effect not attributable to the natural progression of this pathology
To evaluate the safety of the intravenous administration of 3 doses of autologous
Measured: 6 months
Secondary outcomes (6)
Changes in the progression of the disease (modifications in the scale of functionality of the ALS)
Measured: 6 months
Changes in the degree of muscular force
Measured: 6 months
Changes in the vital forced capacity
Measured: 6 months
Changes of the muscular mass estimated by Nuclear Magnetic Resonance (NMR) of the upper and low extremities
Measured: 6 months
Changes in neurophysiological parameters and of quality of life
Measured: 6 months
Need and time to tracheotomy or permanent assisted ventilation
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2014 (estimated)
- Study startJul 2014
- Primary completion2 Mar 2022
- Study completion2 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Single group
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud
- Sponsor type
- University, hospital or other
- Collaborators
- Iniciativa Andaluza en Terapias Avanzadas
- Responsible party
- Sponsor
- Investigators
- Óscar Fernández, MD, Study chair, Hospital Regional U. de Málaga
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02290886
- Sponsor's protocol ID
- CeTMAd/ELA/2011
- Status last verified
- Apr 2022
- First submitted
- 9 Oct 2014
- Last updated
- 6 Apr 2022
- Results posted
- No
- Data monitoring committee
- No
Publications
Ilieva H, Maragakis NJ. Motoneuron Disease: Clinical. Adv Neurobiol. 2017;15:191-210. doi: 10.1007/978-3-319-57193-5_7.
PubMed 28674982
Study at a glance
From the registry record, last updated by the sponsor on 6 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Phase I/II Clinical Trial, Randomized, Controlled With Placebo, Triple Blind to Evaluate Safety, and Indications of Efficiency of the Intravenous Administration of the Therapy With 3 Doses of MSC in Patients With ASL Moderated to Severe
- ClinicalTrials.gov ID
- NCT02290886
- Acronym / sponsor ID
- CeTMAd/ELA/2011
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- safety evaluation, intravenous administration, 3 doses of autologous MSC, ALS
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y SaludUniversity, hospital or other
- Collaborators
- Iniciativa Andaluza en Terapias Avanzadas
- Study start
- Jul 2014 (actual)
- Primary completion
- 2 Mar 2022 (actual)
- Study completion
- 2 Mar 2022 (actual)
- First posted
- 14 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.
A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).
Detailed description
A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).
40 patients will be enrolled and will be randomized into one of the following 4 arms:
- 10 patients in the control group (placebo)
- 10 patients received a dose of 1 million MSC / kg
- 10 patients received a dose of 2 million MSC / kg.
- 10 patients received a dose of 4 million MSC / kg
The follow-up phase of each patient from the cell infusion/placebo will be 6 months.
At the time that each patient completed the follow-up period (i.e., 6 months after the infusion of the cellular product or placebo), the blind will be open, and patients who have been assigned to the control group, will receive the cell product as secondary treatment. These patients will be randomized to receive each of the doses used in the first phase. From this point, they begin a second period of follow up of 6 months.
In addition, after 6 months of MSC infusion, every patient will continue in an open extension study for 36 months to assess the safety of MSC.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Other: Intravenous administration of placeboIntravenous administration of placebo |
| 1 million of MSC | Experimental | Drug: Intravenous administration of 1 million of MSCIntravenous administration of 1 million of MSC/ kg |
| 2 million of MSC | Experimental | Drug: Intravenous administration of 2 million of MSCIntravenous administration of 2 million of MSC/ kg |
| 4 million of MSC | Experimental | Drug: Intravenous administration of 4 million of MSCIntravenous administration of 4 million of MSC/ kg |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Women and males over 18-year-old.
- Good understanding of the protocol and aptitude to grant the informed assent.
- Diagnosis of sporadic ALS, with diagnosis of certainty, that is to say, definite or probable, in agreement with the criteria of "El Escorial", of the World Federation of Neurology.
- Forced vital capacity of at least 50 % of the one that would correspond to them for sex, height and age.
- More than 6 and less than 36 months of evolution of the disease (from the beginning of the symptoms).
Key exclusion criteria (10 listed)
- Any concomitant disease that under investigator's criteria could concern the measures of the clinical variables of the trial (hepatic, renal or cardiac insufficiency, diabetes mellitus, etc).
- Previous therapy with stem cells.
- Participation in another clinical trial during 3 months previous to the entry in this trial.
- Any disease lymphoproliferative
- Tracheostomy and /or gastrostomy.
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 |
|---|---|---|
| Intravenous administration of placebo | Other | Placebo |
| Intravenous administration of 1 million of MSC | Drug | 1 million of MSC |
| Intravenous administration of 2 million of MSC | Drug | 2 million of MSC |
| Intravenous administration of 4 million of MSC | Drug | 4 million of MSC |
Primary outcome
Number of adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)
Time frame: 6 months
To evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells (MSC) from adipose tissue in patients with Amyotrophic lateral Sclerosis (ALS) ABSENCE of: complications in the place of the infusion, appearance of a new neurological effect not attributable to the natural progression of this pathology and adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)
Complications in the place of the infusion
Time frame: 6 months
To evaluate the safety of the intravenous administration of 3 doses of autologous
Appearance of a new neurological effect not attributable to the natural progression of this pathology
Time frame: 6 months
To evaluate the safety of the intravenous administration of 3 doses of autologous
6 secondary outcomes
Changes in the progression of the disease (modifications in the scale of functionality of the ALS)
Time frame: 6 months
Changes in the degree of muscular force
Time frame: 6 months
Changes in the vital forced capacity
Time frame: 6 months
Changes of the muscular mass estimated by Nuclear Magnetic Resonance (NMR) of the upper and low extremities
Time frame: 6 months
Changes in neurophysiological parameters and of quality of life
Time frame: 6 months
Need and time to tracheotomy or permanent assisted ventilation
Time frame: 6 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 6 Apr 2022.
- Derived from this study: PubMed 28674982Ilieva H, Maragakis NJ. Motoneuron Disease: Clinical. Adv Neurobiol. 2017;15:191-210. doi: 10.1007/978-3-319-57193-5_7.
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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.
Óscar Fernández, MD study chair
Hospital Regional U. de Málaga
4 sites 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 Intravenous administration of 1 million of MSC, Intravenous administration of 2 million of MSC and Intravenous administration of 4 million of MSC in people with Amyotrophic Lateral Sclerosis, run by Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud at 4 sites 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 7 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: finished, as of the sponsor's last update on 6 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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?
Number of adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE) (time frame: 6 months). The study lists 3 primary outcomes in all. 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 NCT02290886, which also lists 1 linked publication.
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 6 Apr 2022.