Motor Neuron DiseasePhase 1NCT02987413
Escalated Application of Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis Patients
A Phase 1 study testing Autologous Mesenchymal stem cells (MSCs) in people with Motor Neuron Disease, run by Hospital e Maternidade Dr. Christóvão da Gama at 2 sites in Brazil.
Trial summary
Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that selectively affects motor neurons in the brain and spinal cord, leading to bulbar, respiratory, and limb weakness. There is no effective treatment, and the disease usually progresses to death within 2 to 4 years. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Motor Neuron Disease
- What is being tested?
- Autologous Mesenchymal stem cells (MSCs)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 2 sitesBrazil
- Study dates
- 28 Apr 2015to 5 Apr 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Women and males over 18-year-old.
- Diagnosis of ALS in agreement with the criteria of "EL SCORE"
- Less than 24 months of evolution of the disease (from the beginning of the symptoms).
- Good understanding of the protocol and aptitude to grant the informed consent
- Infertile women (post-menopause or hysterectomized)
- Brazilian citizen and permanent resident.
Who cannot join
- Any significant medical condition (congestive heart failure, angina, respiratory failure, and others)
- Any auto-immune disease
- Any malignant diseases
- Systemic infection
- Mental illness
- Depressive state
Trial locations and contacts
2 sites in 1 country.
Brazil2 sites
Hospital e Maternidade Dr Christovao da Gama
Santo André, São Paulo, 09030010
Instituto de Ensino e Pesquisas - IEP-São Lucas
São Paulo, São Paulo, 01236-030
About this study
Official title: Safety and Efficacy of Mesenchymal Stem Cells Escalated Application in Amyotrophic Lateral Sclerosis Patients: Study Design of a Phase I Trial
Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that selectively affects motor neurons in the brain and spinal cord, leading to bulbar, respiratory, and limb weakness. There is no effective treatment, and the disease usually progresses to death within 2 to 4 years. The therapeutic plasticity of mesenchymal stem cells (MSCs) may be an attractive therapy to this complex disease, turning MSCs strong candidates for cellular therapy in ALS.
Design-A phase 1 open-safety clinical trial. 4 patients will be selected according to a restricted inclusion and exclusion criteria and after 2 escalated infusions of MSCs, there will be a follow up period of one year Methods - Primary endpoint: safety of mesenchymal autologous stem cells infusions escalated in two intrathecal administrations in patients with ALS defined as severe adverse events (SAe). Secondary endpoints: clinical response, laboratorial and magnetic resonance imaging of patients submitted to cellular escalating doses applied in the study. Quality of life, according to El Escorial criteria, ALSFR scale and functional scales.
Conclusion: This study is a primary step before a large randomized double-blind clinical trial for ALS. It is expected to confirm the safety of escalated MSCs therapy in ALS patients, initial data of efficacy in addition to improved quality of life.
- Conditions
- Motor Neuron Disease
- Being tested
- Autologous Mesenchymal stem cells (MSCs)
- Keywords
- Amyotrophic Lateral Sclerosis
- Medical subject headings
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Mesenchymal stem cells (MSCs)Biological
2 intrathecal autologous MSCs infusions (1x10^8 cells) will be performed, escalated from 30 days apart
| Study group | Role | What they receive |
|---|---|---|
| Autologous Mesenchymal stem cells | Experimental | Two escalated intrathecal infusions of autologous mesenchymal stem cell Receives: Biological: Autologous Mesenchymal stem cells (MSCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serious Adverse Events related to the treatment
Serious Adverse Events (Death, Life-threatening, Disability or Permanent Damage) will be monitored and documented during follow up
Measured: 12 months
Secondary outcomes (1)
Revised ALS Functional Rating Scale (ALSFRS-R)
Quality of life from patients according to functional scales will be evaluated before and after interventions with 1, 3, 6 and 12 months.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2016 (estimated)
- Study start28 Apr 2015
- Primary completionMar 2016
- Study completion5 Apr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Motor Neuron DiseaseStudies 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
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital e Maternidade Dr. Christóvão da Gama
- Sponsor type
- University, hospital or other
- Collaborators
- IEP São Lucas - Instituto de Ensino e Pesquisa, Clinica Jordy Sinapse, TECHLIFE - Centro de Tecnologia Celular
- Responsible party
- Sponsor
- Investigators
- Leandro B Agati, PhD, Study director, Hospital e Maternidade Dr Christovao da GamaEliseo J Sekiya, MD, PhD, Study chair, Instituto de Ensino e Pesquisas - IEP-São LucasAdelson A Silva, MD, Study chair, Instituto de Ensino e Pesquisas - IEP-São LucasAndresa Forte, MSc, Study chair, TECHLIFE - Centro de Tecnologia CelularSergio S Jordy, MD, Study chair, Clínica Jordy Sinapse
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02987413
- Sponsor's protocol ID
- HospitalMCG
- Other IDs
- IEPSaoLucas
- Status last verified
- Aug 2017
- First submitted
- 1 Jul 2016
- Last updated
- 7 Aug 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Mesenchymal Stem Cells Escalated Application in Amyotrophic Lateral Sclerosis Patients: Study Design of a Phase I Trial
- ClinicalTrials.gov ID
- NCT02987413
- Acronym / sponsor ID
- HospitalMCG
- Conditions
- Motor Neuron Disease
- Keywords
- Amyotrophic Lateral Sclerosis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Hospital e Maternidade Dr. Christóvão da GamaUniversity, hospital or other
- Collaborators
- IEP São Lucas - Instituto de Ensino e Pesquisa, Clinica Jordy Sinapse, TECHLIFE - Centro de Tecnologia Celular
- Study start
- 28 Apr 2015 (actual)
- Primary completion
- Mar 2016 (actual)
- Study completion
- 5 Apr 2017 (actual)
- First posted
- 9 Dec 2016 (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.
Background: Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that selectively affects motor neurons in the brain and spinal cord, leading to bulbar, respiratory, and limb weakness. There is no effective treatment, and the disease usually progresses to death within 2 to 4 years. The therapeutic plasticity of mesenchymal stem cells (MSCs) may be an attractive therapy to this complex disease, turning MSCs strong candidates for cellular therapy in ALS.
Design-A phase 1 open-safety clinical trial. 4 patients will be selected according to a restricted inclusion and exclusion criteria and after 2 escalated infusions of MSCs, there will be a follow up period of one year Methods - Primary endpoint: safety of mesenchymal autologous stem cells infusions escalated in two intrathecal administrations in patients with ALS defined as severe adverse events (SAe). Secondary endpoints: clinical response, laboratorial and magnetic resonance imaging of patients submitted to cellular escalating doses applied in the study. Quality of life, according to El Escorial criteria, ALSFR scale and functional scales.
Conclusion: This study is a primary step before a large randomized double-blind clinical trial for ALS. It is expected to confirm the safety of escalated MSCs therapy in ALS patients, initial data of efficacy in addition to improved quality of life.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous Mesenchymal stem cells | Experimental | Biological: Autologous Mesenchymal stem cells (MSCs)Two escalated intrathecal infusions of autologous mesenchymal stem cell |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Women and males over 18-year-old.
- Diagnosis of ALS in agreement with the criteria of "EL SCORE"
- Less than 24 months of evolution of the disease (from the beginning of the symptoms).
- Good understanding of the protocol and aptitude to grant the informed consent
- Infertile women (post-menopause or hysterectomized)
Key exclusion criteria (6 listed)
- Any significant medical condition (congestive heart failure, angina, respiratory failure, and others)
- Any auto-immune disease
- Any malignant diseases
- Systemic infection
- Mental illness
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 |
|---|---|---|
| Autologous Mesenchymal stem cells (MSCs)2 intrathecal autologous MSCs infusions (1x10^8 cells) will be performed, escalated from 30 days apart | Biological | Autologous Mesenchymal stem cells |
Primary outcome
Serious Adverse Events related to the treatment
Time frame: 12 months
Serious Adverse Events (Death, Life-threatening, Disability or Permanent Damage) will be monitored and documented during follow up
1 secondary outcome
Revised ALS Functional Rating Scale (ALSFRS-R)
Time frame: 12 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 7 Aug 2017.
Related trials for Motor Neuron Disease
Active, not recruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
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)
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.
Leandro B Agati, PhD study director
Hospital e Maternidade Dr Christovao da Gama
Eliseo J Sekiya, MD, PhD study chair
Instituto de Ensino e Pesquisas - IEP-São Lucas
Adelson A Silva, MD study chair
Instituto de Ensino e Pesquisas - IEP-São Lucas
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Mesenchymal stem cells (MSCs) in people with Motor Neuron Disease, run by Hospital e Maternidade Dr. Christóvão da Gama at 2 sites in Brazil.
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 6 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 7 Aug 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Brazil. 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 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?
Serious Adverse Events related to the treatment (time frame: 12 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 7 Aug 2017.