AMYOTROPHIC LATERAL SCLEROSISPhase 1/2NCT02917681
Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis
A Phase 1/2 study testing Two intrathecal MSC injections in people with AMYOTROPHIC LATERAL SCLEROSIS, run by University of Sao Paulo General Hospital at 1 site in Brazil.
Trial summary
The study aims to evaluate primarily safety of two injections of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis patients. Secondary outcomes of efficacy will also be evaluated 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?
- Two intrathecal MSC injections
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 1 siteBrazil
- Study dates
- Sep 2016to Aug 2019
- 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
- Age between 18 and 70
- Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000)
- ALSFRS-r ≥30 at enrollment
- Forced Vital Capacity ≥65% of the height and weight standard
- No-pregnancy agreement
- Regional accessibility to the study site
- Capability to give away agreed consensus
- Patients will be followed at Academic Institutions at their hometown
Who cannot join
- Previous cellular therapy
- Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections
- Personal history of auto-Immune, myeloproliferative or myelodysplastic diseases, leukemia, lymphoma, whole-body irradiation, hip fracture, severe scoliosis or incapacity to undergo any of the study's proposed procedures
- Any other disease that may interfere with the study
- Any other neurological diseases
- Aspartate or alanine aminotransferases elevated >3x normality upper limit
Show all 16Show fewer
- Serum creatinine >2x normality upper limit
- Hepatitis B and C, HIV, HTLV I and II and syphilis
- Immunosuppressant drug use within 6 weeks from the study's screening
- Pregnancy or breast-feeding
- Acquired or inherited Immunodeficiency
- Participation in other clinical trials
- Non-invasive ventilation, tracheostomy or diaphragm pacing
- Substance abuse within one year and other unstable mental health diseases according to researcher's judgement
- Gastrostomy or any alternative feeding means
- Inappropriate in-vitro MSC expansion
Trial locations and contacts
1 site in 1 country.
Brazil1 site, 1 recruiting
- Recruiting
University of Sao Paulo School of Medicine Clinics Hospital
São Paulo, São Paulo, 05403000
About this study
The study aims to evaluate primarily safety of two injections of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis patients. Secondary outcomes of efficacy will also be evaluated
Read the detailed description
The study is an open-label, unmasked, uncontrolled phase I and II trial to evaluate safety and efficacy of two injections of autologous mesenchymal stem cells (MSC) in Amyotrophic Lateral Sclerosis (ALS) patients. Patients will be recruited trough a web-based registration system, open for all ALS Brazilian patients. The patients will be enrolled after in-person screening at the University of Sao Paulo Medical School Clinics Hospital and inclusion criteria fulfilled. There will be 9 national calls for registration, two months apart each. The enrolled patients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions.
- Conditions
- AMYOTROPHIC LATERAL SCLEROSIS
- Being tested
- Two intrathecal MSC injections
- Keywords
- ALS, mesenchymal stem cells, intrathecal
- 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.
Two intrathecal MSC injectionsOther
| Study group | Role | What they receive |
|---|---|---|
| MSC injection | Experimental | Patients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions. Receives: Other: Two intrathecal MSC injections |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery
Measured: 10 months
Secondary outcomes (6)
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
Specific scale for assessing ALS disease progression
Measured: 10 months
Change in Handheld dynamometry
Measured: 10 months
Change in Electrical Impedance Myography
Measured: 10 months
Change in Forced Vital Capacity (FVC)
Measured: 10 months
Change in Isometric Strength
Measured: 10 months
Changes in morphometric parameters of MRI of encephalon and spinal cord
Measured: 10 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2016 (estimated)
- Study startSep 2016
- Primary completionFeb 2019 (estimated)
- Study completionAug 2019 (estimated)
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.
- More trials run by University of Sao Paulo General HospitalThe sponsor's other registered studies.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Sao Paulo General Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Pontifícia Universidade Católica do Paraná
- Responsible party
- Principal investigator, Gerson Chadi, University of Sao Paulo General Hospital
- Investigators
- Gerson Chadi, MD, PhD, Principal investigator, Full Professor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02917681
- Sponsor's protocol ID
- 401922/2014-6
- Status last verified
- Sep 2016
- First submitted
- 26 Sep 2016
- Last updated
- 28 Sep 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT02917681
- Acronym / sponsor ID
- 401922/2014-6
- Conditions
- AMYOTROPHIC LATERAL SCLEROSIS
- Keywords
- ALS, mesenchymal stem cells, intrathecal
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- University of Sao Paulo General HospitalUniversity, hospital or other
- Collaborators
- Pontifícia Universidade Católica do Paraná
- Study start
- Sep 2016
- Primary completion
- Feb 2019 (estimated)
- Study completion
- Aug 2019 (estimated)
- First posted
- 28 Sep 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.
The study aims to evaluate primarily safety of two injections of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis patients. Secondary outcomes of efficacy will also be evaluated
Detailed description
The study is an open-label, unmasked, uncontrolled phase I and II trial to evaluate safety and efficacy of two injections of autologous mesenchymal stem cells (MSC) in Amyotrophic Lateral Sclerosis (ALS) patients. Patients will be recruited trough a web-based registration system, open for all ALS Brazilian patients. The patients will be enrolled after in-person screening at the University of Sao Paulo Medical School Clinics Hospital and inclusion criteria fulfilled. There will be 9 national calls for registration, two months apart each. The enrolled patients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MSC injection | Experimental | Other: Two intrathecal MSC injectionsPatients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Age between 18 and 70
- Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000)
- ALSFRS-r ≥30 at enrollment
- Forced Vital Capacity ≥65% of the height and weight standard
- No-pregnancy agreement
Key exclusion criteria (16 listed)
- Previous cellular therapy
- Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections
- Personal history of auto-Immune, myeloproliferative or myelodysplastic diseases, leukemia, lymphoma, whole-body irradiation, hip fracture, severe scoliosis or incapacity to undergo any of the study's proposed procedures
- Any other disease that may interfere with the study
- Any other neurological diseases
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 |
|---|---|---|
| Two intrathecal MSC injections | Other | MSC injection |
Primary outcome
Safety
Time frame: 10 months
Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery
6 secondary outcomes
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)
Time frame: 10 months
Change in Handheld dynamometry
Time frame: 10 months
Change in Electrical Impedance Myography
Time frame: 10 months
Change in Forced Vital Capacity (FVC)
Time frame: 10 months
Change in Isometric Strength
Time frame: 10 months
Changes in morphometric parameters of MRI of encephalon and spinal cord
Time frame: 10 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2016.
- 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.
Gerson Chadi, MD, PhD principal investigator
Full Professor
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 Two intrathecal MSC injections in people with AMYOTROPHIC LATERAL SCLEROSIS, run by University of Sao Paulo General Hospital at 1 site 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 8 inclusion and 16 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 28 Sep 2016. 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 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 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fabrício Castro Borba, MD (551130617460, contato@projetoelabrasil.com.br), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (time frame: 10 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 28 Sep 2016.