Amyotrophic Lateral SclerosisNCT03085706
Transplantation of Autologous Peripheral Blood Mononuclear Cells for Amyotrophic Lateral Sclerosis
A study testing PBMC autotransplantation in people with Amyotrophic Lateral Sclerosis, run by The First Affiliated Hospital of Dalian Medical University at 1 site in China.
Trial summary
To assess the safety of peripheral blood mononuclear cell transplantation into the subarachnoid space for the treatment of amyotrophic lateral sclerosis. Read more about this study
- Who is it for?
- Ages 31 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- PBMC autotransplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteChina
- Study dates
- Oct 2010to Jun 2013
- Phase
- Interventional
- 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 31 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- all subjects had a verifiable diagnosis of ALS for 0.5 to 2 years based on a diagnosis using the Revised Criteria of the World Federation of Neurology. The grades of diagnosis were clinically definite ALS or clinically probable ALS;
- ALS was mild-to-moderate based on the ALS Functional Rating Scale-Revised. Electrophysiological features showed compound muscle action potential (CMAP) amplitude of motor nerve normal or mild declining;
- serum creatine kinase was normal or mild upper, less than 500 U/L.
Who cannot join
- use of any other investigational agent within 30 days before treatment;
- severe cardiac, pulmonary, hepatic or/and hematic disease;
- human immunodeficiency virus positivity or signs and symptoms consistent with human immunodeficiency virus infection;
- pregnant or nursing women;
- history of cancer with less than 5 years documentation of a disease-free state;
- history of anaphylactic reaction or hypersensitivity to granulocyte colony- stimulating factor (G-CSF);
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- alcohol or drug abuse in recent 1 year;
- cannot understand or obey the rules of treatment;
- blood donor in recent 30 days.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital of Dalian Medical University
Dalian, Liaoning, 116011
About this study
Official title: Transplantation of Autologous Peripheral Blood Mononuclear Cells in the Subarachnoid Space for Amyotrophic Lateral Sclerosis: a Safety Analysis of 14 Patients
To assess the safety of peripheral blood mononuclear cell transplantation into the subarachnoid space for the treatment of amyotrophic lateral sclerosis.
Read the detailed description
Amyotrophic lateral sclerosis (ALS) is a rapidly evolving, fatal neurodegenerative disease resulting from the degeneration of cortical, bulbar and spinal motor neurons. The disease progresses inexorably to death, usually because by failure of respiratory function, with a median duration of 3 years.
Recent clinical trials using various types of stem cells, including mesenchymal stromal cells, neural stem cells, and peripheral blood mononuclear cells (PBMCs), represent promising strategies for stem cell-based treatment in ALS. It has been demonstrated that the inflammation and neuronal death were reduced in ALS patients after bone marrow transplantation. In addition, the incidence of immune response was decreased by autologous transplantation of bone marrow cells in ALS patients. PBMCs are multi-potent stem cells that are very attractive for a cell therapy approach in ALS because of their plasticity and ability to provide the host tissue with growth factors or modulate the host immune system. PBMCs were used clinically and few adverse effects were attributed to their administration. Early clinical investigations indicated that the transplantation of autologous PBMCs into the dura is feasible in ALS patients; however, one study was limited to three patients and the other recruited eight patients. There are still many questions regarding the intrathecal transplantation of PBMCs for ALS. Therefore, a retrospective study was performed to assess further the safety and efficacy of the procedure and to test the impact of a cell therapy approach in ALS patients.
Statistical analysis Data, expressed as the mean ± SD, were analyzed using SPSS version 17.0 for Windows (SPSS Inc., Chicago, IL, USA). Statistical analyses were performed by paired sample t-test. A value of P < 0.05 was considered statistically significant.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- PBMC autotransplantation
- 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.
PBMC autotransplantationBiological
Fourteen amyotrophic lateral sclerosis (ALS) patients are received peripheral blood mononuclear cell (PBMC) autotransplantation.
| Study group | Role | What they receive |
|---|---|---|
| PBMC autotransplantation | Experimental | Fourteen amyotrophic lateral sclerosis (ALS) patients are received peripheral blood mononuclear cell (PBMC) autotransplantation. Receives: Biological: PBMC autotransplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
adverse events of autologous peripheral blood mononuclear cell mobilization
To assess the safety of autologous peripheral blood mononuclear cell transplantation
Measured: 1 week after operation
Secondary outcomes (3)
Functional independence measurement(FIM)
To assess the self-care ability of daily living
Measured: changes of preoperation and week 1, week 2, week 4, week 12 after operation
Berg Balance Scale
To assess the trunk balance capability and limb movement function
Measured: changes of preoperation and week 1, week 2, week 4, week 12 after operation
Dysarthria Assessment Scale
To assess the progression of bulbar paralysis
Measured: changes of preoperation and week 1, week 2, week 4, week 12 after operation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2017
- Study startOct 2010
- Primary completionDec 2012
- Study completionJun 2013
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.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- 14 patients conducted at the First Affiliated Hospital of Dalian Medical University, China were eligible if they had definite or probable sporadic amyotrophic lateral sclerosis (ALS).
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital of Dalian Medical University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Liu Jing, The First Affiliated Hospital of Dalian Medical University
- Investigators
- Jing Liu, Ph.D., Principal investigator, The First Affiliated Hospital of Dalian Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03085706
- Sponsor's protocol ID
- LCKY2016-58
- Status last verified
- Feb 2018
- First submitted
- 15 Mar 2017
- Last updated
- 9 Feb 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Transplantation of Autologous Peripheral Blood Mononuclear Cells in the Subarachnoid Space for Amyotrophic Lateral Sclerosis: a Safety Analysis of 14 Patients
- ClinicalTrials.gov ID
- NCT03085706
- Acronym / sponsor ID
- LCKY2016-58
- Conditions
- Amyotrophic Lateral Sclerosis
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- The First Affiliated Hospital of Dalian Medical UniversityUniversity, hospital or other
- Study start
- Oct 2010 (actual)
- Primary completion
- Dec 2012 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 21 Mar 2017 (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.
To assess the safety of peripheral blood mononuclear cell transplantation into the subarachnoid space for the treatment of amyotrophic lateral sclerosis.
Detailed description
Amyotrophic lateral sclerosis (ALS) is a rapidly evolving, fatal neurodegenerative disease resulting from the degeneration of cortical, bulbar and spinal motor neurons. The disease progresses inexorably to death, usually because by failure of respiratory function, with a median duration of 3 years.
Recent clinical trials using various types of stem cells, including mesenchymal stromal cells, neural stem cells, and peripheral blood mononuclear cells (PBMCs), represent promising strategies for stem cell-based treatment in ALS. It has been demonstrated that the inflammation and neuronal death were reduced in ALS patients after bone marrow transplantation. In addition, the incidence of immune response was decreased by autologous transplantation of bone marrow cells in ALS patients. PBMCs are multi-potent stem cells that are very attractive for a cell therapy approach in ALS because of their plasticity and ability to provide the host tissue with growth factors or modulate the host immune system. PBMCs were used clinically and few adverse effects were attributed to their administration. Early clinical investigations indicated that the transplantation of autologous PBMCs into the dura is feasible in ALS patients; however, one study was limited to three patients and the other recruited eight patients. There are still many questions regarding the intrathecal transplantation of PBMCs for ALS. Therefore, a retrospective study was performed to assess further the safety and efficacy of the procedure and to test the impact of a cell therapy approach in ALS patients.
Statistical analysis Data, expressed as the mean ± SD, were analyzed using SPSS version 17.0 for Windows (SPSS Inc., Chicago, IL, USA). Statistical analyses were performed by paired sample t-test. A value of P < 0.05 was considered statistically significant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- 14 patients conducted at the First Affiliated Hospital of Dalian Medical University, China were eligible if they had definite or probable sporadic amyotrophic lateral sclerosis (ALS).
| Arm | Type | Receives |
|---|---|---|
| PBMC autotransplantation | Experimental | Biological: PBMC autotransplantationFourteen amyotrophic lateral sclerosis (ALS) patients are received peripheral blood mononuclear cell (PBMC) autotransplantation. |
Who may qualify
- Ages
- 31 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- all subjects had a verifiable diagnosis of ALS for 0.5 to 2 years based on a diagnosis using the Revised Criteria of the World Federation of Neurology. The grades of diagnosis were clinically definite ALS or clinically probable ALS;
- ALS was mild-to-moderate based on the ALS Functional Rating Scale-Revised. Electrophysiological features showed compound muscle action potential (CMAP) amplitude of motor nerve normal or mild declining;
- serum creatine kinase was normal or mild upper, less than 500 U/L.
Key exclusion criteria (9 listed)
- use of any other investigational agent within 30 days before treatment;
- severe cardiac, pulmonary, hepatic or/and hematic disease;
- human immunodeficiency virus positivity or signs and symptoms consistent with human immunodeficiency virus infection;
- pregnant or nursing women;
- history of cancer with less than 5 years documentation of a disease-free state;
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 |
|---|---|---|
| PBMC autotransplantationFourteen amyotrophic lateral sclerosis (ALS) patients are received peripheral blood mononuclear cell (PBMC) autotransplantation. | Biological | PBMC autotransplantation |
Primary outcome
adverse events of autologous peripheral blood mononuclear cell mobilization
Time frame: 1 week after operation
To assess the safety of autologous peripheral blood mononuclear cell transplantation
3 secondary outcomes
Functional independence measurement(FIM)
Time frame: changes of preoperation and week 1, week 2, week 4, week 12 after operation
Berg Balance Scale
Time frame: changes of preoperation and week 1, week 2, week 4, week 12 after operation
Dysarthria Assessment Scale
Time frame: changes of preoperation and week 1, week 2, week 4, week 12 after operation
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 9 Feb 2018.
- Individual participant dataSharing planned: Undecided
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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.
Jing Liu, Ph.D. principal investigator
The First Affiliated Hospital of Dalian Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing PBMC autotransplantation in people with Amyotrophic Lateral Sclerosis, run by The First Affiliated Hospital of Dalian Medical University at 1 site in China.
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 3 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: finished, as of the sponsor's last update on 9 Feb 2018. 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 China. 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.
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?
adverse events of autologous peripheral blood mononuclear cell mobilization (time frame: 1 week after operation). 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 9 Feb 2018.