Parkinson's DiseasePhase 1/2NCT01446614
Mesenchymal Stem Cells Transplantation to Patients With Parkinson's Disease
A Phase 1/2 study testing bone marrow derived mesenchymal stem cells in people with Parkinson's Disease, run by Guangzhou General Hospital of Guangzhou Military Command at 1 site in China.
Trial summary
The study is a phase I/II trial designed to establish the safety and efficacy of intravenous administration of autologous bone marrow derived mesenchymal stem cells to patients with Parkinson's disease. Read more about this study
- Who is it for?
- Ages 30 to 65 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- bone marrow derived mesenchymal stem cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- Oct 2011to Jun 2014
- 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 30 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient with current diagnosis of idiopathic Parkinson's disease.
- Age 30 to 65.
- Experiencing motor complications despite optimized levodopa treatment.
- PD of Stage 2,2.5,3 or 4 of Hoehn-Yahr staging.
- Time between diagnosis and enrollment greater than 2 years.
- No significant cognitive impairment. MMSE > 24.
- Ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- Patients may not be receiving any other investigational agents within 4 weeks of study entry.
- History of allergic reactions attributed to compounds of similar biologic composition to mesenchymal stem cells.
- Primary hematologic diseases.
- Patients undergo intracranial surgeries or implantation of a device for Parkinson's disease.
- Psychiatric, addictive or any other disorder that compromises ability to give a truly informed consent and perform all study assessments.
- Atypical or secondary parkinsonism.
Show all 10Show fewer
- Malignancy within the last 5 years.
- Any other serious medical illness that might preclude safe participation in the study.
- Pregnant or breastfeeding women.
- HIV-positive patients.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Guangzhou General Hospital of Guangzhou Military Command
Guangzhou, Guangdong, 510010
Yang Xiao, MD, 86-20-36653562, jdxiao111@163.com
Li Li, MD, 86-20-36654678, Lily17155@yahoo.com
About this study
Official title: PhaseⅠ/ⅡTrial of Autologous Bone Marrow Derived Mesenchymal Stem Cells to Patients With Parkinson's Disease.
The study is a phase I/II trial designed to establish the safety and efficacy of intravenous administration of autologous bone marrow derived mesenchymal stem cells to patients with Parkinson's disease.
Read the detailed description
Parkinson's disease (PD) is a common progressive neurodegenerative disorder caused by the loss of dopaminergic neurons in the substantia nigra. A combination of genetic and environmental factors is likely to be important in producing abnormal protein aggregation within select groups of neurones, leading to cell dysfunction and then death. A large number of agents together with surgical interventions are now available to treat early and late complications of PD, but they are suffer from two main drawbacks: side effects and loss of efficacy with disease progression.
Bone marrow (BM) derived mesenchymal stem cells (MSCs) an differentiate under certain circumstances into cells from various neuronal and glial type lineages; they also exert immunomodulatory effects. PD-derived MSCs are similar to normal MSCs in phenotype, morphology, and multidifferentiation capacity. Moreover, PD-derived MSCs are capable of differentiating into neurons in a specific medium with up to 30% having the characteristics of dopamine cells. These findings indicate that MSCs derived from PD patients' bone marrow may be a promising cell type for cellular therapy.
BM-MSCs cultured with a cocktail of growth factors (containing FGF and BDNF) differentiate into neuronal/glial lineage cells with a predominance of cells expressing astrocytes' markers. They were effective in suppression of chronic EAE in mice and induced neuroprotection, preserving most of the axons in the CNS of successfully-treated animals. Histopathological studies revealed that MSCs could efficiently migrate into the CNS inflamed tissue (both when administered intravenously and intraventricularly) and differentiated into cells expressing neural-glial lineage markers. Such an approach may provide a feasible and practical way for PD.
- Conditions
- Parkinson's Disease
- Being tested
- bone marrow derived mesenchymal stem cells
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
bone marrow derived mesenchymal stem cellsBiological
Also called Mesenchymal Stem Cells, Multipotent Mesenchymal Stem Cells, Multipotent Mesenchymal Stromal Cells
Intravenous administration of up to 6x10^5 MSCs per kg,qw,for 4 weeks
| Study group | Role | What they receive |
|---|---|---|
| MSC | Experimental | Intravenous autologous bone marrow derived mesenchymal stem cells infusion to patients with Parkinson's disease. Receives: Biological: bone marrow derived mesenchymal stem cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with adverse events
Measured: 1 month after transplantation
Secondary outcomes (4)
Effect assessment
Assessed by Unified Parkinson's Disease Rating Scale (UPDRS).
Measured: 1 month after transplantation
Effect assessment
Assessed by UPDRS
Measured: 3 months after transplantation
Effect assessment
Assessed by UPDRS
Measured: 6 months after transplantation
Effect assessment
Assessed by UPDRS
Measured: 12 months after transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2011 (estimated)
- Study startOct 2011
- Primary completionJun 2013 (estimated)
- Study completionJun 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Parkinson's 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guangzhou General Hospital of Guangzhou Military Command
- Sponsor type
- University, hospital or other
- Investigators
- Yang Xiao, MD, Study director, Guangzhou General Hospital of Guangzhou Military Command
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01446614
- Sponsor's protocol ID
- HM-2011-10
- Status last verified
- Oct 2011
- First submitted
- 4 Oct 2011
- Last updated
- 5 Oct 2011
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Wang Y, Chen S, Yang D, Le WD. Stem cell transplantation: a promising therapy for Parkinson's disease. J Neuroimmune Pharmacol. 2007 Sep;2(3):243-50. doi: 10.1007/s11481-007-9074-2. Epub 2007 May 9.
PubMed 18040857Karussis D, Kassis I, Kurkalli BG, Slavin S. Immunomodulation and neuroprotection with mesenchymal bone marrow stem cells (MSCs): a proposed treatment for multiple sclerosis and other neuroimmunological/neurodegenerative diseases. J Neurol Sci. 2008 Feb 15;265(1-2):131-5. doi: 10.1016/j.jns.2007.05.005. Epub 2007 Jul 3.
PubMed 17610906Schwarz J, Storch A. Transplantation in Parkinson's disease: will mesenchymal stem cells help to reenter the clinical arena? Transl Res. 2010 Feb;155(2):55-6. doi: 10.1016/j.trsl.2009.08.008. Epub 2009 Sep 19. No abstract available.
PubMed 20129484Glavaski-Joksimovic A, Virag T, Mangatu TA, McGrogan M, Wang XS, Bohn MC. Glial cell line-derived neurotrophic factor-secreting genetically modified human bone marrow-derived mesenchymal stem cells promote recovery in a rat model of Parkinson's disease. J Neurosci Res. 2010 Sep;88(12):2669-81. doi: 10.1002/jnr.22435.
PubMed 20544825Somoza R, Juri C, Baes M, Wyneken U, Rubio FJ. Intranigral transplantation of epigenetically induced BDNF-secreting human mesenchymal stem cells: implications for cell-based therapies in Parkinson's disease. Biol Blood Marrow Transplant. 2010 Nov;16(11):1530-40. doi: 10.1016/j.bbmt.2010.06.006. Epub 2010 Jun 10.
PubMed 20542127Shetty P, Ravindran G, Sarang S, Thakur AM, Rao HS, Viswanathan C. Clinical grade mesenchymal stem cells transdifferentiated under xenofree conditions alleviates motor deficiencies in a rat model of Parkinson's disease. Cell Biol Int. 2009 Aug;33(8):830-8. doi: 10.1016/j.cellbi.2009.05.002. Epub 2009 May 22.
PubMed 19465139Zhang Z, Wang X, Wang S. Isolation and characterization of mesenchymal stem cells derived from bone marrow of patients with Parkinson's disease. In Vitro Cell Dev Biol Anim. 2008 May-Jun;44(5-6):169-77. doi: 10.1007/s11626-008-9093-1. Epub 2008 Apr 10.
PubMed 18401666Park HJ, Lee PH, Bang OY, Lee G, Ahn YH. Mesenchymal stem cells therapy exerts neuroprotection in a progressive animal model of Parkinson's disease. J Neurochem. 2008 Oct;107(1):141-51. doi: 10.1111/j.1471-4159.2008.05589.x. Epub 2008 Jul 28.
PubMed 18665911
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PhaseⅠ/ⅡTrial of Autologous Bone Marrow Derived Mesenchymal Stem Cells to Patients With Parkinson's Disease.
- ClinicalTrials.gov ID
- NCT01446614
- Acronym / sponsor ID
- HM-2011-10
- Conditions
- Parkinson's Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Guangzhou General Hospital of Guangzhou Military CommandUniversity, hospital or other
- Study start
- Oct 2011
- Primary completion
- Jun 2013 (estimated)
- Study completion
- Jun 2014 (estimated)
- First posted
- 5 Oct 2011 (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 is a phase I/II trial designed to establish the safety and efficacy of intravenous administration of autologous bone marrow derived mesenchymal stem cells to patients with Parkinson's disease.
Detailed description
Parkinson's disease (PD) is a common progressive neurodegenerative disorder caused by the loss of dopaminergic neurons in the substantia nigra. A combination of genetic and environmental factors is likely to be important in producing abnormal protein aggregation within select groups of neurones, leading to cell dysfunction and then death. A large number of agents together with surgical interventions are now available to treat early and late complications of PD, but they are suffer from two main drawbacks: side effects and loss of efficacy with disease progression.
Bone marrow (BM) derived mesenchymal stem cells (MSCs) an differentiate under certain circumstances into cells from various neuronal and glial type lineages; they also exert immunomodulatory effects. PD-derived MSCs are similar to normal MSCs in phenotype, morphology, and multidifferentiation capacity. Moreover, PD-derived MSCs are capable of differentiating into neurons in a specific medium with up to 30% having the characteristics of dopamine cells. These findings indicate that MSCs derived from PD patients' bone marrow may be a promising cell type for cellular therapy.
BM-MSCs cultured with a cocktail of growth factors (containing FGF and BDNF) differentiate into neuronal/glial lineage cells with a predominance of cells expressing astrocytes' markers. They were effective in suppression of chronic EAE in mice and induced neuroprotection, preserving most of the axons in the CNS of successfully-treated animals. Histopathological studies revealed that MSCs could efficiently migrate into the CNS inflamed tissue (both when administered intravenously and intraventricularly) and differentiated into cells expressing neural-glial lineage markers. Such an approach may provide a feasible and practical way for PD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MSC | Experimental | Biological: bone marrow derived mesenchymal stem cellsIntravenous autologous bone marrow derived mesenchymal stem cells infusion to patients with Parkinson's disease. |
Who may qualify
- Ages
- 30 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patient with current diagnosis of idiopathic Parkinson's disease.
- Age 30 to 65.
- Experiencing motor complications despite optimized levodopa treatment.
- PD of Stage 2,2.5,3 or 4 of Hoehn-Yahr staging.
- Time between diagnosis and enrollment greater than 2 years.
Key exclusion criteria (10 listed)
- Patients may not be receiving any other investigational agents within 4 weeks of study entry.
- History of allergic reactions attributed to compounds of similar biologic composition to mesenchymal stem cells.
- Primary hematologic diseases.
- Patients undergo intracranial surgeries or implantation of a device for Parkinson's disease.
- Psychiatric, addictive or any other disorder that compromises ability to give a truly informed consent and perform all study assessments.
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 |
|---|---|---|
| bone marrow derived mesenchymal stem cellsAlso: Mesenchymal Stem Cells, Multipotent Mesenchymal Stem Cells, Multipotent Mesenchymal Stromal CellsIntravenous administration of up to 6x10^5 MSCs per kg,qw,for 4 weeks | Biological | MSC |
Primary outcome
Number of participants with adverse events
Time frame: 1 month after transplantation
4 secondary outcomes
Effect assessment
Time frame: 1 month after transplantation
Effect assessment
Time frame: 3 months after transplantation
Effect assessment
Time frame: 6 months after transplantation
Effect assessment
Time frame: 12 months after transplantation
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2011.
- Background: PubMed 18040857Wang Y, Chen S, Yang D, Le WD. Stem cell transplantation: a promising therapy for Parkinson's disease. J Neuroimmune Pharmacol. 2007 Sep;2(3):243-50. doi: 10.1007/s11481-007-9074-2. Epub 2007 May 9.
- Background: PubMed 17610906Karussis D, Kassis I, Kurkalli BG, Slavin S. Immunomodulation and neuroprotection with mesenchymal bone marrow stem cells (MSCs): a proposed treatment for multiple sclerosis and other neuroimmunological/neurodegenerative diseases. J Neurol Sci. 2008 Feb 15;265(1-2):131-5. doi: 10.1016/j.jns.2007.05.005. Epub 2007 Jul 3.
- Background: PubMed 20129484Schwarz J, Storch A. Transplantation in Parkinson's disease: will mesenchymal stem cells help to reenter the clinical arena? Transl Res. 2010 Feb;155(2):55-6. doi: 10.1016/j.trsl.2009.08.008. Epub 2009 Sep 19. No abstract available.
- Background: PubMed 20544825Glavaski-Joksimovic A, Virag T, Mangatu TA, McGrogan M, Wang XS, Bohn MC. Glial cell line-derived neurotrophic factor-secreting genetically modified human bone marrow-derived mesenchymal stem cells promote recovery in a rat model of Parkinson's disease. J Neurosci Res. 2010 Sep;88(12):2669-81. doi: 10.1002/jnr.22435.
- Background: PubMed 20542127Somoza R, Juri C, Baes M, Wyneken U, Rubio FJ. Intranigral transplantation of epigenetically induced BDNF-secreting human mesenchymal stem cells: implications for cell-based therapies in Parkinson's disease. Biol Blood Marrow Transplant. 2010 Nov;16(11):1530-40. doi: 10.1016/j.bbmt.2010.06.006. Epub 2010 Jun 10.
- Background: PubMed 19465139Shetty P, Ravindran G, Sarang S, Thakur AM, Rao HS, Viswanathan C. Clinical grade mesenchymal stem cells transdifferentiated under xenofree conditions alleviates motor deficiencies in a rat model of Parkinson's disease. Cell Biol Int. 2009 Aug;33(8):830-8. doi: 10.1016/j.cellbi.2009.05.002. Epub 2009 May 22.
- Background: PubMed 18401666Zhang Z, Wang X, Wang S. Isolation and characterization of mesenchymal stem cells derived from bone marrow of patients with Parkinson's disease. In Vitro Cell Dev Biol Anim. 2008 May-Jun;44(5-6):169-77. doi: 10.1007/s11626-008-9093-1. Epub 2008 Apr 10.
- Background: PubMed 18665911Park HJ, Lee PH, Bang OY, Lee G, Ahn YH. Mesenchymal stem cells therapy exerts neuroprotection in a progressive animal model of Parkinson's disease. J Neurochem. 2008 Oct;107(1):141-51. doi: 10.1111/j.1471-4159.2008.05589.x. Epub 2008 Jul 28.
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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.
Yang Xiao, MD study director
Guangzhou General Hospital of Guangzhou Military Command
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 bone marrow derived mesenchymal stem cells in people with Parkinson's Disease, run by Guangzhou General Hospital of Guangzhou Military Command 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 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: status not confirmed, as of the sponsor's last update on 5 Oct 2011. 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.
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 Yang Xiao, MD (86-20-36653562, jdxiao111@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with adverse events (time frame: 1 month after transplantation). 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 NCT01446614, which also lists 8 linked publications.
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 5 Oct 2011.