NeurodegenerationPhase 1/2NCT02983708
Neuroregenerative Potential of Intravenous G-CSF and Autologous Peripheral Blood Stem Cells
A Phase 1/2 study testing Peripheral blood mononuclear cells (mPBMC) and G-CSF in people with Neurodegeneration and G-CSF, run by Hanyang University Seoul Hospital.
Trial summary
The current study describes a randomized, double-blind, cross-over study of intravenous G-CSF followed by infusion with autologous mobilized peripheral blood mononuclear cells (mPBMCs) in children with cerebral palsy (CP) to determine the safety and feasibility of the procedure, as well as the potential efficacy for… Read more about this study
- Who is it for?
- Ages 2 to 10 years
- Conditions the study is for
- Neurodegeneration, G-CSF, Peripheral Blood Mononuclear Cells
- What is being tested?
- Peripheral blood mononuclear cells (mPBMC) and G-CSF
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 57 participants
- Where
- No sites listed yet
- Study dates
- Aug 2011to Sep 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 2 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Non severe type of cerebral palsy
- Evidences of abnormal MRI findings such as periventricular leukomalacia
- Collected mobilized peripheral blood mononuclear cell counts > 1×10^8/kg or CD34+ cell counts > 1×10^6/kg
- Consent form
Who cannot join
- Previous trials of autologous cord blood infusion or erythropoietin/G-CSF
- Chromosomal abnormalities
About this study
Official title: Neuroregenerative Potential of Intravenous G-CSF and Autologous Peripheral Blood Stem Cells in Children With Cerebral Palsy: a Randomized, Double-blind Cross-over Study
The current study describes a randomized, double-blind, cross-over study of intravenous G-CSF followed by infusion with autologous mobilized peripheral blood mononuclear cells (mPBMCs) in children with cerebral palsy (CP) to determine the safety and feasibility of the procedure, as well as the potential efficacy for improving neurological impairment.
Read the detailed description
We hypothesized that mobilized peripheral blood mononuclear cells (mPBMCs) would be a better source of cell therapy for children with CP, if these cells had a similar neuroregenerative potential to bone marrow/cord blood mononuclear cells (MNCs). Multipotent precursor cells exist in peripheral blood, and a fraction of elutriated blood cells from normal individuals contains MNCs that have the potential to be MSCs. There are several advantages to using mPBMCs for cell therapy in children with CP: the G-CSF that is used to mPBMCs has neuroregenerative potential; the collection and fractionation of stem cells can be repeated; and, the therapy is suitable for most children with CP.
- Being tested
- Peripheral blood mononuclear cells (mPBMC), G-CSF, Placebo
- Keywords
- Neuroregeneration, G-CSF, Peripheral blood mononuclear cells, Cerebral palsy
- Medical subject headings
- Nerve Degeneration, Cerebral Palsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Peripheral blood mononuclear cells (mPBMC)Biological
G-CSFDrug
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| mPBMC group | Experimental | G-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. mPBMCs group would be included all patients who received mPBMCs at M1 or M7. Receives: Biological: Peripheral blood mononuclear cells (mPBMC); Drug: G-CSF |
| Placebo group | Placebo comparator | G-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. Placebo group would be included all patients who received placebo at M1 or M7. Receives: Drug: G-CSF; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall improvement as a score changes in GMFM > 4 points
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 posted6 Dec 2016 (estimated)
- Study startAug 2011
- Primary completionSep 2014
- Study completionSep 2014
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 NeurodegenerationStudies 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
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 57 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hanyang University Seoul Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Ministry of Health & Welfare, Korea
- Responsible party
- Principal investigator, Young-Ho Lee, Hanyang University Seoul Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02983708
- Sponsor's protocol ID
- CP-PB-2011
- Status last verified
- Dec 2016
- First submitted
- 2 Dec 2016
- Last updated
- 7 Dec 2016
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided: We have to get individual permissions to make IPD available to other researchers.
Publications
Rah WJ, Lee YH, Moon JH, Jun HJ, Kang HR, Koh H, Eom HJ, Lee JY, Lee YJ, Kim JY, Choi YY, Park K, Kim MJ, Kim SH. Neuroregenerative potential of intravenous G-CSF and autologous peripheral blood stem cells in children with cerebral palsy: a randomized, double-blind, cross-over study. J Transl Med. 2017 Jan 21;15(1):16. doi: 10.1186/s12967-017-1120-0.
PubMed 28109298
Study at a glance
From the registry record, last updated by the sponsor on 7 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neuroregenerative Potential of Intravenous G-CSF and Autologous Peripheral Blood Stem Cells in Children With Cerebral Palsy: a Randomized, Double-blind Cross-over Study
- ClinicalTrials.gov ID
- NCT02983708
- Acronym / sponsor ID
- CP-PB-2011
- Keywords
- Neuroregeneration, G-CSF, Peripheral blood mononuclear cells, Cerebral palsy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 57 participants (actual)
- Lead sponsor
- Hanyang University Seoul HospitalUniversity, hospital or other
- Collaborators
- Ministry of Health & Welfare, Korea
- Study start
- Aug 2011
- Primary completion
- Sep 2014 (actual)
- Study completion
- Sep 2014 (actual)
- First posted
- 6 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.
The current study describes a randomized, double-blind, cross-over study of intravenous G-CSF followed by infusion with autologous mobilized peripheral blood mononuclear cells (mPBMCs) in children with cerebral palsy (CP) to determine the safety and feasibility of the procedure, as well as the potential efficacy for improving neurological impairment.
Detailed description
We hypothesized that mobilized peripheral blood mononuclear cells (mPBMCs) would be a better source of cell therapy for children with CP, if these cells had a similar neuroregenerative potential to bone marrow/cord blood mononuclear cells (MNCs). Multipotent precursor cells exist in peripheral blood, and a fraction of elutriated blood cells from normal individuals contains MNCs that have the potential to be MSCs. There are several advantages to using mPBMCs for cell therapy in children with CP: the G-CSF that is used to mPBMCs has neuroregenerative potential; the collection and fractionation of stem cells can be repeated; and, the therapy is suitable for most children with CP.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| mPBMC group | Experimental | Biological: Peripheral blood mononuclear cells (mPBMC), Drug: G-CSFG-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. mPBMCs group would be included all patients who received mPBMCs at M1 or M7. |
| Placebo group | Placebo comparator | Drug: G-CSF, Drug: PlaceboG-CSF would be administered for 5 days and then mobilized peripheral blood mononuclear cells (mPBMCs) would be collected in all included patients. One month after cryopreservation of the mPBMCs (M1), patients will be randomized to receive either mPBMCs or placebo. Six months after randomization (M7), cross-over infusion of mPBMCs or placebo will be performed and the patients are observed for another 6 months. Placebo group would be included all patients who received placebo at M1 or M7. |
Who may qualify
- Ages
- 2 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Non severe type of cerebral palsy
- Evidences of abnormal MRI findings such as periventricular leukomalacia
- Collected mobilized peripheral blood mononuclear cell counts > 1×10^8/kg or CD34+ cell counts > 1×10^6/kg
- Consent form
Key exclusion criteria (2 listed)
- Previous trials of autologous cord blood infusion or erythropoietin/G-CSF
- Chromosomal abnormalities
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 |
|---|---|---|
| Peripheral blood mononuclear cells (mPBMC) | Biological | mPBMC group |
| G-CSF | Drug | Placebo group, mPBMC group |
| Placebo | Drug | Placebo group |
Primary outcome
Overall improvement as a score changes in GMFM > 4 points
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 7 Dec 2016.
- Derived from this study: PubMed 28109298Rah WJ, Lee YH, Moon JH, Jun HJ, Kang HR, Koh H, Eom HJ, Lee JY, Lee YJ, Kim JY, Choi YY, Park K, Kim MJ, Kim SH. Neuroregenerative potential of intravenous G-CSF and autologous peripheral blood stem cells in children with cerebral palsy: a randomized, double-blind, cross-over study. J Transl Med. 2017 Jan 21;15(1):16. doi: 10.1186/s12967-017-1120-0.
- Individual participant dataSharing planned: Undecided. We have to get individual permissions to make IPD available to other researchers.
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Peripheral blood mononuclear cells (mPBMC) and G-CSF in people with Neurodegeneration and G-CSF, run by Hanyang University Seoul Hospital.
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 4 inclusion and 2 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 Dec 2016. Status can change between updates, so confirm with the study contact before making plans.
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?
Overall improvement as a score changes in GMFM > 4 points (time frame: 6 months). 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 NCT02983708, 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 7 Dec 2016.