Alzheimer DiseaseNCT01696591
The Long-Term Safety and Efficacy Follow-Up Study of Subjects Who Completed the Phase I Clinical Trial of Neurostem®-AD
An observational study of Alzheimer Disease and Dementia, run by Duk Lyul Na at 1 site in South Korea.
Trial summary
The purpose of the study is to determine the long-term safety and exploratory efficacy of NEUROSTEM®-AD, administered via an open brain surgery to subjects with dementia of the Alzheimer's type, who were eligible for and enrolled in the earlier part of the phase I. Read more about this study
- Who is it for?
- Ages 50 to 75 years
- Conditions the study is for
- Alzheimer Disease, Dementia, Brain Diseases
- What is being tested?
- NEUROSTEM®-AD
- How many people
- 14 participants
- Where
- 1 siteSouth Korea
- Study dates
- Mar 2012to Sep 2013
- Phase
- Observational
- 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 50 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The patients, who were administered with NEUROSTEM®-AD in the earlier part of the Phase I study, will be compared with the patients, who have similar demographics and disease characteristics as the subjects in the test group but have not been treated with NEUROSTEM®-AD.
Who may be able to join
- Subjects who have enrolled and completed the Phase I cliical trial: The Safety and The Efficacy Evaluation of NEUROSTEM®-AD in Patients With Alzheimer's Disease
- Subjects who are willing to participate in the study and sign the consent form
- Patients with a moderate alzheimer's disease, diagnosed with a dementia of alzheimer's type, according to the DSM-VI and NINCDS-ADRDA criteria, and shows amyloid-positive in a PIB-PET
Who cannot join
- Females who are pregnant or nursing
- Subjects who have participated in another clinical study within the 3 months prior to the initiation of this study
- Subjects who are restricted from undergoing exams perfomed during the study (i.e. MRI, CT, or PET screening)
- Subjects who the principal investigator considers inappropriate for participation in the study due to any reasons other than those listed above
- CONTROL GROUP
- Subjects with a psychological disease (i.e. depression, schizophrenia, bipolar disorder, etc)
Show all 26Show fewer
- Subjects with a dementia caused by other degenerative neurological diseases (infection of the CNS, such as HIV or Syphilis), head trauma, Creutzfeld-Jacob disease, Pick's disease, Huntington's disease, and Parkinson's disease)
- Subjects with a vascular dementia as determined by the clinical criteria of DSM IV and the imaging criteria of Erkinjuntii
- Subjects with severe white matter hyperintensities (WMH); Severe WMH is defined as, according to Clinical Research Center for Dementia of South Korea, a condition in which the deep white matter is 25 mm or greater and the periventricular capping/banding is 10 mm or greater in lengths.
- Subjects with a history of stroke within the 3 months prior to the study enrollment
- Subjects with a severe liver disease (ALT/AST values are higher than twice the normal range)
- Subjects with a severe renal disease (1.5mg/dL or more of serum creatinine)
- Pregnant or lactating women
- Subjects with abnormal findings of the clinical laboratory values at Visit 1:
- Hemoglobin < 9.5g/dL in male < 9.0 g/dL in female
- Total WBC count < 3000/mm3
- Total bilirubin ≥ 3 mg/dL
- Subjects with a suspected active lung disease, based on the chest X-ray result at Visit 1
- Females of childbearing age who does not practice medically acceptable method of contraception during the study
- Subjects who have previously failed Screening for participation in this study
- Subjects who have participated in another clinical study within the 3 months prior to the initiation of this study
- Subjects with a bleeding disorder (platelet count < 150,000/mm3; PT ≥ 1.5; INR or aPTT ≥ 1.5 X control
- Subjects with a cancer (including brain tumor)
- Subjects with a history of alcohol or drug abuse
- Subjects who are restricted from undergoing exams perfomed during the study (i.e. MRI, CT, or PET screening)
- Patients who the principal investigator considers inappropriate for participation in the study due to any reasons other than those listed above
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Samsung Medical Center
Seoul, 135-710
Duk L. Na, MD, PhD, +82-2-3410-3594, dukna@naver.com
About this study
Official title: The Long-Term Safety and Efficacy Follow-up Study of Subjects Who Completed the Phase I Clinical Trial of Neurostem®-AD
The purpose of the study is to determine the long-term safety and exploratory efficacy of NEUROSTEM®-AD, administered via an open brain surgery to subjects with dementia of the Alzheimer's type, who were eligible for and enrolled in the earlier part of the phase I. Aside from the subjects who completed the earlier part of the Phase I, 3 additional subjects with comparable demographics and disease characteristics as the treatment group will be enrolled into a control group, followed-up for 3 months, and compared for various disease progression indicators with the treatment group.
The hypothesis is that NEUROSTEM®-AD is safe and effective in the treatment of dementia of the Alzheimer's type.
Read the detailed description
This is a long-term follow up study of the earlier part of the phase I, during which the safe and effective dose(safety) of NEUROSTEM®-AD was determined for implantation into the brains of subjects with Dementia of the Alzheimer's type. Subjects with Dementia of the Alzheimer's type, who signed the informed consent form and meet the eligibility criteria, were implanted with a single dose of NEUROSTEM®-AD, hUBC-MSCs, into the brain. The subjects were hospitalized for 5 to 10 days following the surgical implantation and were observed for acute adverse events: Gradient echo MRI within the the 24 hours post-op, vital signs, clinical laboratory tests, chest x-rays within Day 2. On Day 14, DLT was assessed, and the subjects were followed up on the safety and disease progression of dementia (of the Alzheimer's type) for 12 weeks post-implantation.
In this part of the study, the subjects described above will be followed-up for upto Month 24, and 3 additional subjects with comparable demographics and disease characteristics as the treatment group (refer to Inclusion/Exclusion Criteria) will be enrolled as a control group, followed up for 3 months and compared with the treatment group for various indicators of the disease progression.
- Conditions
- Alzheimer Disease, Dementia, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Tauopathies
- Being tested
- NEUROSTEM®-AD
- Keywords
- Alzheimer, Mesenchymal Stem Cells, Umbilical Cord Blood
- Medical subject headings
- Alzheimer Disease, Dementia, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Tauopathies, Neurodegenerative Diseases, Neurocognitive Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NEUROSTEM®-ADBiological
Also called human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs)
NEUROSTEM®-AD was administered to eligible subjects in the early part of the Phase I clinical study. In this follow-up study, no intervention will be performed.
| Study group | Role | What they receive |
|---|---|---|
| NEUROSTEM®-AD | A single administration of human umbilical cord blood-derived mesenchymal stem cells through a brain surgery
DOSE A - 250,000 cells per entry site, 3 million cells per brain; DOSE B - 500,000 cells per entry site, 6 million cells per brain Receives: Biological: NEUROSTEM®-AD | |
| Control Group | A group of subjects with comparable demographics (age and gender) and disease characteristics [Clinical Dementia Rating Scale Sum of Boxes (CDR-SOB)] as the NEUROSTEM®-AD-treated group, but did not receive the treatment with NEUROSTEM®-AD and were continued on conventional therapy. Restrictions in the concurrent use of drug therapy are as follows: Patients are, in principle, permitted to continue the drug therapy they were on prior to the enrollment, for the treatment of concurrent illnesses other than Dementia, such as hypertension, diabetes mellitus, or hyperlipidemia. For drugs used in the treatment of dementia, behavior-modifying drugs can be added to the pharmacological regimen of a subject during the course of the study. However, adding a new cognitive enhancer, such as donepezil, memantine, galantamine, rivastigmine, is not permitted while dose adjustment is permitted given that the drug had been in use prior to the initiation of the study. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
Incidence rate ot adverse events (vital signs, physical examination, mixed lymphocyte reaction, and laboratory tests)
Measured: upto 24 months post-op
Secondary outcomes (1)
Efficacy
Primary Efficacy Variable:
ADAS-cog response rate, ADAS-cog response is defined as when ADAS-cog score at the end of the study is not worse than the Baseline score.
Secondary Efficacy Variables:
- Changes in Seoul Instrumental Activities of Daily Living (S-IADL)
- Changes in Mini Mental State Examination Korean verson (K-MMSE)
- Changes in Caregiver-administered Neuropsuchiatric Inventory
- Changes in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog)
- Changes in CMRglc: regional cerebral metabolic rate for glucose (FDG-PET)
Measured: upto 24 months post-op
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2012 (estimated)
- Study startMar 2012
- Primary completionSep 2013 (estimated)
- Study completionSep 2013 (estimated)
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 Alzheimer 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 14 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Duk Lyul Na
- Sponsor type
- University, hospital or other
- Collaborators
- Medipost Co Ltd.
- Responsible party
- Sponsor-investigator, Duk Lyul Na, Samsung Medical Center
- Investigators
- Duk L. Na, MD,PhD, Principal investigator, Samsung Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01696591
- Sponsor's protocol ID
- MP-CR-007-F/U
- Status last verified
- Sep 2012
- First submitted
- 7 Sep 2012
- Last updated
- 1 Oct 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kim HJ, Seo SW, Chang JW, Lee JI, Kim CH, Chin J, Choi SJ, Kwon H, Yun HJ, Lee JM, Kim ST, Choe YS, Lee KH, Na DL. Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: A phase 1 clinical trial. Alzheimers Dement (N Y). 2015 Jul 26;1(2):95-102. doi: 10.1016/j.trci.2015.06.007. eCollection 2015 Sep.
PubMed 29854930
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Long-Term Safety and Efficacy Follow-up Study of Subjects Who Completed the Phase I Clinical Trial of Neurostem®-AD
- ClinicalTrials.gov ID
- NCT01696591
- Acronym / sponsor ID
- MP-CR-007-F/U
- Conditions
- Alzheimer Disease, Dementia, Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Tauopathies
- Keywords
- Alzheimer, Mesenchymal Stem Cells, Umbilical Cord Blood
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 14 participants (estimated)
- Lead sponsor
- Duk Lyul NaUniversity, hospital or other
- Collaborators
- Medipost Co Ltd.
- Study start
- Mar 2012
- Primary completion
- Sep 2013 (estimated)
- Study completion
- Sep 2013 (estimated)
- First posted
- 1 Oct 2012 (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 purpose of the study is to determine the long-term safety and exploratory efficacy of NEUROSTEM®-AD, administered via an open brain surgery to subjects with dementia of the Alzheimer's type, who were eligible for and enrolled in the earlier part of the phase I. Aside from the subjects who completed the earlier part of the Phase I, 3 additional subjects with comparable demographics and disease characteristics as the treatment group will be enrolled into a control group, followed-up for 3 months, and compared for various disease progression indicators with the treatment group.
The hypothesis is that NEUROSTEM®-AD is safe and effective in the treatment of dementia of the Alzheimer's type.
Detailed description
This is a long-term follow up study of the earlier part of the phase I, during which the safe and effective dose(safety) of NEUROSTEM®-AD was determined for implantation into the brains of subjects with Dementia of the Alzheimer's type. Subjects with Dementia of the Alzheimer's type, who signed the informed consent form and meet the eligibility criteria, were implanted with a single dose of NEUROSTEM®-AD, hUBC-MSCs, into the brain. The subjects were hospitalized for 5 to 10 days following the surgical implantation and were observed for acute adverse events: Gradient echo MRI within the the 24 hours post-op, vital signs, clinical laboratory tests, chest x-rays within Day 2. On Day 14, DLT was assessed, and the subjects were followed up on the safety and disease progression of dementia (of the Alzheimer's type) for 12 weeks post-implantation.
In this part of the study, the subjects described above will be followed-up for upto Month 24, and 3 additional subjects with comparable demographics and disease characteristics as the treatment group (refer to Inclusion/Exclusion Criteria) will be enrolled as a control group, followed up for 3 months and compared with the treatment group for various indicators of the disease progression.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| NEUROSTEM®-AD | – | Biological: NEUROSTEM®-ADA single administration of human umbilical cord blood-derived mesenchymal stem cells through a brain surgery DOSE A - 250,000 cells per entry site, 3 million cells per brain; DOSE B - 500,000 cells per entry site, 6 million cells per brain |
| Control Group | – | –A group of subjects with comparable demographics (age and gender) and disease characteristics [Clinical Dementia Rating Scale Sum of Boxes (CDR-SOB)] as the NEUROSTEM®-AD-treated group, but did not receive the treatment with NEUROSTEM®-AD and were continued on conventional therapy. Restrictions in the concurrent use of drug therapy are as follows: Patients are, in principle, permitted to continue the drug therapy they were on prior to the enrollment, for the treatment of concurrent illnesses other than Dementia, such as hypertension, diabetes mellitus, or hyperlipidemia. For drugs used in the treatment of dementia, behavior-modifying drugs can be added to the pharmacological regimen of a subject during the course of the study. However, adding a new cognitive enhancer, such as donepezil, memantine, galantamine, rivastigmine, is not permitted while dose adjustment is permitted given that the drug had been in use prior to the initiation of the study. |
Who may qualify
- Ages
- 50 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The patients, who were administered with NEUROSTEM®-AD in the earlier part of the Phase I study, will be compared with the patients, who have similar demographics and disease characteristics as the subjects in the test group but have not been treated with NEUROSTEM®-AD.
Key inclusion criteria (3 listed)
- Subjects who have enrolled and completed the Phase I cliical trial: The Safety and The Efficacy Evaluation of NEUROSTEM®-AD in Patients With Alzheimer's Disease
- Subjects who are willing to participate in the study and sign the consent form
- Patients with a moderate alzheimer's disease, diagnosed with a dementia of alzheimer's type, according to the DSM-VI and NINCDS-ADRDA criteria, and shows amyloid-positive in a PIB-PET
Key exclusion criteria (26 listed)
- Females who are pregnant or nursing
- Subjects who have participated in another clinical study within the 3 months prior to the initiation of this study
- Subjects who are restricted from undergoing exams perfomed during the study (i.e. MRI, CT, or PET screening)
- Subjects who the principal investigator considers inappropriate for participation in the study due to any reasons other than those listed above
- CONTROL GROUP
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 |
|---|---|---|
| NEUROSTEM®-ADAlso: human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs)NEUROSTEM®-AD was administered to eligible subjects in the early part of the Phase I clinical study. In this follow-up study, no intervention will be performed. | Biological | NEUROSTEM®-AD |
Primary outcome
Safety
Time frame: upto 24 months post-op
Incidence rate ot adverse events (vital signs, physical examination, mixed lymphocyte reaction, and laboratory tests)
1 secondary outcome
Efficacy
Time frame: upto 24 months post-op
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2012.
- Derived from this study: PubMed 29854930Kim HJ, Seo SW, Chang JW, Lee JI, Kim CH, Chin J, Choi SJ, Kwon H, Yun HJ, Lee JM, Kim ST, Choe YS, Lee KH, Na DL. Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: A phase 1 clinical trial. Alzheimers Dement (N Y). 2015 Jul 26;1(2):95-102. doi: 10.1016/j.trci.2015.06.007. eCollection 2015 Sep.
- The Safety and The Efficacy Evaluation of NEUROSTEM®-AD in Patients With Alzheimer's DiseaseLink provided by the sponsor.
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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.
Duk L. Na, MD,PhD principal investigator
Samsung Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alzheimer Disease and Dementia, run by Duk Lyul Na at 1 site in South Korea.
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 26 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 1 Oct 2012. 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 South Korea. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Duk-Lyul Na, MD, PhD (+82-2-3410-3594, dukna@naver.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (time frame: upto 24 months post-op). 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 NCT01696591, 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 1 Oct 2012.