The Long-Term Safety and Efficacy Follow-Up Study of Subjects Who Completed the Phase I Clinical Trial of Neurostem®-AD

September 27, 2012 updated by: Duk Lyul Na

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Unknown

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
14

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • Name: Duk-Lyul Na, MD, PhD
  • Phone Number: +82-2-3410-3594
  • Email: dukna@naver.com

Study Locations

  • Korea, Republic of
      • Seoul, Korea, Republic of, 135-710
        • Recruiting
        • Samsung Medical Center
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

50 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

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.

Description

TEST GROUP

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • Subjects with a psychological disease (i.e. depression, schizophrenia, bipolar disorder, etc)
  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Control
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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
Biological: NEUROSTEM®-AD
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.
Other Names:
  • human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs)
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 the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety
Time Frame: upto 24 months post-op
Incidence rate ot adverse events (vital signs, physical examination, mixed lymphocyte reaction, and laboratory tests)
upto 24 months post-op

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Efficacy
Time Frame: upto 24 months post-op

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)
upto 24 months post-op

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Duk Lyul Na

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Medipost Co Ltd.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Duk L. Na, MD,PhD, Samsung Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2012

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 1, 2013

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 1, 2013

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 7, 2012

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 27, 2012

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 1, 2012

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 1, 2012

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 27, 2012

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2012

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • MP-CR-007-F/U

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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