A Study to Evaluate the Safety and Efficacy of AstroStem in Treatment of Alzheimer's Disease

Phase 1

Completed

Conditions: Alzheimer Disease

Interventions: Other: Placebo-Control
Drug: AstroStem

Registration Number: NCT03117738

Lead Sponsor: Nature Cell Co. Ltd.

Brief Summary: This is a randomized, double-blind, placebo-controlled, parallel-group comparison study in subjects with Alzheimer's Disease. Following first screening period, subjects will be randomly assigned into one of the following arms: AstroStem and placebo control in a 1:1 ratio. AstroStem or placebo control will be administered via I.V. at Week 0. This procedure will be repeated 9 times at 2-week interval. Subjects will be scheduled for two follow-up visits at Weeks 30 and 52 to evaluate primary and secondary outcome endpoints.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 21

Inclusion Criteria

Male or female subjects aged 50 and above at the time of signing the Informed Consent form
Subjects who can understand and provide written informed consent (assent)
Subjects who have diagnosis of probable mild-to-moderate Alzheimer disease according to NINCDS-ADRDA (National Institute of Neurological and Communicative Disorders and Stroke; Alzheimer's Disease and Related Disorders Association) criteria
Subjects who have MMSE Score of 16 to 26 at screening
Subjects who are taking FDA-approved AD medications (donepezil, galantamine, memantine, rivastigmine or their combinations) treatment on a stable dosage for at least 3 months prior to screening.
Subjects who have one (or more) identified adult caregiver who is able to read, understand, and speak the designated language at the study site; either lives with the subject or sees the subject for ≥2 hours/day ≥4 days/week; and agrees to accompany the subject to each study visit
Subjects who have a designated study partner who will accompany the subject to all clinic visits and participate in the subject's clinical assessments

Exclusion Criteria

Subjects who are females who are pregnant, nursing, or of childbearing potential while not practicing effective contraception
Subjects who have signs of delirium
Subjects who have had cortical stroke within the preceding 2 years
Subjects who have a prolonged QTc interval; >450 msec in male or >470 msec in female at screening
Subjects who have diagnosis of severe white matter hyperintensity (WMH), which is defined as ≥ 25mm of the deep white matter and ≥ 10mm of the periventricular capping/banding in lengths
Subjects who have diagnosis of dementia or cause of cognitive impairment other than Alzheimer's disease
Subjects who have a significant abnormal result in laboratory tests, in the opinion of the investigator
Subjects who have participated in any investigational drug, stem cell therapy, or device trial within the previous 3 months at screening
Subjects with any current psychiatric diagnosis other than AD if, in the judgment of the investigator, the psychiatric disorder or symptom is likely to confound interpretation of drug effect, affect cognitive assessments, or affect the subject´s ability to complete the study
Subjects who are known to have autosomal dominant mutation-associated presenile AD
Subjects who show signs of AIDS (Acquired Immunodeficiency Syndrome), HBV (Hepatitis B Virus), HCV (Hepatitis C), VDRL (Venereal Disease Research Laboratory)
Subjects who have any conditions that would contraindicate an MRI, such as the presence metallic objects in the eyes, skin, or heart
Subjects who have > 4 cerebral microhemorrhages (regardless of their anatomical location or diagnostic characterization as "possible" or "definite"), a single area of superficial siderosis, or evidence of a prior microhemorrhage as assessed by MRI
Subjects who have history of malignant cancer within the last 5 years (The following is a partial list of conditions that are permissible for study entry: non-metastatic basal and/or squamous cell carcinoma of the skin, in situ cervical, or non-progressive prostate cancer)
Subjects who have suspected active lung disease based on chest X-ray
Subjects who are hypersensitive to fetal bovine serum or penicillin
Subjects who are currently using immunosuppressants, cytotoxic drug, corticosteroids or similar steroidal anti-inflammatory medication (e.g., Prednisone) on a regular basis (exceptions allowed include; regular use of steroidal nasal sprays, topical steroids, and estrogen-replacement therapy)
Subjects for whom the investigator judges the liposuction can cause any problems
Subjects who have history of local anesthetic allergy

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo-Control	Placebo-Control	-
AstroStem	AstroStem	-

Primary Outcome Measures

Name	Time	Method
Treatment Related Adverse Events	30 Weeks	Number of subjects with treatment related adverse events as assessed by analysis of adverse events including symptoms, and abnormal findings on physical examination, vital signs, ECG, and standard laboratory examination results
ADAS-Cog (Alzheimer's Disease Assessment Scale-cognitive Subscale)	Baseline and 30 Weeks	Change of ADAS-Cog (Alzheimer's Disease Assessment Scale-cognitive subscale) from Baseline at Week 30 Score range: 0-70 A score of 70 represents the most severe impairment and 0 represents the least impairment

Secondary Outcome Measures

Name	Time	Method
C-SSRS (Columbia Suicide Severity Rating Scale)	Baseline and 30 Weeks	Changes of C-SSRS from baseline at Week 30 Score range: 0-25 Higher score indicates higher severity.
MMSE (Mini-mental Status Examination)	Baseline and 30 Weeks	Change of MMSE from baseline at Week 30 Score range: 0-30 A score of 20 to 24 suggests mild dementia, 13 to 20 suggests moderate dementia, and less than 12 indicates severe dementia.
NPI (Neuropsychiatric Inventory)	Baseline and 30 Weeks	Changes of NPI from baseline at Week 30 Score range: 0-144 higher score indicates higher disturbance.
CDR-SOB (Clinical Dementia Rating-Sum of Boxes)	Baseline and 30 Weeks	Changes of CDR-SOB from baseline at Week 30 Score range: 0-18.0 0 = normal, 0.5-4.0 = questionable cognitive dementia, 4.5-9.0 = mild dementia, 9.5-15.5 = moderate dementia, and 16.0-18.0 = severe dementia
GDS (Geriatric Depression Scale)	Baseline and 30 Weeks	Changes of GDS from baseline at Week 30 Score range: 0-15 Scores of 0-4 are considered normal, depending on age, education, and complaints; 5-8 indicate mild depression; 9-11 indicate moderate depression; and 12-15 indicate severe depression.
ADCS-ADL (Alzheimer's Disease Cooperative Study Activities of Daily Living)	Baseline and 30 Weeks	Change of ADCS-ADL from baseline at Week 30 Score range: 0-78 Lower score indicates greater severity.

Trial Locations

Locations (3): ATP Clinical Research
🇺🇸
Costa Mesa, California, United States
Syrentis Clinical Research
🇺🇸
Santa Ana, California, United States
Valden Medical
🇺🇸
Honolulu, Hawaii, United States