Clinical trial NCT03117738: A Study to Evaluate the Safety and Efficacy of AstroStem in Treatment of Alzheimer's Disease
Official title: A Phase 1/2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of AstroStem, Autologous Adipose Tissue Derived Mesenchymal Stem Cells, in Patients With Alzheimer's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Alzheimer Disease
Lead sponsor: Nature Cell Co. Ltd.
Sponsor class: INDUSTRY
First posted: 2017-04-18
Enrollment: 21
Sex: ALL
Minimum age (years): 50
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Treatment Related Adverse Events
timeFrame: 30 Weeks
measure: ADAS-Cog (Alzheimer's Disease Assessment Scale-cognitive Subscale)
timeFrame: Baseline and 30 Weeks
Secondary outcomes:
measure: MMSE (Mini-mental Status Examination)
timeFrame: Baseline and 30 Weeks
measure: CDR-SOB (Clinical Dementia Rating-Sum of Boxes)
timeFrame: Baseline and 30 Weeks
measure: NPI (Neuropsychiatric Inventory)
timeFrame: Baseline and 30 Weeks
measure: GDS (Geriatric Depression Scale)
timeFrame: Baseline and 30 Weeks
measure: ADCS-ADL (Alzheimer's Disease Cooperative Study Activities of Daily Living)
timeFrame: Baseline and 30 Weeks
measure: C-SSRS (Columbia Suicide Severity Rating Scale)
timeFrame: Baseline and 30 Weeks
Other outcomes:
Locations:
city: Costa Mesa
country: United States
facility: ATP Clinical Research
state: California
city: Santa Ana
country: United States
facility: Syrentis Clinical Research
state: California
city: Honolulu
country: United States
facility: Valden Medical
state: Hawaii
Study officials:
References: