Clinical trial NCT03724136: Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study
Official title: Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: NA
Conditions: Alzheimer Disease
Alzheimer Dementia
Vascular Dementia
Lewy Body Disease
Lewy Body Dementia With Behavioral Disturbance (Disorder)
Dementia, Mixed
Parkinson-Dementia Syndrome
Chronic Traumatic Encephalopathy
Huntington's Dementia
Wernicke Korsakoff Syndrome
Traumatic Brain Injury
Dementia, Multi-Infarct
Autism
Autism Spectrum Disorder
Autistic Behavior
Autistic Disorder, Current or Active State
Cadasil
LATE Limbic-predominant Age-related TDP-43 Encephalopathy
Keywords: Dementia
Alzheimer's Disease
CADASIL
Autism Spectrum Disorder
Adult Autism
High Functioning Autism
Asperger's Syndrome
CTE Chronic Traumatic Encephalopathy
TBI Traumatic Brain Injury
LATE Limbic-predominant Age-related TDP-43 Encephalopathy
Lead sponsor: MD Stem Cells
Sponsor class: INDUSTRY
First posted: 2018-10-30
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Patients will be assigned to one of 3 arms with monitoring and retesting at 1,3,6 and 12 months following treatment.
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to evaluate the use of autologous Bone Marrow Derived Stem Cells (BMSC) as a means to improve cognitive impairment as occurs in Alzheimer's Disease and other dementias and to improve behavior and socialization issues which occur in adult Autism Spectrum Disorder. The use of Near Infrared Light, in conjunction with the use of BMSC, will also be assessed.

Detailed description: Cognition is the process of generating thoughts, recalling memories, processing information, and higher order associations including social interactivity that all take place in the brain. It requires sufficient health and interactivity of neurons in the brain including their ability to form and maintain synaptic connections.

Cognitive impairment results from the loss of these abilities. ACIST will test the hypothesis that the delivery of Bone Marrow Derived Stem Cells (BMSC) via the methods in the study with or without the addition of Near Infrared Light will improve cognition through the ability of BMSC to positively affect the health and function of neurons and the brain.

Patients enrolling with cognitive impairment will require assessment with the Mini-Mental Status Exam (MMSE). A score of 24 or less will be required. Progressive dementias such as Alzheimer's Disease (ALZ) show a decline of 2 to 4 points per year on MMSE. The goal for ACIST in progressive and stable dementia will be stability over the 1 year follow up and ideally an improvement of 3 points on MMSE.

Patients enrolling with Autism Spectrum Disorder (ASD) will be required to be adults (over 18 years of age) and to have a score on the Autism Spectrum Quotient of 20 or above. The goal will be a decrease of 5 or more on the scale over the 1 year follow up period.

Eligibility criteria: Inclusion Criteria:

1. Have documented cognitive impairment or diagnosis of disease associated with cognitive impairment such as Alzheimer's Disease, Autism Spectrum Disorder.
2. If under current medical therapy (pharmacologic or surgical treatment) for the condition be considered stable on that treatment and unlikely to have reversal of the associated cognitive impairment as a result of the ongoing pharmacologic or surgical treatment.
3. In the estimation of the investigator have the potential for improvement with BMSC treatment and be at minimal risk of any potential harm from the procedure.
4. Be over the age of 18
5. Be medically stable and able to be medically cleared by their primary care physician or a licensed primary care practitioner for the procedure. Medical clearance means that in the estimation of the primary care practitioner, the patient can reasonably be expected to undergo the procedure without significant medical risk to health.

Exclusion Criteria:

1. All patients must be capable of an adequate neurologic examination and evaluation to document the pathology.
2. Patients must be capable and willing to undergo follow up neurologic exams with the the investigators or their own neurologists as outlined in the protocol.
3. Patients or their designated responsible party for medical decisions must be capable of providing informed consent. Cognitive or memory impairment does not necessarily mean the patient is incapable of giving informed consent. They may simply need more time to process or repetition of the content of the consent to reach understanding and provide informed consent.
4. In the estimation of the investigator the BMSC collection and treatment will not present a significant risk of harm to the patient's general health or to their neurologic function.
5. Patients who are not medically stable or who may be at significant risk to their health undergoing the procedure will not be eligible.
6. Women of childbearing age must not be pregnant at the time of treatment and should refrain from becoming pregnant for 3 months post treatment.

Primary outcomes:
measure: Mini-Mental Status Exam (MMSE)
timeFrame: 1,3,6 and 12 months post treatment.
measure: Autism Spectrum Quotient Exam
timeFrame: 1,3,6 and 12 months post treatment.
Secondary outcomes:
measure: Activities of Daily Living
timeFrame: 1,3,6,and 12 months post treatment
Other outcomes:
Locations:
city: Westport
country: United States
facility: MD Stem Cells
state: Connecticut
city: Coral Springs
country: United States
facility: MD Stem Cells
state: Florida
city: Dubai
country: United Arab Emirates
facility: Medcare Orthopaedics & Spine Hospital
Study officials:
affiliation: MD Stem Cells
name: Steven Levy, MD
role: STUDY_CHAIR
affiliation: Coral Springs
name: Jeffrey Weiss, MD
role: PRINCIPAL_INVESTIGATOR
References:
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citation: Shroff G. Human Embryonic Stem Cells in the Treatment of Autism: A Case Series. Innov Clin Neurosci. 2017 Apr 1;14(3-4):12-16. eCollection 2017 Mar-Apr.
citation: Chez M, Lepage C, Parise C, Dang-Chu A, Hankins A, Carroll M. Safety and Observations from a Placebo-Controlled, Crossover Study to Assess Use of Autologous Umbilical Cord Blood Stem Cells to Improve Symptoms in Children with Autism. Stem Cells Transl Med. 2018 Apr;7(4):333-341. doi: 10.1002/sctm.17-0042. Epub 2018 Feb 6.
citation: Weiss JN, Levy S. Neurologic Stem Cell Treatment Study (NEST) using bone marrow derived stem cells for the treatment of neurological disorders and injuries: study protocol for a nonrandomized efficacy trial. Clin Trials Degener Dis. 2016 [cited 2019 Jun 18];1:176-80.