Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study (ACIST)
Primary Purpose
Alzheimer Disease, Alzheimer Dementia, Vascular Dementia
Status
Enrolling by invitation
Phase
Not Applicable
Locations
International
Study Type
Interventional
Intervention
Intravenous Bone Marrow Stem Cell (BMSC) Fraction
Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction
Near Infrared Light
Sponsored by
About this trial
This is an interventional treatment trial for Alzheimer Disease focused on measuring 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
Eligibility Criteria
Inclusion Criteria:
- Have documented cognitive impairment or diagnosis of disease associated with cognitive impairment such as Alzheimer's Disease, Autism Spectrum Disorder.
- 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.
- 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.
- Be over the age of 18
- 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:
- All patients must be capable of an adequate neurologic examination and evaluation to document the pathology.
- 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.
- 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.
- 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.
- Patients who are not medically stable or who may be at significant risk to their health undergoing the procedure will not be eligible.
- Women of childbearing age must not be pregnant at the time of treatment and should refrain from becoming pregnant for 3 months post treatment.
Sites / Locations
- MD Stem Cells
- MD Stem Cells
- Medcare Orthopaedics & Spine Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Active Comparator
Active Comparator
Active Comparator
Arm Label
Arm 1
Arm 2
Arm 3
Arm Description
Intravenous Bone Marrow Stem Cell (BMSC) Fraction
Intravenous Bone Marrow Stem Cell (BMSC) Fraction combined with Near Infrared Light exposure .
Intravenous Bone Marrow Stem Cell (BMSC) Fraction combined with Intranasal topical Bone Marrow Stem Cell (BMSC) Fraction.
Outcomes
Primary Outcome Measures
Mini-Mental Status Exam (MMSE)
In cognitive impairment patients this standard cognitive function test will be administered. The change from pretreatment baseline to each time point post treatment will be assessed.
Autism Spectrum Quotient Exam
Patients with Autism Spectrum Disorder (ASD) or Autism will undergo testing with the Autism Spectrum Quotient (AQ) Exam, an autism assessment for adults. The change in scoring from pretreatment baseline to each time point post treatment will be assessed.
Secondary Outcome Measures
Activities of Daily Living
The patients, friends and family will describe changes in abilities to function on a day to day basis.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03724136
Brief Title
Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study
Acronym
ACIST
Official Title
Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
October 24, 2018 (Actual)
Primary Completion Date
October 2024 (Anticipated)
Study Completion Date
October 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MD Stem Cells
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Model Description
Patients will be assigned to one of 3 arms with monitoring and retesting at 1,3,6 and 12 months following treatment.
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
100 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Arm 1
Arm Type
Active Comparator
Arm Description
Intravenous Bone Marrow Stem Cell (BMSC) Fraction
Arm Title
Arm 2
Arm Type
Active Comparator
Arm Description
Intravenous Bone Marrow Stem Cell (BMSC) Fraction combined with Near Infrared Light exposure .
Arm Title
Arm 3
Arm Type
Active Comparator
Arm Description
Intravenous Bone Marrow Stem Cell (BMSC) Fraction combined with Intranasal topical Bone Marrow Stem Cell (BMSC) Fraction.
Intervention Type
Procedure
Intervention Name(s)
Intravenous Bone Marrow Stem Cell (BMSC) Fraction
Intervention Description
14 cc of BMSC fraction separated from bone marrow aspirate and filtered with 150 micron filter and administered intravenously.
Intervention Type
Procedure
Intervention Name(s)
Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction
Intervention Description
Approximately 1 cc of BMSC fraction separated from bone marrow aspirate and administered to the nasal mucosa topically.
Intervention Type
Procedure
Intervention Name(s)
Near Infrared Light
Intervention Description
Near Infrared Light will be administered using an FDA cleared medical device on the preoperative day and the first postoperative day as tolerated to the general area of the frontal bone.
Primary Outcome Measure Information:
Title
Mini-Mental Status Exam (MMSE)
Description
In cognitive impairment patients this standard cognitive function test will be administered. The change from pretreatment baseline to each time point post treatment will be assessed.
Time Frame
1,3,6 and 12 months post treatment.
Title
Autism Spectrum Quotient Exam
Description
Patients with Autism Spectrum Disorder (ASD) or Autism will undergo testing with the Autism Spectrum Quotient (AQ) Exam, an autism assessment for adults. The change in scoring from pretreatment baseline to each time point post treatment will be assessed.
Time Frame
1,3,6 and 12 months post treatment.
Secondary Outcome Measure Information:
Title
Activities of Daily Living
Description
The patients, friends and family will describe changes in abilities to function on a day to day basis.
Time Frame
1,3,6,and 12 months post treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Have documented cognitive impairment or diagnosis of disease associated with cognitive impairment such as Alzheimer's Disease, Autism Spectrum Disorder.
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.
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.
Be over the age of 18
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:
All patients must be capable of an adequate neurologic examination and evaluation to document the pathology.
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.
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.
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.
Patients who are not medically stable or who may be at significant risk to their health undergoing the procedure will not be eligible.
Women of childbearing age must not be pregnant at the time of treatment and should refrain from becoming pregnant for 3 months post treatment.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Steven Levy, MD
Organizational Affiliation
MD Stem Cells
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Jeffrey Weiss, MD
Organizational Affiliation
Coral Springs
Official's Role
Principal Investigator
Facility Information:
Facility Name
MD Stem Cells
City
Westport
State/Province
Connecticut
ZIP/Postal Code
06880
Country
United States
Facility Name
MD Stem Cells
City
Coral Springs
State/Province
Florida
ZIP/Postal Code
33065
Country
United States
Facility Name
Medcare Orthopaedics & Spine Hospital
City
Dubai
Country
United Arab Emirates
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
19324456
Citation
Danielyan L, Schafer R, von Ameln-Mayerhofer A, Buadze M, Geisler J, Klopfer T, Burkhardt U, Proksch B, Verleysdonk S, Ayturan M, Buniatian GH, Gleiter CH, Frey WH 2nd. Intranasal delivery of cells to the brain. Eur J Cell Biol. 2009 Jun;88(6):315-24. doi: 10.1016/j.ejcb.2009.02.001. Epub 2009 Mar 25.
Results Reference
background
PubMed Identifier
28494803
Citation
Duncan T, Valenzuela M. Alzheimer's disease, dementia, and stem cell therapy. Stem Cell Res Ther. 2017 May 12;8(1):111. doi: 10.1186/s13287-017-0567-5.
Results Reference
background
PubMed Identifier
26793049
Citation
Johnstone DM, Moro C, Stone J, Benabid AL, Mitrofanis J. Turning On Lights to Stop Neurodegeneration: The Potential of Near Infrared Light Therapy in Alzheimer's and Parkinson's Disease. Front Neurosci. 2016 Jan 11;9:500. doi: 10.3389/fnins.2015.00500. eCollection 2015.
Results Reference
background
PubMed Identifier
28805182
Citation
Robbins JP, Price J. Human induced pluripotent stem cells as a research tool in Alzheimer's disease. Psychol Med. 2017 Nov;47(15):2587-2592. doi: 10.1017/S0033291717002124. Epub 2017 Aug 14.
Results Reference
background
PubMed Identifier
29185257
Citation
Park SE, Lee NK, Na DL, Chang JW. Optimal mesenchymal stem cell delivery routes to enhance neurogenesis for the treatment of Alzheimer's disease: optimal MSCs delivery routes for the treatment of AD. Histol Histopathol. 2018 Jun;33(6):533-541. doi: 10.14670/HH-11-950. Epub 2017 Nov 29.
Results Reference
background
PubMed Identifier
28584692
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.
Results Reference
background
PubMed Identifier
29405603
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.
Results Reference
background
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.
Results Reference
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Links:
URL
https://www.cnn.com/2017/04/05/health/autism-cord-blood-stem-cells-duke-study/index.html
Description
Report from CNN on Duke University stem cell autism study
Learn more about this trial
Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study
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