Alzheimer's Disease Stem Cells Multiple Infusions
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|ClinicalTrials.gov Identifier: NCT04040348|
Recruitment Status : Recruiting
First Posted : July 31, 2019
Last Update Posted : May 28, 2020
|Condition or disease||Intervention/treatment||Phase|
|Alzheimer Disease||Biological: 100 million cells allogeneic hMSC||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I, Prospective, Open-label Trial to Evaluate the Safety, Tolerability and Exploratory Outcomes of Multiple Allogeneic Human Mesenchymal Stem Cells (HMSC) Infusions in Patients With Mild to Moderate Alzheimer's Disease|
|Actual Study Start Date :||October 8, 2019|
|Estimated Primary Completion Date :||September 1, 2021|
|Estimated Study Completion Date :||September 1, 2021|
Experimental: hMSC Treatment group
Participants in the hMSC treatment group will receive a total of 4 doses of the hMSC intervention. Each dose will be administered once about every 13 weeks within a year period.
Biological: 100 million cells allogeneic hMSC
Umbilical cord-derived, allogeneic hMSC administered intravenously at a dose of 100 million cells per infusion.
- Number of Incidence of any Treatment-Emergent Serious Adverse Events (TE-SAEs) [ Time Frame: One month post-infusion ]
All adverse events will be evaluated by the investigator for relationship with the study intervention. Treatment-Emergent Serious Adverse Events is defined as any untoward medical occurrence with a reasonable possibility that it is caused by the study intervention that:
- Is life-threatening (e.g.; leads to stroke or non-fatal pulmonary embolism);
- Requires inpatient hospitalization or prolongation of existing hospitalization;
- Results in persistent or significant disability/incapacity
- Results in other clinically significant sign(s) or symptom(s), (e.g.; clinically asymptomatic brain microhemorrhages); or
- Results in death
- Cognitive function over time as assessed by the Alzheimer's Disease Assessment Scale - Cognitive (ADAS-Cog 11) [ Time Frame: Up to Week 65 ]The ADAS-Cog 11 is a 13-item version of ADAS-Cog comprising of the original 11-item ADAS-Cog as well as Delayed Recall and Digit Cancellation items. The total score ranges from 0-85 points, with a lower score indicating better performance.
- Cognitive function over time as assessed by the Mini Mental State Examination (MMSE) of Folstein test [ Time Frame: Up to Week 65 ]The MMSE assesses orientation to time and place, immediate and delayed recall of words, attention and calculation, language (naming, comprehension and repetition), and spatial ability (copying a figure). The total score ranges from 0-30, with a higher score indicating better cognitive performance.
- Depressive symptoms over time as assessed by the Geriatric Depression Scale (GDS) Short Version [ Time Frame: Up to Week 65 ]The GDS is a 15-item questionnaire with each item counting as one point. The total score ranges from 0 to 15 with a score greater than 5 indicating possible depression.
- Olfactory function over time as assessed by the Odor Identification test. [ Time Frame: Up to Week 65 ]Olfactory function is assessed via a self-administered 16-item odor identification test that provides an indication of anosmia (mild, moderate, or severe), and an index to detect malingering. Total scores range from 0-16 with the higher score indicating higher smell function.
- Participant quality of life over time assessed via Alzheimer's Disease Related Quality of Life (ADRQL-40) Questionnaire as completed by the caregiver [ Time Frame: Up to Week 65 ]ADRQL-40 is a questionnaire completed by the caregiver assessing the quality of life of the participant with AD. The total score for the ADRQL is computed by summing the values assigned to the responses, dividing the sum by the maximum value for the scale and multiplying the results by 100 to obtain a percentage score of 0 to 100. A higher score reflects a higher quality of life.
- Participant quality of life over time as assessed via the Alzheimer's Disease Cooperative Study Activities of Daily Living (ADCS-ADL) Questionnaire as completed by the caregiver [ Time Frame: Up to Week 65 ]The ADCS-ADL is a 23 item questionnaire completed by the caregiver assessing the basic and instrumental activities of daily living by the AD participant. Total score range from 0-78 with the higher score indicating increased independence.
- Neuropsychiatric Inventory-Q (NPI-Q) Scores over time [ Time Frame: Up to Week 52 ]The NPI-Q is a questionnaire used to assess behavioral changes common in dementia patients. This questionnaire is completed by the caregiver. The questionnaire consists of 12 items with each item having a scoring range between 0-3. The higher score indicates a more severe neuropsychiatric symptomatology.
- Caregiver's Quality of life over time as assessed by the Caregiver Self-Assessment Questionnaire scores [ Time Frame: Up to Week 52 ]The Caregiver Self-Assessment questionnaire is completed by the caregiver. It is an 18-item questionnaire answered with a "yes" or "no". Evidence of distress is indicated for having over 10 "yes" answers.
- Biomarker levels over time [ Time Frame: Up to Week 65 ]Serum blood inflammatory and biomarker levels will be evaluated including Interleukin-6 (IL-6), Neurofilament light (NfL), Amyloid Beta 40 (Aβ40) and Amyloid Beta 42 (Aβ42) in pg/mL.
- Serum ApoE level over time [ Time Frame: Up to Week 65 ]Serum blood Apolipoprotein E (ApoE) will be evaluated in mg/dL.
- Serum PRA level over time [ Time Frame: Up to Week 65 ]Serum blood Plasma Renin Activity (PRA) will be evaluated in ng/mL per hour.
- Serum Tau protein level over time [ Time Frame: Up to Week 65 ]Serum blood Tau protein level will be evaluated in ng/L.
- Cerebrospinal Fluid (CSF) Biomarker levels over time [ Time Frame: Up to Week 52 ]CSF inflammatory and biomarker levels will be evaluated including Interleukin-6 (IL-6), Neurofilament light (NfL), Amyloid Beta 40 (Aβ40) and Amyloid Beta 42 (Aβ42) in pg/mL.
- CSF ApoE level over time [ Time Frame: Up to Week 52 ]CSF Apolipoprotein E (ApoE) levels will be evaluated in mg/dL.
- CSF PRA level over time [ Time Frame: Up to Week 52 ]CSF Plasma Renin Activity (PRA) levels will be evaluated in ng/mL per hour.
- CSF Tau protein level over time [ Time Frame: Up to Week 52 ]CSF Tau protein levels will be evaluated in ng/L.
- Change in hippocampal volume [ Time Frame: Baseline to Week 6, Baseline to Week 52 ]Change in hippocampal volume will be assessed via MRI Brain volumetric studies
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04040348
|Contact: Carmen Perezfirstname.lastname@example.org|
|United States, Florida|
|University of Miami||Recruiting|
|Miami, Florida, United States, 33136|
|Contact: Carmen Perez 305-243-0184 email@example.com|
|Principal Investigator: Bernard (Barry) Baumel, MD|
|Principal Investigator:||Bernard (Barry) Baumel, MD||University of Miami|