DementiaNDDNCT03297177
Autologous Stem/Stromal Cells in Neurological Disorders and Disease
A study testing Microcannula Harvest Adipose, Centricyte 1000 and Sterile Normal Saline Infusion in people with Dementia and Parkinson, run by Regeneris Medical at 2 sites in United States.
Trial summary
The study deals with evaluation of safety and efficacy of use of stem/stromal cell isolates from autologous microvasculature in neurological, non-neoplastic disease. Autologous cells are acquired via microcannula aspiration of subdermal fat deposits, isolated through a digestive process, and concentrated via standard… Read more about this study
- Who is it for?
- Ages 18 to 90 years
- Conditions the study is for
- Dementia, Parkinson, Altered Behavior in Alzheimer Disease
- What is being tested?
- Microcannula Harvest Adipose, Centricyte 1000 and Sterile Normal Saline Infusion
- Type of treatment
- Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 300 participants
- Where
- 2 sitesUnited States
- Study dates
- 1 Jan 2020to 1 Jan 2023
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Documented Functional Neurological Damage To Central or Peripheral Nervous System Unlikely To Improve With Present Standard of Care Approaches
- At least 6 months after onset or diagnosis of disease process
- Current Medical therapy for the condition is either failing or not tolerated by patient
- Patient must be capable of interval neurologic exams with investigators or their own neurologic specialists
- Patient must be capable and determined competent to provide detailed informed consent for study participation
- In estimation of investigators, that there are minimal or no significant risk of harm to general health or conditions for collection of autologous stem cell collection and use
Who cannot join
- Inability of patient to have diagnostic examinations or studies (MRI) to evaluate and document the disease state or unwilling/unable to cooperate with such documentation
- Patients not medically stable, or whom may have ongoing conditions which increases may place the patient at significant risk of major complications, to be determined by investigator or patient's medical provider or neurologic specialists
- History of active cancer or ongoing anticancer therapy within six months of such care
- Women of childbearing age must not be pregnant at the time of treatment, and should refrain from becoming pregnant of at minimum of 3 month after study treatment
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Regeneris Medical Inc
North Attleboro, Massachusetts, 02760
Ryan Welter, MD, PhD, 508-345-5492, r.welter@regenerismedical.com
Kristin Corrado, 508.316.4268, k.corrado@regenerismedical.com
Regeneris Medical
North Attleboro, Massachusetts, 02760
Ryan Welter, MD, PhD, 508-316-4268, k.corrado@regenerismedical.com
Kristin Corrado, 508.316.4268, k.corrado@regenerismedical.com
About this study
Official title: Use Of Autologous Stem Cell Use In Neurological Non-neoplastic Disorders And Disease
The study deals with evaluation of safety and efficacy of use of stem/stromal cell isolates from autologous microvasculature in neurological, non-neoplastic disease. Autologous cells are acquired via microcannula aspiration of subdermal fat deposits, isolated through a digestive process, and concentrated via standard centrifugation. The cellular stromal vascular fraction (cSVF) created is neutralized and rinsed to eliminate residual enzymatic molecules.
These cells are suspending in sterile Normal Saline Solution (500cc) and re-administered via an intravenous parenteral route, passed through a standard sterile 150 u (micron) filter in line.
Multiple tracking and questionnaire followup is intended over a 5 year period, with objective and subjective criteria being met. Compilation and analysis of data to be completed after that period.
Read the detailed description
Dementia refers to neurodegenerative conditions causing gradual decrease in ability to think and recall, to the point that is impacts daily functions. It is often accompanied by language difficulties, wide ranging emotional issues, loss of language skills, and lowering of activity motivation. These changes are often in the face of unchanged consciousness.
Most common type is Alzheimer's Disease (50-70% of cas.es), but include vascular dementia (25%), Lewy Body dementia (15%) and frontotemporal dementia. There are many interrelated disorders that mask the underlying issues, such as in Parkinson's Disease, and whose diagnosis may overlap with the others.
Most common symptoms include memory lapses, visual-spatial language changes, attention loss and problem solving difficulties. A wide variety of other symptoms may be seen, such as balance issues, tremors, difficulty swallowing, hallucinations, wandering, restlessness or agitation/depression, mood swings, etc..
Parkinson's Disease (PD) is a long-term neurodegenerative disorder of the Central Nervous System (CNS) that usually impacts the motor system, in a progressive and long-term fashion. Early signs include tremors, rigidity, slow movements and walking issues. It is often associated with thinking problems and depression, plus emotional states that impact daily living.
The causation of PD is felt to involve genetic predisposition and environmental factors. History of head injuries, chemical exposures, smokers, caffeine intake and other dietary issues. The motor symptoms seem to result from cellular death in the midbrain area (particularly the substantia nigra) and associated with inadequate dopamine. Plaques (protein build up following cellular death) may be a prominent feature, and use of neuroimaging (like MRI with/without contrast) is of value to track progressive changes in the brain.
Conventional therapy is use of L-DOPA and dopamine agonists and pharmaceutical agents have not proved of long-term reversal or arrest of the process. Evaluation of non-traditional methods are currently gaining popularity, especially in the area of stem/stromal cellular therapy.
CNS Autoimmune Demyelination is group of disorders of the CNS which feature loss of myelin from unknown causes but include variants of encephalomyelitis, cerebral sclerosis of Schilder, multiple sclerosis, and idiopathic inflammatory demyelination.
In cases involving myelin sheath of neurons is damaged, reducing the conduction of nerve signals causing loss of sensory, motor, cognitive abilities, and a wide variety of functions dependent on actual location of changes.
There is strong evidence that autoimmune, infectious agents, and chemical reactions may contribute to the evolution of the disease complex. In cases of multiple sclerosis, it appears that T-cells and macrophages, and mast cells may be present in the CNS plaques that are characteristically seen in areas of the ventricles and scattered areas of the brain. These are typically identified and tracked by MRI studies.
Traditional pharmaceutical approaches have proven to be of little advantage in reversing or completely stopping the disease processes. Many side effects and reactions, or loss of drug effectiveness have led researchers to explore use of stem/stromal cells with their effects on immunomodulation and inflammatory modulation effects. Early trials suggest that these may be able to actually reverse visible lesions and improve quality of function and life, with reported improvement in neurological conditions.
Management of symptoms is often accompanied by lifestyle changes, rest periods, physical and mental rehabilitations, diet, chelation and hyper-vitamin infusions, and a number of antioxidant therapies. The progressive phases seem to include the innate immune system, and is felt to explain the potential value of use of autologous stem/stromal cellular groups, particularly involving the mesenchymal and pericytic/endothelial cellular groups.
Sensorimotor demyelination neuropathies (HMSN) is group of neuropathies characterized by impact on afferent and efferent neural communications. They feature atypical neural development and degradation of nerve tissues.
Two common forms of HMSN are either hypertrophic demyelinated nerves or atrophy of nerve (peripheral) and neural tissues. Hypertrophy is featured by neural stiffness rather than demyelination of peripheral nerves. Whereas atrophy causes loss of axons and neural cell bodies. They often experience progressive muscle atrophy and sensory neuropathy of the extremities. The term "hereditary motor and sensory neuropathy has been commonly referred to a forms of Charcot-Marie-Tooth Disease (CMT). There are distinct genetic and phenotypically distinct forms of CMT, and the term of HMSN is less frequently used.
Usual onset is childhood or young adulthood, with motor involvement more prominent that sensory. Often mistaken for causalgia or fibromyalgia, the symptoms of fatigue, pain, balance loss, areflexia, visual changes, foot drop and scoliosis. As in the causalgia or Sympathetic Reflex Dystrophy (SRD), these patients are treated according to symptoms, with medication and traditional interventions proving of limited benefit. Some preliminary findings using the modulatory action of stem/stromal cells suggest some help with the symptomatology, particularly relative fatigue and pain.
Huntington's Disease is an inherited disorder resulting in death of brain cells. A gradual change in mood or mental abilities, is followed by unsteady gait, jerky body movements, leading to speech loss or severe depression. Although associated with heredity or mutation, pharmaceuticals and stem/stromal cell therapy are felt to offer some assistance in maintaining quality of life (Qol).
The huntington protein (HP) is poorly understood, but believe toxic to certain cell types, particularly in the brain. Damage is most noticed in the striatum of the brain. The changes associated with the HP relate to the cell transcription, cell signaling and intracellular transport.
Most prominent changes are within the basal ganglia (neostriatum) and includes the caudate nucleus and putamen. Substantia nigra and cerebellum seem particularly impacted. Physical therapy, occupational therapy and speech therapy have been used in conjunction with trials using autologous stem/stromal cellular therapies are offering some slowing of the processes.
Medications have been developed, but remain modestly effective and possessing major side effects and poorly tolerated. Most of the pharmaceutical approaches have not proven effective in reversal of the disease, many accepting a slowing or remission induction without inducing the body ability to provide any regenerative effects. Dietary controls, use of mind exercising, hyperoxygenation protocols, chelation, vitamin infusions, etc. have proven to be helpful adjunctive therapies. Alternative treatments, including physical therapy and some stem/stromal therapies have become more common.
Three main characteristics of the neurodegenerative group are: 1). Lesion formations in the central nervous system (called Plaques); 2). Inflammation; 3). Demyelination and Destruction of myelin sheaths of neurons. This demyelination is thought to stimulate the inflammatory processes due to action of a lymphocyte group known at T-cell which seems to recognize patient's own myelin as foreign and proceeds to attack it (known as "autoreactive lymphocytes"). These findings are often concluded to represent an autoimmune response to one's own tissues. The inherent properties of modulation and inflammatory modulation make potential uses of autologous reparative group of cells located throughout the body in association with microcapillary structures in essentially every tissue in the body. These cells have been identified by cell surface markers and studied in tissue culture to have "multipotent" capabilities that potentially participate in the regenerative processes to help arrest or improve the autoimmune group of disorders.
Traditionally, exacerbation's are often treated with high dose intravenous steroids which may be of short term reduction of symptoms, not addressing the underlying causation. Current medications available for treatment are expensive an fraught with major side effects, making their use very difficult and producing limited measured value.
With the advent of convenient ability to acquire significant numbers of alive (viable) an easily gathered by harvesting from microvascular stores in the body (adipose tissue complex). Technological advances in sterile, closed processing, has provided the ability to easily and safely acquire significant of stem/stromal cells for use in clinical studies.
This study is aimed at evaluation of the safety profile (adverse reactions \& severe adverse reactions) of the closed syringe, microcannula harvesting of subdermal fat deposits (largest known source of microvascular tissues in the body). This autologous cell group obtained with isolation and concentration of cells within the stromal vascular fraction (SVF) via enzymatic digestion, and deployed via intravascular r rebral fluids in defects of the blood brain barrier (BBB) or are small enough to pass into the fluids of the CNS (central nervous system). Effects are believed to be a function of both cellular multipotent abilities, but also regards the autocrine/paracrine secretory capacity of the cell group within the body. The body has the ability to request (chemotaxis) certain needed cellular and secreted chemicals to help healing and regeneration. Current thought are trending that the secretions may, in fact, be more important to the reparative and regenerative processes than the cellular contributions.
In this study, disorders which involve autoimmune and inflammatory changes that impact the brain and nervous system are to be study, tracked and reported over time to determine ultimate efficacy and the "interval" therapy needs (or lack thereof) to maintain the accomplished arrest or improvement in the underlying tissues. Currently these intervals are examined by brain magnetic resonance imaging (MRI) with and without contrast. Correlation of image changes and clinical function will be made with subjective Quality of Life (Qol) and diary type observations by patients and caregivers.
- Conditions
- Dementia, Parkinson, Altered Behavior in Alzheimer Disease, Demyelinating Autoimmune Diseases, CNS, Demyelinating Sensorimotor Neuropathy, Corticobasal Degeneration
- Being tested
- Microcannula Harvest Adipose, Centricyte 1000, Sterile Normal Saline Infusion
- Medical subject headings
- Dementia, Demyelinating Autoimmune Diseases, CNS, Corticobasal Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Microcannula Harvest AdiposeProcedure or surgery
Use Closed Syringe Microcannula Harvest Adipose, Autologous Subdermal Deposits
Centricyte 1000Device
Closed System, Digestive Isolation \& Concentration of AD-cSVF
Sterile Normal Saline InfusionProcedure or surgery
Sterile Normal Saline Re-Suspension of cSVF and Delivery of AD-cSVF via IV route
| Study group | Role | What they receive |
|---|---|---|
| Microcannula Harvest Adipose | Experimental | Acquisition AD-tSVF Via Closed Syringe Microcannula Receives: Procedure: Microcannula Harvest Adipose |
| Centricyte 1000 | Experimental | Autologous Adipose-Derived Tissue Stromal Vascular Fraction (tSVF) via enzymatic digestive isolation \& concentration in Centricyte 1000 closed system to create AD-cSVF Receives: Device: Centricyte 1000 |
| Sterile Normal Saline Infusion | Experimental | Sterile Normal Saline to Re-Suspend Autologous cSVF pellet for delivery via intravascular (IV) route Receives: Procedure: Sterile Normal Saline Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participant with adverse events (AE) or severe adverse events (SAE)
Activities of Daily Living (ADL)
Measured: 6 month intervals for up to 5 years
Secondary outcomes (3)
Neurological Function
Change from baseline of deficits of neurologic function identified by patient as impaired prior to study
Measured: 6 month Intervals for up to 5 years
Neurological Tested Functions
Change from baseline of deficits of neurological function identified by medical provider as impaired prior to study
Measured: 6 month interval for up to 5 years
Magnetic Resonance Imaging (MRI)
Change in baseline of MRI with and without contrast at 6 month intervals
Measured: 6 month intervals for up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2017
- Study start1 Jan 2020 (estimated)
- Primary completion1 Jan 2023 (estimated)
- Study completion1 Jan 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (9)Papers the sponsor has linked to this study.
- Other trials for DementiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Regeneris Medical
- Sponsor type
- University, hospital or other
- Collaborators
- Global Alliance for Regenerative Medicine, Alexander, Robert W., M.D., Regeneris Medical Inc
- Responsible party
- Principal investigator, Ryan Welter, MD, PhD, Healeon Medical Inc
- Investigators
- Ryan Welter, MD, PhD, Principal investigator, Regeneris Medical IncGlenn C Terry, Principal investigator, Global Alliance for Regenerative Medicine (GARM)Robert W Alexander, MD, Principal investigator, Healeon Medical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03297177
- Sponsor's protocol ID
- GARM NDD
- Acronym
- NDD
- Status last verified
- Oct 2018
- First submitted
- 26 Sep 2017
- Last updated
- 18 Oct 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- Yes
- Individual data sharing
- Undecided: Annual Summary of Enrolled Cases By All collaborators, principle investigators, or accept sub-investigators
Publications
Litvan I, Agid Y, Goetz C, Jankovic J, Wenning GK, Brandel JP, Lai EC, Verny M, Ray-Chaudhuri K, McKee A, Jellinger K, Pearce RK, Bartko JJ. Accuracy of the clinical diagnosis of corticobasal degeneration: a clinicopathologic study. Neurology. 1997 Jan;48(1):119-25. doi: 10.1212/wnl.48.1.119.
PubMed 9008506Rizzo G, Martinelli P, Manners D, Scaglione C, Tonon C, Cortelli P, Malucelli E, Capellari S, Testa C, Parchi P, Montagna P, Barbiroli B, Lodi R. Diffusion-weighted brain imaging study of patients with clinical diagnosis of corticobasal degeneration, progressive supranuclear palsy and Parkinson's disease. Brain. 2008 Oct;131(Pt 10):2690-700. doi: 10.1093/brain/awn195. Epub 2008 Sep 26.
PubMed 18819991Alladi S, Xuereb J, Bak T, Nestor P, Knibb J, Patterson K, Hodges JR. Focal cortical presentations of Alzheimer's disease. Brain. 2007 Oct;130(Pt 10):2636-45. doi: 10.1093/brain/awm213.
PubMed 17898010Sha SJ, Ghosh PM, Lee SE, Corbetta-Rastelli C, Jagust WJ, Kornak J, Rankin KP, Grinberg LT, Vinters HV, Mendez MF, Dickson DW, Seeley WW, Gorno-Tempini M, Kramer J, Miller BL, Boxer AL, Rabinovici GD. Predicting amyloid status in corticobasal syndrome using modified clinical criteria, magnetic resonance imaging and fluorodeoxyglucose positron emission tomography. Alzheimers Res Ther. 2015 Mar 2;7(1):8. doi: 10.1186/s13195-014-0093-y. eCollection 2015.
PubMed 25733984Jankovic J. Parkinson's disease: clinical features and diagnosis. J Neurol Neurosurg Psychiatry. 2008 Apr;79(4):368-76. doi: 10.1136/jnnp.2007.131045.
PubMed 18344392Gibb WR, Lees AJ. The relevance of the Lewy body to the pathogenesis of idiopathic Parkinson's disease. J Neurol Neurosurg Psychiatry. 1988 Jun;51(6):745-52. doi: 10.1136/jnnp.51.6.745.
PubMed 2841426Pedrosa DJ, Timmermann L. Review: management of Parkinson's disease. Neuropsychiatr Dis Treat. 2013;9:321-40. doi: 10.2147/NDT.S32302. Epub 2013 Mar 3.
PubMed 23487540Mayo L, Quintana FJ, Weiner HL. The innate immune system in demyelinating disease. Immunol Rev. 2012 Jul;248(1):170-87. doi: 10.1111/j.1600-065X.2012.01135.x.
PubMed 22725961Ransohoff RM, Howe CL, Rodriguez M. Growth factor treatment of demyelinating disease: at last, a leap into the light. Trends Immunol. 2002 Nov;23(11):512-6. doi: 10.1016/s1471-4906(02)02321-9.
PubMed 12401395
Study at a glance
From the registry record, last updated by the sponsor on 18 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use Of Autologous Stem Cell Use In Neurological Non-neoplastic Disorders And Disease
- ClinicalTrials.gov ID
- NCT03297177
- Acronym / sponsor ID
- NDD / GARM NDD
- Conditions
- Dementia, Parkinson, Altered Behavior in Alzheimer Disease, Demyelinating Autoimmune Diseases, CNS, Demyelinating Sensorimotor Neuropathy, Corticobasal Degeneration
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- Regeneris MedicalUniversity, hospital or other
- Collaborators
- Global Alliance for Regenerative Medicine, Alexander, Robert W., M.D., Regeneris Medical Inc
- Study start
- 1 Jan 2020 (estimated)
- Primary completion
- 1 Jan 2023 (estimated)
- Study completion
- 1 Jan 2023 (estimated)
- First posted
- 29 Sep 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study deals with evaluation of safety and efficacy of use of stem/stromal cell isolates from autologous microvasculature in neurological, non-neoplastic disease. Autologous cells are acquired via microcannula aspiration of subdermal fat deposits, isolated through a digestive process, and concentrated via standard centrifugation. The cellular stromal vascular fraction (cSVF) created is neutralized and rinsed to eliminate residual enzymatic molecules.
These cells are suspending in sterile Normal Saline Solution (500cc) and re-administered via an intravenous parenteral route, passed through a standard sterile 150 u (micron) filter in line.
Multiple tracking and questionnaire followup is intended over a 5 year period, with objective and subjective criteria being met. Compilation and analysis of data to be completed after that period.
Detailed description
Dementia refers to neurodegenerative conditions causing gradual decrease in ability to think and recall, to the point that is impacts daily functions. It is often accompanied by language difficulties, wide ranging emotional issues, loss of language skills, and lowering of activity motivation. These changes are often in the face of unchanged consciousness.
Most common type is Alzheimer's Disease (50-70% of cas.es), but include vascular dementia (25%), Lewy Body dementia (15%) and frontotemporal dementia. There are many interrelated disorders that mask the underlying issues, such as in Parkinson's Disease, and whose diagnosis may overlap with the others.
Most common symptoms include memory lapses, visual-spatial language changes, attention loss and problem solving difficulties. A wide variety of other symptoms may be seen, such as balance issues, tremors, difficulty swallowing, hallucinations, wandering, restlessness or agitation/depression, mood swings, etc..
Parkinson's Disease (PD) is a long-term neurodegenerative disorder of the Central Nervous System (CNS) that usually impacts the motor system, in a progressive and long-term fashion. Early signs include tremors, rigidity, slow movements and walking issues. It is often associated with thinking problems and depression, plus emotional states that impact daily living.
The causation of PD is felt to involve genetic predisposition and environmental factors. History of head injuries, chemical exposures, smokers, caffeine intake and other dietary issues. The motor symptoms seem to result from cellular death in the midbrain area (particularly the substantia nigra) and associated with inadequate dopamine. Plaques (protein build up following cellular death) may be a prominent feature, and use of neuroimaging (like MRI with/without contrast) is of value to track progressive changes in the brain.
Conventional therapy is use of L-DOPA and dopamine agonists and pharmaceutical agents have not proved of long-term reversal or arrest of the process. Evaluation of non-traditional methods are currently gaining popularity, especially in the area of stem/stromal cellular therapy.
CNS Autoimmune Demyelination is group of disorders of the CNS which feature loss of myelin from unknown causes but include variants of encephalomyelitis, cerebral sclerosis of Schilder, multiple sclerosis, and idiopathic inflammatory demyelination.
In cases involving myelin sheath of neurons is damaged, reducing the conduction of nerve signals causing loss of sensory, motor, cognitive abilities, and a wide variety of functions dependent on actual location of changes.
There is strong evidence that autoimmune, infectious agents, and chemical reactions may contribute to the evolution of the disease complex. In cases of multiple sclerosis, it appears that T-cells and macrophages, and mast cells may be present in the CNS plaques that are characteristically seen in areas of the ventricles and scattered areas of the brain. These are typically identified and tracked by MRI studies.
Traditional pharmaceutical approaches have proven to be of little advantage in reversing or completely stopping the disease processes. Many side effects and reactions, or loss of drug effectiveness have led researchers to explore use of stem/stromal cells with their effects on immunomodulation and inflammatory modulation effects. Early trials suggest that these may be able to actually reverse visible lesions and improve quality of function and life, with reported improvement in neurological conditions.
Management of symptoms is often accompanied by lifestyle changes, rest periods, physical and mental rehabilitations, diet, chelation and hyper-vitamin infusions, and a number of antioxidant therapies. The progressive phases seem to include the innate immune system, and is felt to explain the potential value of use of autologous stem/stromal cellular groups, particularly involving the mesenchymal and pericytic/endothelial cellular groups.
Sensorimotor demyelination neuropathies (HMSN) is group of neuropathies characterized by impact on afferent and efferent neural communications. They feature atypical neural development and degradation of nerve tissues.
Two common forms of HMSN are either hypertrophic demyelinated nerves or atrophy of nerve (peripheral) and neural tissues. Hypertrophy is featured by neural stiffness rather than demyelination of peripheral nerves. Whereas atrophy causes loss of axons and neural cell bodies. They often experience progressive muscle atrophy and sensory neuropathy of the extremities. The term "hereditary motor and sensory neuropathy has been commonly referred to a forms of Charcot-Marie-Tooth Disease (CMT). There are distinct genetic and phenotypically distinct forms of CMT, and the term of HMSN is less frequently used.
Usual onset is childhood or young adulthood, with motor involvement more prominent that sensory. Often mistaken for causalgia or fibromyalgia, the symptoms of fatigue, pain, balance loss, areflexia, visual changes, foot drop and scoliosis. As in the causalgia or Sympathetic Reflex Dystrophy (SRD), these patients are treated according to symptoms, with medication and traditional interventions proving of limited benefit. Some preliminary findings using the modulatory action of stem/stromal cells suggest some help with the symptomatology, particularly relative fatigue and pain.
Huntington's Disease is an inherited disorder resulting in death of brain cells. A gradual change in mood or mental abilities, is followed by unsteady gait, jerky body movements, leading to speech loss or severe depression. Although associated with heredity or mutation, pharmaceuticals and stem/stromal cell therapy are felt to offer some assistance in maintaining quality of life (Qol).
The huntington protein (HP) is poorly understood, but believe toxic to certain cell types, particularly in the brain. Damage is most noticed in the striatum of the brain. The changes associated with the HP relate to the cell transcription, cell signaling and intracellular transport.
Most prominent changes are within the basal ganglia (neostriatum) and includes the caudate nucleus and putamen. Substantia nigra and cerebellum seem particularly impacted. Physical therapy, occupational therapy and speech therapy have been used in conjunction with trials using autologous stem/stromal cellular therapies are offering some slowing of the processes.
Medications have been developed, but remain modestly effective and possessing major side effects and poorly tolerated. Most of the pharmaceutical approaches have not proven effective in reversal of the disease, many accepting a slowing or remission induction without inducing the body ability to provide any regenerative effects. Dietary controls, use of mind exercising, hyperoxygenation protocols, chelation, vitamin infusions, etc. have proven to be helpful adjunctive therapies. Alternative treatments, including physical therapy and some stem/stromal therapies have become more common.
Three main characteristics of the neurodegenerative group are: 1). Lesion formations in the central nervous system (called Plaques); 2). Inflammation; 3). Demyelination and Destruction of myelin sheaths of neurons. This demyelination is thought to stimulate the inflammatory processes due to action of a lymphocyte group known at T-cell which seems to recognize patient's own myelin as foreign and proceeds to attack it (known as "autoreactive lymphocytes"). These findings are often concluded to represent an autoimmune response to one's own tissues. The inherent properties of modulation and inflammatory modulation make potential uses of autologous reparative group of cells located throughout the body in association with microcapillary structures in essentially every tissue in the body. These cells have been identified by cell surface markers and studied in tissue culture to have "multipotent" capabilities that potentially participate in the regenerative processes to help arrest or improve the autoimmune group of disorders.
Traditionally, exacerbation's are often treated with high dose intravenous steroids which may be of short term reduction of symptoms, not addressing the underlying causation. Current medications available for treatment are expensive an fraught with major side effects, making their use very difficult and producing limited measured value.
With the advent of convenient ability to acquire significant numbers of alive (viable) an easily gathered by harvesting from microvascular stores in the body (adipose tissue complex). Technological advances in sterile, closed processing, has provided the ability to easily and safely acquire significant of stem/stromal cells for use in clinical studies.
This study is aimed at evaluation of the safety profile (adverse reactions \& severe adverse reactions) of the closed syringe, microcannula harvesting of subdermal fat deposits (largest known source of microvascular tissues in the body). This autologous cell group obtained with isolation and concentration of cells within the stromal vascular fraction (SVF) via enzymatic digestion, and deployed via intravascular r rebral fluids in defects of the blood brain barrier (BBB) or are small enough to pass into the fluids of the CNS (central nervous system). Effects are believed to be a function of both cellular multipotent abilities, but also regards the autocrine/paracrine secretory capacity of the cell group within the body. The body has the ability to request (chemotaxis) certain needed cellular and secreted chemicals to help healing and regeneration. Current thought are trending that the secretions may, in fact, be more important to the reparative and regenerative processes than the cellular contributions.
In this study, disorders which involve autoimmune and inflammatory changes that impact the brain and nervous system are to be study, tracked and reported over time to determine ultimate efficacy and the "interval" therapy needs (or lack thereof) to maintain the accomplished arrest or improvement in the underlying tissues. Currently these intervals are examined by brain magnetic resonance imaging (MRI) with and without contrast. Correlation of image changes and clinical function will be made with subjective Quality of Life (Qol) and diary type observations by patients and caregivers.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Microcannula Harvest Adipose | Experimental | Procedure: Microcannula Harvest AdiposeAcquisition AD-tSVF Via Closed Syringe Microcannula |
| Centricyte 1000 | Experimental | Device: Centricyte 1000Autologous Adipose-Derived Tissue Stromal Vascular Fraction (tSVF) via enzymatic digestive isolation \& concentration in Centricyte 1000 closed system to create AD-cSVF |
| Sterile Normal Saline Infusion | Experimental | Procedure: Sterile Normal Saline InfusionSterile Normal Saline to Re-Suspend Autologous cSVF pellet for delivery via intravascular (IV) route |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Documented Functional Neurological Damage To Central or Peripheral Nervous System Unlikely To Improve With Present Standard of Care Approaches
- At least 6 months after onset or diagnosis of disease process
- Current Medical therapy for the condition is either failing or not tolerated by patient
- Patient must be capable of interval neurologic exams with investigators or their own neurologic specialists
- Patient must be capable and determined competent to provide detailed informed consent for study participation
Key exclusion criteria (4 listed)
- Inability of patient to have diagnostic examinations or studies (MRI) to evaluate and document the disease state or unwilling/unable to cooperate with such documentation
- Patients not medically stable, or whom may have ongoing conditions which increases may place the patient at significant risk of major complications, to be determined by investigator or patient's medical provider or neurologic specialists
- History of active cancer or ongoing anticancer therapy within six months of such care
- Women of childbearing age must not be pregnant at the time of treatment, and should refrain from becoming pregnant of at minimum of 3 month after study treatment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Microcannula Harvest AdiposeUse Closed Syringe Microcannula Harvest Adipose, Autologous Subdermal Deposits | Procedure or surgery | Microcannula Harvest Adipose |
| Centricyte 1000Closed System, Digestive Isolation \& Concentration of AD-cSVF | Device | Centricyte 1000 |
| Sterile Normal Saline InfusionSterile Normal Saline Re-Suspension of cSVF and Delivery of AD-cSVF via IV route | Procedure or surgery | Sterile Normal Saline Infusion |
Primary outcome
Number of participant with adverse events (AE) or severe adverse events (SAE)
Time frame: 6 month intervals for up to 5 years
Activities of Daily Living (ADL)
3 secondary outcomes
Neurological Function
Time frame: 6 month Intervals for up to 5 years
Neurological Tested Functions
Time frame: 6 month interval for up to 5 years
Magnetic Resonance Imaging (MRI)
Time frame: 6 month intervals for up to 5 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2018.
- Background: PubMed 9008506Litvan I, Agid Y, Goetz C, Jankovic J, Wenning GK, Brandel JP, Lai EC, Verny M, Ray-Chaudhuri K, McKee A, Jellinger K, Pearce RK, Bartko JJ. Accuracy of the clinical diagnosis of corticobasal degeneration: a clinicopathologic study. Neurology. 1997 Jan;48(1):119-25. doi: 10.1212/wnl.48.1.119.
- Background: PubMed 18819991Rizzo G, Martinelli P, Manners D, Scaglione C, Tonon C, Cortelli P, Malucelli E, Capellari S, Testa C, Parchi P, Montagna P, Barbiroli B, Lodi R. Diffusion-weighted brain imaging study of patients with clinical diagnosis of corticobasal degeneration, progressive supranuclear palsy and Parkinson's disease. Brain. 2008 Oct;131(Pt 10):2690-700. doi: 10.1093/brain/awn195. Epub 2008 Sep 26.
- Background: PubMed 17898010Alladi S, Xuereb J, Bak T, Nestor P, Knibb J, Patterson K, Hodges JR. Focal cortical presentations of Alzheimer's disease. Brain. 2007 Oct;130(Pt 10):2636-45. doi: 10.1093/brain/awm213.
- Background: PubMed 25733984Sha SJ, Ghosh PM, Lee SE, Corbetta-Rastelli C, Jagust WJ, Kornak J, Rankin KP, Grinberg LT, Vinters HV, Mendez MF, Dickson DW, Seeley WW, Gorno-Tempini M, Kramer J, Miller BL, Boxer AL, Rabinovici GD. Predicting amyloid status in corticobasal syndrome using modified clinical criteria, magnetic resonance imaging and fluorodeoxyglucose positron emission tomography. Alzheimers Res Ther. 2015 Mar 2;7(1):8. doi: 10.1186/s13195-014-0093-y. eCollection 2015.
- Background: PubMed 18344392Jankovic J. Parkinson's disease: clinical features and diagnosis. J Neurol Neurosurg Psychiatry. 2008 Apr;79(4):368-76. doi: 10.1136/jnnp.2007.131045.
- Background: PubMed 2841426Gibb WR, Lees AJ. The relevance of the Lewy body to the pathogenesis of idiopathic Parkinson's disease. J Neurol Neurosurg Psychiatry. 1988 Jun;51(6):745-52. doi: 10.1136/jnnp.51.6.745.
- Background: PubMed 23487540Pedrosa DJ, Timmermann L. Review: management of Parkinson's disease. Neuropsychiatr Dis Treat. 2013;9:321-40. doi: 10.2147/NDT.S32302. Epub 2013 Mar 3.
- Background: PubMed 22725961Mayo L, Quintana FJ, Weiner HL. The innate immune system in demyelinating disease. Immunol Rev. 2012 Jul;248(1):170-87. doi: 10.1111/j.1600-065X.2012.01135.x.
- Background: PubMed 12401395Ransohoff RM, Howe CL, Rodriguez M. Growth factor treatment of demyelinating disease: at last, a leap into the light. Trends Immunol. 2002 Nov;23(11):512-6. doi: 10.1016/s1471-4906(02)02321-9.
- Individual participant dataSharing planned: Undecided. Annual Summary of Enrolled Cases By All collaborators, principle investigators, or accept sub-investigators
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ryan Welter, MD, PhD principal investigator
Regeneris Medical Inc
Glenn C Terry principal investigator
Global Alliance for Regenerative Medicine (GARM)
Robert W Alexander, MD principal investigator
Healeon Medical
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Microcannula Harvest Adipose, Centricyte 1000 and Sterile Normal Saline Infusion in people with Dementia and Parkinson, run by Regeneris Medical at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ryan Welter, MD, PhD (508.345.5492, r.welter@regenerismedical.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participant with adverse events (AE) or severe adverse events (SAE) (time frame: 6 month intervals for up to 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03297177, which also lists 9 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Oct 2018.