These experimental therapies are being tested in MSA patients in approved clinical trials in order to evaluate whether they can alter the course of the disease. Scroll down and click on each therapy to learn more including links to opportunities to enroll in clinical trials in progress.
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CURRENT MSA TRIALS
The below experimental therapies are currently being tested in MSA Patients. Click on each experimental therapy to learn more details, including links to clinical trial opportunities.
Tllsh2910 - Phase 3 - no longer recruiting
Cerebellar NMDA (N-methyl-D-aspartate) receptors play a considerable role in motor learning and coordination, thus, may be a feasible target for treating ataxia. Tllsh2910, a NMDA modulator, has been found to attenuate the ataxic gait in the mouse model.
For Phase 3 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT03901638
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
EXENDIN-4(EXENATIDE) - Phase 2 - no longer recruiting
:** Preclinical work funded by the MSA Coalition **
Laboratory work to test Exendin-4 in MSA experimental mouse models was previously carried out at Bordeaux University, France. This project was funded by the MSA Coalition in 2015-2016 and showed positive results, paving the way for MSA patient trials.
Exendin-4 (Exenatide) is an FDA approved drug for treating Diabetes.
Clinical Trial Details:
Phase 2 pilot study details: https://clinicaltrials.gov/ct2/show/NCT04431713
Related Articles:
November 2019: Researchers begin trial of drug to slow progression of neurodegenerative condition Multiple System Atrophy
May 2017: Insulin resistance and exendin-4 treatment for multiple system atrophy
March 2017: Report on MSA Coalition funded research Exendin-4
June 2016: Glucagon-like peptide 1 analogues for treating multiple system atrophy: A translational study
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
COQ10 (Coenzyme Q10) - Phase 2 Completed 2022
A high dose Phase 2 CoQ10 trial in MSA patients was completed in 2022.
Clinical Trial Details:
For historical Phase 2 clinical trial details See: https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000036134
Related Articles:
May 2016: Decreased Coenzyme Q10 Levels in Multiple System Atrophy Cerebellum
July 2013: Mutations in COQ2 in Familial and Sporadic Multiple-System Atrophy
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
NBMI - Phase 2 Completed 2021
NBMI (N1, N3-Bis-(2-Mercaptoethyl) Isophthalamide) is a new metal chelator drug proposed as an alternative to the current chelators, and it is widely different; compared to the current chelators, consisting of two cysteamine molecules coupled to a single molecule of dicarboxybenzoate. It is used as a chelating agent and has the designation of an orphan drug, in the EU and USA; in the EU it is used for the treatment of mercury toxicity. It is freely soluble in solutions of dimethylformamide (DMF), dimethyl sulfoxide (DMSO) and sodium hydroxide diluted NaOH, slightly soluble in methanol and acetone, and insoluble in water. Pre-clinical data indicates low to no toxicity, and that it reduces the toxicity associated with acute exposure to Hg2+.
No other chelator has been reported to prevent acute mercury toxicity with only one exposure to the chelator. It has the ability to penetrate cell membranes and cross the blood-brain barrier and chelate Hg2+ in a complex that eliminates the availability of Hg2+ and essentially eliminates toxic effects. The antioxidant properties of NBMI could also reduce the toxicity levels of hydroxyl free radicals immediately, upon entering cells suffering from oxidative stress. It is possible that the combined chelation of Hg2+ and the elimination of hydroxyl free radicals contribute significantly to the protective effects observed with the NBMI.
Previous clinical studies conducted in subjects of the Phase I and Phase II a studies conducted, did not show significant adverse events in patients intoxicated with mercury, all patients who received the study medication have tolerated it well, with only mild or moderate adverse events reported; None of these were considered related to the pharmacological treatment of the study. In addition, there is no potential identified with safety problems in laboratory tests, or vital signs evaluations.
Clinical Trial Details:
For historical Phase 2 clinical trials details See: https://www.clinicaltrials.gov/ct2/show/NCT04184063
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
Rituximab - Phase 2 - no longer recruiting
The CD20 antibody rituximab, which inhibits B-cell activation, is already approved for autoimmune diseases such as Multiple Scleroisis or rheumatoid arthritis. In a rodent spinal cord injury model rituximab reduced the expression of TNFα in neuronal and glial cells. However, it remains unclear whether rituximab affects the production of naturally occurring autoantibodies possibly playing a crucial role in blocking pathological proteins such as α-syn.
Clinical Trial Details:
For more details see: https://clinicaltrials.gov/ct2/show/NCT04004819
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
MSC (Mesenchymal Stem Cells) - Phase 2 Clinical Trial in Progress **
Clinical Trial Details:
A Phase 2 clinical trial in MSA patients is in progress in the USA.
See: https://www.clinicaltrials.gov/ct2/show/NCT05167721
See also: https://www.mayo.edu/research/clinical-trials/cls-20523723
Historical Clinical Trials:
https://www.clinicaltrials.gov/ct2/show/NCT02315027 and Mesenchymal Stem Cell Therapy in Multiple System Atrophy
https://www.clinicaltrials.gov/ct2/show/NCT00911365
https://clinicaltrials.gov/ct2/show/NCT03265444
Related Videos:
April 2017: VIDEO: Pilot stem cell trial for multiple system atrophy shows promising results
Related Articles:
May 2019: Intrathecal administration of autologous mesenchymal stem cells in multiple system atrophy.
July 2012: A randomized trial of mesenchymal stem cells in multiple system atrophy.
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
ATH434 - Phase 2 Clinical Trial in Progress **
ATH434 (formerly called PBT434) is a small molecule designed to block the accumulation and aggregation of the protein alpha-synclein.
Clinical Trial Details:
See: https://www.clinicaltrials.gov/ct2/show/NCT05109091
Historical Clinical Trials:
A Phase 1 trial in MSA patients was previously completed. See: https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=374741
Related Video:
Related Slide Presentations:
June 2019: Alterity Therapeutics Investor Presentation
May 2019: Alterity Therapeutics AAN Presentation
Related Articles:
January 2022: The Compound ATH434 Prevents Alpha-Synuclein Toxicity in a Murine Model of Multiple System Atrophy
October 2021: Alterity Therapeutics Announces Expanded ATH434 Phase 2 Clinical Development Program
September 2021: Florey Institute of Neuroscience and Mental Health publishes research funded by The Multiple System Atrophy Coalition
July 2021: New Publication Demonstrates ATH434 is Neuroprotective
July 2021: ATH434 Reduces α-Synuclein-Related Neurodegeneration in a Murine Model of Multiple System Atrophy
June 2021: European Union Regulatory Guidance for ATH434 Phase 2 Clinical Trial
April 2021: ATH434 protects brain cells and improves motor function in Parkinsonian disorder
November 2020: Alterity Announces Approval of US Patent for Next Generation Compounds to Treat Neurodegenerative Diseases (ATH434)
October 2020: Alterity commences enrolling Multiple System Atrophy patients in bioMUSE Study (ATH434)
August 2020: New data independently confirms and extends laboratory findings and expands safety profile of ATH434 (PBT-434)
July 2020: Alterity Therapeutics meeting with US FDA provides development pathway for ATH434 (PBT-434)
January 2020: European Commission approves Orphan Designation for Alterity’s lead drug candidate (PBT-434)
July 2019: Alterity Therapeutics Announces Successful Completion of Phase 1 Clinical Trial
June 2019: Alterity Therapeutics Presents to Finance News Network
January 2019: Prana Receives Orphan Drug Designation for PBT434 for the Treatment of Multiple System Atrophy
October 2018: Pre-clinical evidence demonstrates PBT434 as a potential treatment for MSA
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
Anle138B - Phase 1 Completed 2020
Anle138B (TEV-56286) is a small molecule compound that specifically binds toxic oligomeric structures of alpha-synuclein, the core aggregating protein species in Parkinsonian disorders. Through the binding, it effectively dissolves the toxic oligomers and prevents new oligomer formation, addressing the disease at its core. Initial pre-clinical studies in Parkinson’s and MSA animal models have demonstrated the ability to halt disease progression and alleviate symptoms in vivo, effectively preventing the disease from causing further damage by stopping the accumulation of pathological protein aggregates in the brain.
See: ARTEMIS: Targeting Alpha-Synuclein for Treating Multiple System Atrophy
Clinical Trial Details:
A Phase 1 clinical trial to assess safety in healthy volunteers was recently completed. Details may be found at this link: https://www.clinicaltrials.gov/ct2/show/NCT04208152
Related Articles:
October 2021: Teva and MODAG Announce Licensing Collaboration for Neurodegenerative Disease Drug Candidate
December 2019: MODAG Initiates First-in-Human Phase 1 Clinical Trial for Anle138b
June 2019: Modag to Develop Treatments for Parkinsonian Disorders, including Multiple System Atrophy
February 2014: New drug against Alzheimer’s and Parkinson’s on the way
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
ION464 - Phase 1 Clinical Trial In Progress *
ION464 (BIIB101/IONIS-BIIB6Rx ) is an antisense drug targeting alpha-synuclein (SNCA) messenger ribonucleic acid (mRNA). BIIB101 is designed to prevent the production of alpha-synuclein protein and is being developed as a potential therapy for Parkinson’s disease (PD), Multiple System Atrophy (MSA) and related synucleinopathies. Alpha-synuclein protein aberrantly accumulates in the brains of PD and MSA patients and is thought to be one of the key drivers of pathogenesis. It is hypothesized that reduction of SNCA mRNA and, subsequently, reduced synthesis of alpha-synuclein protein will ameliorate the toxic effects of gain-of-function mutations as well as the primary pathology in PD and MSA patients without SNCA mutations.
Clinical Trial Details:
See: https://www.clinicaltrials.gov/ct2/show/NCT04165486
Related Articles:
August 2020: Ionis reports second quarter 2020 financial results and recent business achievements
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
AAV2-GDNF Gene Therapy - Phase 1 Clinical Trial in Progress *
The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.
Clinical Trial Details:
For Phase 1 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT04680065
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
Lu AF82422 - Phase 2 - no longer recruiting
Lu AF82422 is a monoclonal antibody targeting alpha-synuclein. Misfolding, aggregation and extracellular spreading of alpha-synuclein is believed to play a major role in disease pathology and progression in Multiple System Atrophy (MSA), Parkinson’s disease and other neurodegenerative disorders.
Clinical Trial Details:
A Phase 2 Clinical trial is currently underway – as of October 2022 this trial is no longer recruiting new patients.
See: https://www.clinicaltrials.gov/ct2/show/NCT05104476
See also: http://amulet.researchstudytrial.com
Historical Clinical Trials:
A Phase 1 Clinical trial in Healthy subjects and in Patients With Parkinson’s Disease was previously completed.
For historial Phase 1 clinical trial details see: https://www.clinicaltrials.gov/ct2/show/NCT03611569
Related Articles:
November 2021: Lundbeck launches a phase II study for potential new treatment of multiple system atrophy
August 2018: Lundbeck Brings Potential New Biologic Treatment of Parkinson’s Disease into Clinical Development
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
TAK-341 - Phase 2 Clinical Trial in Progress **
“Passive Immunization”
TAK-341 (MEDI-1341; BIIB054) is an anti-alpha-synuclein antibody being tested in people with Multiple System Atrophy and Parkinson’s Disease.
Clinical Trial Details:
See: https://msa-clinicalstudy.com/en-US#home
See: https://www.clinicaltrials.gov/ct2/show/NCT05526391
See: https://clinicaltrials.takeda.com/study-detail/e0316361bd7044cd?idFilter=%5B%22TAK-341-2001%22%5D
Historical Clinical Trials:
A Phase I Clinical trial of the alpha-synuclein antibody MEDI-1341 was completed in Parkinson’s Disease patients. See: https://clinicaltrials.gov/ct2/show/NCT04449484
Phase I Clinical trial of the alpha-synuclein antibody MEDI-1341 was completed in healthy volunteers. See: https://clinicaltrials.gov/ct2/show/NCT03272165
Related Articles:
August 2017: AstraZeneca and Takeda establish collaboration to develop and commercialise MEDI1341 for Parkinson’s disease
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
ACI-7104 - Phase 1 Completed 2017
“Active Immunization”
ACI-7104 is an optimized formulation of a clinically validated anti-a-syn predecessor vaccine (Affitope PD01A PD03A). It generates a target-specific antibody response against pathological oligomeric a-syn to inhibit spreading and downstream neurodegeneration in early Parkinson’s disease. It produces a strong and boostable antibody response with evidence of target engagement and a signal of clinical efficacy.
Historical Clinical Trials:
For historical Phase 1 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT02270489
Related Videos:
October 2019: A vaccine for Parkinson’s disease shows promising results
Related Articles:
July 2021: AC Immune Announces Strategic Acquisition of Industry-leading Parkinson’s Disease Vaccine Candidate
September 2020: A Phase 1 Randomized Trial of Specific Active α‐Synuclein Immunotherapies PD01A and PD03A in Multiple System Atrophy
September 2016: Boost Vaccination Data Encourage Continued Development of AFFiRiS Therapeutic Parkinson’s Disease Vaccine Against Alpha-Synuclein
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
HISTORICAL MSA TRIALS
The below experimental therapies were tested in multiple system atrophy patients in past years. No disease modifying effects have been proven. Click on each experimental therapy to learn more details.
VERDIPERSTAT (BHV-3241) - Phase 3 Completed 2022
Preclinical study of Verdiperstat funded by the MSA Coalition
MSA Coalition Grant:
“Verdiperstat target engagement in a unique multiple system atrophy postmortem brain matched to iPSC”
Awardee: Vikram Khurana, MD PhD
Grant #2021-09-006 – $50,000
Verdiperstat is an oral myeloperoxidase (MPO) inhibitor. MPO is a key driver of oxidative and inflammatory processes and is significantly increased in a range of brain disorders. It is thought that inhibiting MPO activity may be a promising therapeutic strategy for neuroinflammatory and neurodegenerative conditions, including MSA.
Historical Clinical Trial Details:
A Phase 3 clinical trial in MSA patients is complete.
See article from September 27, 2021: Biohaven Provides Update On Phase 3 Trial And Multiple System Atrophy (MSA) Program
For Historical Phase 3 clinical trials details See: https://clinicaltrials.gov/ct2/show/NCT03952806
See also: https://www.msaresearchstudy.com/about-the-study
A Phase 2 clinical trial of MSA patients was previously completed. For historical Phase 2 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT02388295
Related Videos:
May 2021: Biohaven Video: MSA MPO Inhibition
Related Posters:
May 2021: Updates on Biohaven’s study to evaluate the efficacy and safety of BHV- 3241 (Verdiperstat)
February 2021: Biohaven Shares Verdiperstat’s Mechanism of Action
November 2020: M-STAR, an Ongoing Phase 3 Study in Participants with Multiple System Atrophy–Baseline Characteristics (Verdiperstat)
Related Articles:
September 27, 2021: Biohaven Provides Update On Phase 3 Trial And Multiple System Atrophy (MSA) Program
September 15, 2021:Theravance Biopharma, Inc. announces top-line results from a Phase 3 study of Ampreloxetine in patients with symptomatic neurogenic orthostatic hypotension
June 2021: Updated Q&A on Verdiperstat
May 2021: Updates on Biohaven’s study to evaluate the efficacy and safety of BHV- 3241 (Verdiperstat)
April 2021: ATH434 protects brain cells and improves motor function in Parkinsonian disorder
February 2021: Biohaven Shares Verdiperstat’s Mechanism of Action
November 2020: M-STAR, an Ongoing Phase 3 Study in Participants with Multiple System Atrophy–Baseline Characteristics
April 2020: MSA Coalition Blog – Q & A about Verdiperstat Clinical Trial
March 2020: Biohaven’s Verdiperstat Receives Fast Track Designation for the Treatment of Multiple System Atrophy
January 2019: Biohaven Receives FDA May Proceed Letter For Phase 3 Clinical Trial Of BHV-3241 For Multiple System Atrophy
September 2018: Biohaven Licenses Novel Myeloperoxidase Inhibitor From AstraZeneca: Potential First-In-Class Treatment For Multiple System Atrophy
July 2015: Failure of Neuroprotection Despite Microglial Suppression by Delayed-Start Myeloperoxidase Inhibition in a Model of Advanced Multiple System Atrophy: Clinical Implications
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
SIROLIMUS (rapamycin) - Phase 2 Terminated 2021
Sirolimus is an FDA approved immunosuppressant drug (also known as rapamycin).
A Phase 2 clinical trial in MSA patients was previously completed.
See posted results: https://www.clinicaltrials.gov/ct2/show/results/NCT03589976
For historical Phase 2 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT03589976
Related Articles:
October 2020: IXICO and NYU Langone Health Sign Agreement to Develop Novel Imaging Markers in Multiple System Atrophy (Sirolimus)
October 2019: Research Speeds Ahead – 1 Year Update from the Sirolimus MSA Trial
December 2018: New study testing a potential drug to slow the progression of MSA opens at NYU
October 2018: Rapamycin for treating MSA: A preclinical proof of concept study
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
EGCG (Epigallocatechin gallate) - Phase 3 Completed 2016
A Phase 3 clinical trial to test green tea extract (EGCG) in MSA patients was previously completed.
For historical Phase 3 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT02008721
EGCG, a polyphenol found in green tea, has shown to inhibit the formation of toxic α-synuclein oligomers in vitro and has shown to transform α-synuclein-oligomers in non-toxic oligomer species. There is also evidence for a neuroprotective effect in MPTP-mouse models of PD and is an antioxidant and iron chelator. There are currently 63 clinical studies (http://clinicaltrial.gov) in which EGCG was applied for various indications, such as Multiple Sclerosis, various forms of cancer and Huntington’s disease. All of which have shown good tolerability and safety with the applied doses of EGCG of up to 1200 mg per day, demonstrating the safety of the drug under controlled clinical conditions. These data provide a solid rationale for testing in a clinical trial if supplementation of EGCG can interfere with the core disease mechanism in MSA and consequently retard the clinical progression of the MSA-related disability.
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
IVIG (intravenous immunoglobulin) - Phase 2 Completed 2015
A Phase 2 clinical trial in MSA patients was previously completed.
For historical Phase 2 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT00750867
Related Articles:
November 2012: Treatment of multiple system atrophy using intravenous immunoglobulin
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
RIFAMPICIN - Phase 3 Terminated 2013
A Phase 3 clinical trial in MSA patients was previously completed.
For historical Phase 3 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT01287221
Rifampicin, because of its ability to inhibit the formation of α-synuclein fibrils and disaggregate fibrils already formed, was hoped to delay progression or reverse neurologic and autonomic functions and symptoms in MSA. In an experimental model of MSA, it was hypothesized that Rifampicin would improve behavioral abnormalities of MSA and halt or reverse the pathological change.
The Data Safety Monitoring Board (DSMB) recommended stopping the study after an interim analysis of the primary endpoint revealed that futility criteria were met.
Related Articles:
March 2014: Efficacy and safety of rifampicin for multiple system atrophy: a randomised, double-blind, placebo-controlled trial.
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
INTRANASAL INSULIN - Phase 2 Completed 2012
A Phase 2 clinical trial in Parkinson and MSA patients was previously completed.
For historical Phase 2 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT02064166
Related Articles:
May 2019: Treatment with Intranasal Insulin May Improve Verbal Fluency and Motor Function, Early Study Shows
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
LITHIUM - Phase 2 Terminated 2011
A Phase 2 clinical trial of Lithium in MSA patients was terminated in 2011.
See: https://clinicaltrials.gov/ct2/show/NCT00997672
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
FLUOXETINE(Prozac) - Phase 2 Completed 2011
A Phase 2 clinical trial in MSA patients was previously completed.
For historical Phase 2 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT01146548
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
RASAGILINE - Phase 2 Completed 2011
A Phase 2 clinical trial in MSA patients was previously completed.
For historical Phase 2 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT00977665
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
MINOCYCLINE - Phase 3 Completed 2005
A Phase3 clinical trial in MSA patients was previously completed
For historical Phase 3 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT00146809
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
RILUZOLE - Phase 3 Terminated 2004
A Phase 3 clinical trial in Progressive Supranuclear Palsy and MSA patients was previously terminated.
For historical Phase 3 clinical trial details See: https://clinicaltrials.gov/ct2/show/NCT00211224
Related Articles:
June 2009: Riluzole Does Not Prolong Survival in Progressive Supranuclear Palsy and Multisystem Atrophy
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
PARKINSON DISEASE TRIALS
The below experimental therapies are being tested in Parkinson’s Disease Patients and there is hope they may eventually be tested in MSA Patients. Click on each experimental therapy to learn more details, including links to clinical trial opportunities.
Prasinezumab - Phase 2 for Parkinson's in progress
“Passive Immunization”
Prasinezumab (also known as PRX002, RO7046015, RG7935, NEOD002) is a humanized IgG1 monoclonal antibody directed against aggregated α-synuclein. Genetic and pathological evidence suggest that this protein plays a central role in the pathogenesis of Parkinson’s disease (PD) and other α-synculeinopathies such as dementia with Lewy bodies (DLB).
Clinical Trial Details:
A Phase 2 clinical trial in Parkinson’s patients is in progress. See: https://clinicaltrials.gov/ct2/show/NCT03100149
Historical Clinical Trials:
A Phase 1 clinical trial in Parkinson’s patients was previously completed. For historical Phase 1 clinical trial details see: https://clinicaltrials.gov/ct2/show/NCT02157714
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
UCB0599 - Phase 2 in Parkinson's in progress
By stabilizing conformations of alpha-synuclein that are then incapable of assembling into toxic pore-like oligomers in cell membranes, UCB0599 (formerly known as NPT200-11), blocks the pathological protein misfolding, aggregation and deposition that contribute to synaptic dysfunction and cell death in PD and related disorders. UCB0599 is orally bioavailable, has promising drug-like properties and, has shown robust beneficial actions on multiple endpoints in animal models.
Clincal Trial Details:
A Phase 2 clinical trial of UCB0599 in Parkinson’s Disease is in progress. See: https://www.clinicaltrials.gov/ct2/show/NCT04658186
Historical Clinical Trials:
A Phase 1 clinical trial of NPT200-11 in healthy volunteers was previously completed. For historical Phase 1 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT02606682
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
ABBV-0805 - Phase 1 for Parkinson's in progress
ABBV-0805 is an alpha-synuclein antibody that works by binding to toxic clumps of alpha-synuclein and helping remove them. Pre-clinical studies showed that it did remove the majority of these clumps, which led to slower disease progression and reduced motor symptoms in Parkinson’s disease models.
Clinical Trial Details:
A Phase 1 clinical trial in Parkinson’s Disease is in progress. See: https://www.clinicaltrials.gov/ct2/show/NCT04127695
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
MEDI-1341 - Phase 1 for Parkinson's in progress
“Passive Immunization”
MEDI-1341 BIIB054 is an anti-alpha-synuclein antibody being tested in people with Parkinson’s Disease.
Clinical Trial Details:
A Phase I Clinical trial of the alpha-synuclein antibody MEDI-1341 is in progress for Parkinson’s Disease. See: https://clinicaltrials.gov/ct2/show/NCT04449484
Historical Clinical Trials:
Phase I Clinical trial of the alpha-synuclein antibody MEDI-1341 was completed in healthy volunteers. See: https://clinicaltrials.gov/ct2/show/NCT03272165
Related Articles:
August 2017: AstraZeneca and Takeda establish collaboration to develop and commercialise MEDI1341 for Parkinson’s disease
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
Ambroxol - Phase 2 for Parkinson's Completed 2019
Ambroxol is the main ingredient of over-the-counter cough medicines sold in many countries. Ambroxol is unavailable in North America. Interest in repurposing ambroxol to treat Parkinson’s disease stems from its activity as a molecular chaperone for the lysosomal enzyme glucocerebrosidase (GCase). Loss-of-function mutations in the GCase gene, GBA1, are the leading genetic risk factor for the synucleinopathies Parkinson’s disease and dementia with Lewy bodies (DLB).
Clinical Trial Details:
A Phase II Clinical Trial in Parkinson’s disease patients was recently completed. Details may be found at this link: https://clinicaltrials.gov/ct2/show/NCT02941822
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
BIIB054 - Phase 2 for Parkinson's Terminated 2021
“Passive Immunization”
BIIB054 is an anti-alpha-synuclein antibody being tested in people with Parkinson’s Disease.
Historical Clinical Trials:
A Phase II clinical trial for Parkinson’s Disease was terminated in 2021. See: https://clinicaltrials.gov/ct2/show/NCT03318523
Related Articles:
January 2018: First patient dosed in the Phase 2 SPARK study of BIIB054 (anti-alpha-synuclein antibody) in Parkinson’s disease
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
NPT088 - Phase 1 in Alzheimer's Completed 2019
NPT088 can bind to several proteins involved in brain diseases, including alpha-synuclein, amyloid-beta and tau.
A Phase I trial in people with Alzheimer’s was recently completed. For historical Phase I clinical trial information see: https://www.clinicaltrials.gov/ct2/show/NCT03008161.
NPT088 will also be tested in people with Parkinson’s as soon as an imaging tool for alpha-synuclein is available.
Related Articles:
NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.
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