Potential Disease-Modifying Therapies in the Parkinson's Disease Clinical Trial Pipeline last update: 3/13/18

Goal: Enable PD patient/family community to more easily identify funding, staffing, and recruiting needs for high-priority research towards a cure. Compiled by PD advocates Sue Buff & Gary Rafaloff
Source: ClinicalTrials.gov, Cure Parkinson's Trust, Michael J. Fox Foundation, industry websites, Parkinson's advocates & researchers Send additions/corrections to: SueBuff@outlook.com
Note 1: Therapies and trials are listed below in no particular order. Additional data for listed trials is available on ClinicalTrials.gov (trial id# = trial NCT#). File available at PDTrialTracker.info/Collaboration.html
Note 2: Table below includes 29 active trials (18 of which are recruiting), 7 trials in design, and 3 recently completed trials, as of 3/13/18.
Potential Disease-Modifying Therapies in the PD Clinical Trial Pipeline: Active Trials, Trials in Design, Recently Completed Trials
Therapy Locations &
(*=repurposed Recruiting Study Est. study Est. Eligibility: link to
compound) Description Phase Study Title status start date end date Enrollment Sponsor Collaborators ClinicalTrials.gov More Info
Michael Alan
Study of Urate Elevation in PSG, MJFF, Inosine Phase 2
Antioxidant / active, not Schwarzschild,
Inosine* Phase 3 Parkinson's Disease, Phase 3 Jun-16 Aug-20 270 UofRochester, NCT02642393 MJFF Blog 9.1.15
Nutritional supplement recruiting Massachusetts completed 12/12
(SURE-PD3) NINDS (NCT00833690)
General Hospital
Efficacy of Isradipine in Early MJFF website:
Calcium channel active, not NINDS, MJFF,
Isradapine* Phase 3 Parkinson Disease (STEADY-PD Sep-14 Mar-19 336 Univ. of Rochester NCT02168842 MJFF Blog 6.29.16 Therapies in
blocker recruiting PSG
III) Development
Safety and Biomarker Study of EPI- Edison CPT - Linked Clinical
EPI-589* Treats childhood Phase 2A recruiting Mar-16 Jul-18 40 NCT02462603
589 in Parkinson's Disease Pharmaceuticals Trials Initiative
mitochondrial diseases
CPT,
Used to prevent build-
Ambroxol in Disease Modification active, not University College, PRO.MED.CS CPT - Linked Clinical
Ambroxol* up of excess mucous in Phase 2A Dec-16 Apr-18 20 NCT02941822
in Parkinson Disease (AiM-PD) recruiting London Praha a.s - Trials Initiative
respiratory diseases
Czech Republic
Journal of PD
Simvastatin as a Neuroprotective
Cholesterol-lowering Plymouth Hospitals University of CPT - Linked Clinical article 11.1.17 on
Simvastatin* Phase 2 Treatment for Moderate recruiting Sep-15 Sep-20 198 NCT02787590
drug NHS Trust Plymouth Trials Initiative rationale for
Parkinson's Disease (PD STAT)
Simvastatin trial
Lovastatin as a Neuroprotective
Cholesterol-lowering National Taiwan
Lovastatin* Phase 2 Treatment for Early Stage recruiting May-17 Dec-19 80 NCT03242499
drug Parkinson's Disease
University Hospital
Funding received
Conservative Iron Chelation as a
Used to reduce iron European from EU Horizon
Disease-modifying Strategy in University Hospital, CPT - Linked Clinical
Deferiprone* levels in blood after Phase 2 recruiting Feb-16 Dec-18 338 Commission, NCT02655315 2020 research and
Parkinson's Disease Lille Trials Initiative
transfusions ApoPharma innovation
(FAIRPARKII)
program.
Used to reduce iron
Study of Parkinson's Early Stage CPT - Linked Clinical
Deferiprone* levels in blood after Phase 2 recruiting Jul-16 Jul-18 140 ApoPharma NCT02728843
with Deferiprone (SKY) Trials Initiative
transfusions
Phase 2
Trial of Exenatide for Parkinson's
completed. MJFF website:
Exenatide Type 2 Diabetes drug Disease (EXENATIDE-PD) University College, CPT - Linked Clinical
View results: completed Jun-14 Aug-16 60 NCT01971242 Therapies in
(Bydureon)* (GLP-1 agonist) (completed) Journal of PD 8.8.17 London Trials Initiative
CPT site & Development
study commentary
Lancet.

Type 2 Diabetes drug Safety and Efficacy of Liraglutide Cedars-Sinai CPT, Novo CPT - Linked Clinical
Liraglutide* Phase 2 recruiting Apr-17 Sep-19 57 NCT02953665
(GLP-1 agonist) in Parkinson's Disease Medical Center Nordisk A/S Trials Initiative
Study to Evaluate the Effect of CPT, Reseau NS-
Type 2 Diabetes drug not yet University Hospital, CPT - Linked Clinical
Lixisenatide* Phase 2 Lixisenatide in Patients With Mar-18 May-21 158 Park, EUCLID, NCT03439943
(GLP-1 agonist) recruiting Toulouse Trials Initiative
Parkinson's Disease (LixiPark) Sanofi
Treats acetaminophen
Physiological Effects of
NAC (n-Acetyl overdose. Used to phase enrolling by Thomas Jefferson
Nutritional Support in Patients Mar-14 Mar-18 65 NCT02445651
Cysteine)* loosen mucous in cystic unspecified invitation Univ.
with PD
fibrosis & COPD.
A Study to Evaluate the Efficacy Prothena 4.2.17 Prothena 7.5.17 12.20.17 mini-
RO0746015 of RO7046015 in Participants Prothena press release re: press release re: review of PD
Alpha-synuclein vaccine Phase 2 recruiting Jun-17 Jun-21 300 Hoffman-La Roche NCT03100149
(PRX002) With Early Parkinson's Disease Biosciences Phase 1b results Phase 2 study alpha-syn
(PASADENA) (NCT02157714) (NCT03100149) initiatives
Therapy Locations &
(*=repurposed Recruiting Study Est. study Est. Eligibility: link to
compound) Description Phase Study Title status start date end date Enrollment Sponsor Collaborators ClinicalTrials.gov More Info

Evaluating the Safety, MJFF Blog

Phase 1 study
Pharmacokinetics, and 12.20.17 mini- 8/14/15: Two-
NCT02459886
BIIB054 Alpha-synuclein focus Phase 2 Pharmacodynamics of BIIB054 in recruiting Dec-17 Jun-22 311 Biogen NCT03318523 review of PD alpha- more-alpha-
completed Nov.
Participants With Parkinson's syn initiatives synuclein-drugs-
2017.
Disease (SPARK) in-clinical-trials

Follow-up Study to Assess a Second

Phase 1B
Boost Immunization With AFFITOPE® MJFF website: See Affiris 6.7.17
completed
Affitope PD01A Alpha-synuclein vaccine PD01A With Regard to Safety and completed Feb-16 Feb-17 26 Affiris AG NCT02618941 Therapies in press release re:
(results Q4 Clinical Activity (AFF008AA) Development PD03A and PD01A
2017) (completed)

ParkinsonsNewsToday Affiris press release

Phase 1 Study Assessing Tolerability and 6.15.17 Parkinson's 6.7.17 re: results of
completed Safety of AFFITOPE® PD03A in vaccine triggers solid
Affitope PD03A Alpha-synuclein vaccine completed Dec-14 Aug-16 36 Affiris AG NCT02267434 clinical study using
(results Q4 Patients With Early Parkinson's immune response,
AFFITOPE PD03A
2017) Disease (AFF011) (completed) Phase 1 clinical trial
(SYMPATH funded)
shows

A Global Study to Assess the Drug Sanofi Initiates

Dynamics, Efficacy, and Safety of Phase 2 Clinical Trial
RO0746015 Genzyme, a Sanofi
Gene therapy Phase 2 GZ/SAR402671 in Parkinson's recruiting Dec-16 Apr-22 243 NCT02906020 to Evaluate Therapy
(PRX002) Company
Disease Patients Carrying a GBA for Genetic Form of
Mutation (MOVES-PD) Parkinson’s Disease
(2/14/17)
Takara Bio Inc.,
AADC Gene AADC Gene Therapy for Oct -17 / Jichi Medical Gene Therapy
Gene therapy Phase 1/2 recruiting Apr-15 6 NCT02418598
Therapy Parkinson's Disease Jan-22 University Research
Institution Co.
UCSF, Veristat,
Preliminary
Safety Study of AADC Gene Feinstein
AADC Gene active, not Voyager MJFF grant info on positive Phase 1
Gene therapy Phase 1 Therapy (VY-AADC01) for Oct-13 Dec-19 15 Institute, NCT01973543
Therapy recruiting Therapeutics this study results announced
Parkinson's Disease Oregon Health
9/17.
and Science U.
Safety and Efficacy Study of VY-
AADC Gene active, not Voyager
Gene therapy Phase 1 AADC01 for Advanced May-17 Dec-21 16 NCT03065192
Therapy recruiting Therapeutics
Parkinson's Disease
AAV2-NTN for PD:
active, not Sangamo AAV2 (CERE-120) for
CERE-120 (AA2- Gene therapy Phase 1/2 Safety and Efficacy of CERE-120 in Sep-09 Mar-18 60 NCT00985517 Lessons Learned
recruiting Therapeutics PD (3/13 article)
NTN) Subjects with Parkinson's Disease (2016 article)
Long Term Safety and Efficay
active, not
ProvSavin Gene therapy Phase 1/2 Study of ProSavin in Parkinson's May-11 May-22 15 Oxford BioMedica NCT01856439
recruiting
Disease
AAV2-GDNF for Advanced actual: 25;
Gene therapy / active, not enrollment target
AAV2-GDNF Phase 1 Parkinson's Disease May-12 Dec-27 est. was NINDS NCT01621581
Neurotrophic factors recruiting lowered early 2018
(NT01621581) 100
Study website
CDNF (Cerebral herantis.com/pipeli
Clinical Study to Test the Safety (TreatER project.
Dopamine Gene therapy / ne/cdnf-in-
Phase 1/2 of CDNF by Brain Infusion in recruiting Sep-17 Nov-19 18 Herantis Pharma Plc. Renishaw Plc. NCT03295786 Funding from EU
Neurotropic Neurotrophic factors parkinsons-
Patients With Parkinson's Disease Horizon 20/20
Factor) disease/
Program)
Impact of Nilotinib on Safety,
Nilotinib Phase 1
Nilotinib* Leukemia drug (C-Abl Tolerability, Pharmacokinetics
Phase 2 recruiting Jan-17 Jul-20 75 Georgetown Univ. NCT02954978 completed
(Tasigna) Inhibitor) and Biomarkers in PD (PD
(NCT02281474)
Nilotinib)
Nilitonib in Parkinson's Disease Univ. of MJFF, VARI and CPT
Nilotinib* Leukemia drug (C-Abl Northwestern Univ.
Phase 2 (NILO-PD) (patients involved in recruiting Oct-17 Oct-20 135 Rochester, Univ. NCT03205488 collaborate to MJFF Blog 3.15.17
(Tasigna) Inhibitor) Feinberg School of
design) of Iowa, MJFF assess Nilotinib
Medicine
Therapy Locations &
(*=repurposed Recruiting Study Est. study Est. Eligibility: link to
compound) Description Phase Study Title status start date end date Enrollment Sponsor Collaborators ClinicalTrials.gov More Info
A Study to Evaluate the Safety of Cyto Therapeutics, a Trial article in San Trial update in San
ISC-hpNSC Stem cell therapy Phase 1 Neural Stem Cells in Patients recruiting Mar-16 Mar-19 12 subsidiary of Int'l NCT02452723 Diego Union Tribune Diego Tribune
With Parkinson’s Disease Stem Cell Corp. 3/7/2016 11/14/16
Collaborative
www.ncbi.nlm.nih.
Phase 1 Dose Escalation Study of Medicinal www.colmeddev.co
Cu(II)ATSM Phase 1 recruiting Jul-17 Mar-19 38 NCT03204929 gov/pmc/articles/P
Cu(II)ATSM in Parkinson's Disease Development Pty m/procypra/
MC3328361/
Limited
Botanical drug product
Safety and Efficacy of DA-9805
DA-9805 composed of 3 main Phase 2 recruiting Feb-18 Mar-19 60 Dong-A ST Co., Ltd. NCT03189563
for Parkinson's Disease
raw herbal materials
Phase 2 active, not
Disease-modifying Potential of
(results recruiting James Boyd MD, MJFF, PSG, GPS, MJFF NIC-PD Grant
Nicoderm Patch Transdermal nicotine Transdermal NICotine in Early Oct-12 Dec-16 160 NCT01560754
expected Q1 (as of Sep. Univ. of Vermont DPG, et al. Information
Parkinson's Disease (NIC-PD)
2018) '15)
www.bioportfolio.c June 2015 TED
The Stanford Parkinson's om: The-Stanford- Talk: "How Young
Blood Plasma Blood plasma infusion Phase 1 recruiting Nov-16 Dec-18 15 Stanford Univ. NCT02968433
Disease Plasma (SPDP) Study Parkinsons-Disease- Blood Might
Plasma-Study Reverse Aging"
Safety, Tolerability,
Phosphodiesterase 1 Pharmacokinetics and Intra-Cellular
ITI-214 Phase 1/2 recruiting Sep-17 Aug-18 24 NCT03257046
(PDE-1) inhibitor Pharmacodynamics of ITI-214 in Therapies, Inc.
Parkinson's Disease
Targeted at Phase 2A
Phase 2B in design (check with CPT - Linked Clinical
EPI-743 mitochondrial and completed
BioElectron/Edison on status) Trials Initiative
other neuro diseases (NCT01923584)
CPT - Linked Clinical
Used to treat liver
Trials Initiative:
UDCA* disease. Acts to recover Trial in design
Trials Pending
mitochondrial function.
(seeking funding)
Originally developed to
CPT - Linked Clinical
treat Type 2 diabetes.
MSDC-0160* Trials: VARI, CPT, www.msdrx.com/pi
May improve Pre-clinical, Phase 1/Phase 2 in design
(Mitoglitazone) Metabolic Solutions peline/msdc-0160
mitochondrial function
collaboration
in PD.
Treats acetaminophen
CPT - Linked Clinical NAC Phase 1
NAC (n-Acetyl overdose. Used to
Trial in design (CPT: NAC Trial-The Facts) Trials Initiative completed 2012
Cysteine)* loosen mucous in cystic
(seeking funding) (NCT01427517)
fibrosis & COPD.
Neuropore Announces Phase 1 study MJFF update
NPT200-11 Alpha-synuclein vaccine Phase 1B planned by UCB/Neuropore Successful Completion of completed on alpha-syn
Phase I with NPT200-11,
(NCT02606682). trials 7/10/17
3.22.16
Proclara Biosciences - MJFF update
Awaiting results from Alzheimer's phase 1B trial Our Programs (Phase 1B
NPT088 Alpha-synuclein vaccine for Alzheimer's Ref. MJFF NPT088 on alpha-syn
NCT03008161 (est. completion 4/19)
NCT03008161) research grant trials 7/10/17
GM608 Phase 2A
(GAP-PD: Genervon.com -
GM608 Neurotrophic factors Phase 2B planned
NCT01850381) Pipeline
completed.