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Sponsor: BlueRock Therapeutics

Official Title: exPDite-2: A Phase 3 Study to Assess the Efficacy and Safety of Midbrain Dopaminergic Neuronal Cell Therapy (Bemdaneprocel) for Participants With Parkinson's Disease

Purpose: To assess the efficacy and safety of bemdaneprocel in adults with Parkinson's Disease (PD).

Study design: Randomized, double-blind, sham-surgery-controlled, with open-label period

Study duration: 78 weeks, up to 60 months

Basic inclusion criteria:

• Individual of any sex ≥45 to ≤75 years of age at informed consent
• Robust and clear response to DA therapy as defined by MDS-UPDRS Part III
• ≥4 and <12 years from time of PD diagnosis at informed consent
• Must demonstrate responsiveness to levodopa therapy
• Receiving medical therapy for the treatment of PD symptoms
• ≥2.5 hours of daily OFF-time
• Vaccinated per current national guidelines or local practice for patients with altered immunocompetence

Basic exclusion criteria:

• PD presenting with recurrent falls
• Diagnosis of primary mitochondrial disorder, epilepsy, stroke, multiple sclerosis, or clinical features suggestive of a neurodegenerative disease other than PD, including multiple system atrophy, progressive supranuclear palsy, corticobasal degeneration, or Lewy body dementia
• Any current or relevant previous history of serious, severe, or unstable physical, neurological, or psychiatric illness that may interfere with study participation, participant's safety, or assessment of endpoints per investigator's judgment
• History of gene therapy or cell therapy
• Prior treatment with intrajejunal or subcutaneous infusion therapies for PD
• Prior surgical or radiation therapy to the brain, including deep brain stimulation (DBS) and lesion therapy, or prior history of intradural spinal cord surgery
• Contraindication to surgery, general anesthesia, cell therapy, immunosuppression, or other required drugs, or anything that prevents use of PET or MRI
• Any active infection (including but not limited to HIV, HCV, HBV, CMV, syphilis, or tuberculosis) or condition that, in the opinion of the investigator could put the participant at significant risk from immunosuppression or impact the participant's ability to perform study assessments
• Current or previously active malignant disease within the past 5 years
• Chronic immunosuppressive therapy
• Receipt of another investigational therapy within 5 half-lives of the active treatment
• Pregnancy or breastfeeding

Sponsor: Asklepios Biopharmaceutical, Inc.

Official Title:  A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease

Purpose: The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.

Study design: Randomized, surgically controlled, double-blinded

Study duration: 18 months

Basic inclusion criteria:

• Adults 45-75 years of age inclusive
• Diagnosed with Parkinson's Disease in the past 4-10 years (inclusive)
• Presence of motor fluctuations as measured by the PD Motor Diary
• Stable anti-parkinsonian medication regiment for >/= 4 weeks prior to screening
• Must demonstrate responsiveness to levodopa therapy

Basic exclusion criteria:

• Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to idiopathic PD diagnosis
• Presence or history of significant vascular and/or cardiovascular disease
• Presence of significant cognitive impairment, poorly controlled depression/anxiety
• Presence or history of psychosis or impulse control disorder
• History of malignancy other than treated cutaneous squamous or basal cell carcinomas
• Presence of clinically relevant conditions that could compromise surgical suitability and/or subject safety
• Contraindication to magnetic resonance imaging and/or use gadolinium-based contrast agents
• Prior history of brain surgery including, but no limited: DBS, pallidotomy focused ultrasound thalamotomy, or other experimental neurosurgical procedure
• Chronic immunosuppressive therapy

Sponsor: Cerevance

Official Title: Phase 3, Randomized, Double-Blind, Placebo-Controlled Multicenter Study of CVN424 in Parkinson's Disease Patients With Motor Complications

Purpose:  This is a randomized, double-blind, placebo-controlled, multicenter study in participants with Parkinson's disease (PD) with motor fluctuations. Participants will be randomized to receive once-daily oral doses of either 75 milligrams (mg) CVN424, 150 mg CVN424, or a matching placebo for 12 weeks. Participants who successfully complete this study and retain eligibility/suitability will be invited to participate in a future open-label extension (OLE) study.

Study design: Randomized, double-blind, placebo-controlled

Study duration: 12 weeks

Basic inclusion criteria:

• 30 years and older
• Diagnosis of PD .
• Body Mass Index (BMI) > 18.0 and < 35.0 Kilograms per meter square (kg/m^2), inclusive at Screening.
• Freely ambulatory at the time of Screening (with/without assistive device).
• PD medications must be stable for at least 4 weeks prior to Screening; monoamine oxidase B (MAO-B) inhibitors must be stable for at least 12 weeks prior to Screening.
• Levodopa administration at least 4 times daily (immediate or extended release) or three times daily (Rytary).
• Stable use of oral anti-sialorrhea medications for 30 days before Screening, without anticipated need for change during the study.
• During Screening, capable of adequately identifying ON, OFF, and dyskinetic states (>80% concordance with a blinded rater) through properly completed ON/OFF diaries.
• Female participants of childbearing potential and male participants with female partners of childbearing potential must agree to either remain abstinent or use adequate and reliable contraception throughout the study and for at least 12 weeks after the last dose of study drug has been taken.
• Able and willing to give written informed consent approved by an institutional review board, and to comply with scheduled visits, treatment plan, laboratory tests, and other study-related procedures.

Basic exclusion criteria:

• Diagnosis of secondary or atypical parkinsonism.
• Severe or disabling dyskinesias or OFF expected to preclude successful study participation, in the opinion of the investigator.
• Any previous procedure or therapy designed to provide continuous levodopa or stimulation of dopaminergic tone (i.e., Duopa, apomorphine), surgery for PD (i.e., deep brain stimulation [DBS]), or anticipation of these during the study.
• Clinically significant orthostatic hypotension (consistently symptomatic or requires medication).
• Clinically significant hallucinations requiring antipsychotic use.
• Routine use of PD on-demand medications (i.e., inhaled levodopa)
• Use of injectable botulinum medication for sialorrhea within 90 days of screening or during the study.
• Clinically significant medical, surgical, psychiatric, or laboratory abnormalities that in the judgment of the investigator would preclude adequate participation or completion of the study.
• Clinically significant ECG abnormalities at Screening.
• Clinically significant heart disease within 2 years of Screening
• Active major depressive disorder
• Has active suicidal ideation within one year prior to Screening or attempted suicide within the last 5 years.
• Has been diagnosed with or history of a substance-related disorder (excluding nicotine and caffeine), including alcohol-related disorder, during the 12 months prior to Screening.
• Tests positive at Screening for drugs of abuse (opiates, tetrahydrocannabinol [THC], methadone, cocaine, and amphetamines [including ecstasy]).
• Has a positive test result for hepatitis B surface antigen (HBsAg), hepatitis C virus antibodies (HCV) antibody, or Human Immunodeficiency Virus (HIV) infection at Screening.
• Currently participating in or has participated in another study of an investigational medicinal product (IMP) or medical device in the last 3 months or within 5 half-lives of the IMP (whichever is longer) prior to Screening.

Sponsor: Neuron23 Inc.

Official Title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of NEU-411 in Companion Diagnostic-Positive Participants with Early Parkinson's Disease

Purpose:  The goal of this Phase 2 clinical trial is to investigate the efficacy and safety of NEU-411 in men and women aged 50-80 years with early Parkinson's Disease (PD) who have predicted elevations in the activity of the "leucine-rich repeat kinase 2" ("LRRK2" for short) pathway based on their genetic profile. A DNA test will be used to identify the "LRRK2-driven" population with predicted elevation in the LRRK2 pathway.

Study design: Randomized, double-blind, placebo-controlled

Study duration: 52 weeks

Basic inclusion criteria:

• Aged 50-80 years at time of screening, inclusive
• Diagnosis of clinically established or clinically probable Parkinson's Disease (PD)
• LRRK2-driven PD using the investigational companion diagnostic genetic test (CDx)

Basic exclusion criteria:

• Secondary or atypical parkinsonian syndromes
• Uncontrolled diabetes mellitus with hemoglobin A1c (HbA1c) >8%
• Other significant medical conditions (as determined by medical history, examination, or clinical investigations at screening)

Sponsor: ACADIA Pharmaceuticals Inc.

Official Title: A Double-Blind, Placebo-Controlled, Phase 2, Efficacy and Safety Study of ACP-204 in Adults With Lewy Body Dementia Psychosis (LBDP)

Study design: Randomized, double-blind, parallel-group, placebo-controlled

Study duration: 6 Weeks

Basic inclusion criteria:

• Male or female ≥55 years to <85 years of age at the Screening visit living in the community
• Can provide written informed consent. If the subject is deemed not competent to provide informed consent, the following requirements for consent must be met:
  • The subject's LAR must provide written informed consent.
  • The subject must provide written (if capable) informed assent per local regulations.

Basic exclusion criteria:

• Is in hospice, is receiving end-of-life palliative care, or is bedridden
• Has psychotic symptoms that are primarily attributable to delirium, substance abuse, or a medical or psychiatric condition other than dementia
• Is actively suicidal at Visit 1 or Visit 2
• Has a history or current evidence of a serious and/or significant unstable cardiovascular, respiratory, endocrine, gastrointestinal, renal, hepatic, hematologic, immunologic, genitourinary, psychiatric or neurologic (including stroke, chronic seizures, or clinically significant head injury) abnormality or disease or other medical disorder, including cancer or malignancies that could interfere with subject’s ability to complete the study or comply with study procedures

Sponsor: Teva Branded Pharmaceutical Products R&D, LLC

Official Title: A Multi-centered, Double-blind, Randomized, Placebo-controlled, Parallel Group Phase 2 Study of TEV-56286 for the Treatment of Patients With Multiple System Atrophy

Purpose: To evaluate the efficacy of TEV-56286 administered orally for the treatment of adult participants with Multiple System Atrophy (MSA).

Study design: Randomized, double-blind, parallel-group, placebo-controlled

Study duration: Approximately 56 weeks including a screening period (up to 4 weeks), a 48-week double-blind treatment period, and a follow-up visit (approximately 4 weeks after the end of the double-blind treatment period).

Basic inclusion criteria:

• “Clinically possible” or “clinically probable” MSA
• Medically and psychiatrically stable, as indicated by medical and psychiatric history, as well as physical and neurological exam

Basic exclusion criteria:

• Has 2 or more relatives with a history of MSA
• Has participated in another clinical study involving administration of an IMP within 3 months or 5 half-lives (whichever is longer) of this IMP prior to screening
• Has a history of, or acknowledges, alcohol or other substance abuse in the 12 months before screening

Sponsor: Ono Pharmaceutical Co. Ltd

Official Title: A Phase 2, Double-blind, Placebo-controlled, Parallel-group Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Potential Efficacy of Multiple Doses of ONO-2808 in Patients With Multiple System Atrophy (MSA)

Purpose: To evaluate 3 doses of ONO-2808 compared to placebo in MSA patients, including: 1) safety and tolerability, 2) pharmacokinetics, and 3) changes in clinical outcome assessments (COA) and biomarkers considered to be related to the pharmacodynamics and potential efficacy of ONO-2808.

Study design: Randomized, double-blind, parallel-group, placebo-controlled

Study duration: 28 weeks

Basic inclusion criteria:

• Female or male patients with a diagnosis of clinically-established or clinically-probable MSA according to the novel Movement Disorder Society (MDS) criteria for MSA diagnosis (2022), including patients with MSA of either subtype (MSA-P or MSA-C).
• Patients at the early stages of the disease, defined as a maximum of 5 years since the onset of one of the following symptoms associated with MSA:
  • Parkinsonism
  • Ataxia
  • Orthostatic hypotension and/or urinary dysfunction
• Patients with an anticipated survival of at least 3 years in the opinion of the Investigator.
• Patients who are able to ambulate without the assistance of another person, defined as the ability to take at least 10 steps and then to turn around and walk at least another 10 steps. Use of assistive devices (e.g., walker or cane) is allowed.
• Ability to swallow oral medication and be willing to adhere to the study intervention regimen.

Basic exclusion criteria:

• Pregnant or lactating females.
• Patients with a clinically-significant or unstable medical or surgical condition other than MSA that, in the opinion of the Investigator, might preclude safe completion of the study or might affect the results of the study (e.g., pulmonary, cardiovascular [including bradyarrhythmia], macular edema, and significant renal or hepatic dysfunction).
• Neurological diseases/disorders other than MSA, such as Parkinson's disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraparesis, corticobasal degeneration, or vascular, normal pressure hydrocephalus, pharmacological, or post-encephalitic parkinsonism.
• Patients with documented liver diseases or cirrhosis.
• Positive results at Screening for active viral infections that include positive human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg) and hepatitis B core antibody, and hepatitis C virus (HCV).
• Patients who have made a suicide attempt in the 6 months before Screening

Parkinson’s Progression Markers Initiative (PPMI)

Sponsor: The Michael J. Fox Foundation

Duration: 5+ years (up to 13 years total)

Brief summary: PPMI aims to better understand how Parkinson’s disease (PD) starts and changes over time. The study follows groups of people, including those both with and without Parkinson’s disease. The information from this study could transform how we diagnose, treat, and potentially prevent PD.

Basic eligibility criteria:

• Individuals who are at least 60 years old and are: First-degree family members (parent, child, sibling) of a person with Parkinson’s OR

• • People who have risk factors for the development of PD (known genetic mutation, loss of smell, history of physically acting out dreams during sleep, and others)
• People without Parkinson’s and no known risk to act as a comparison group

What: The Speech Science and Disorders Lab is conducting a research study to understand how the biomechanics and muscles of the face and mouth affect the ability to speak in people with neurodegenerative diseases such as Parkinson’s disease (PD).

Why: Speaking often becomes difficult as neurodegenerative diseases progress. The causes of these speech difficulties are very complex and need to be better understood in order to provide optimal clinical care so that patients can maintain their ability to communicate.

How: Participants will complete a 2-2.5 hour experimental session every 3-4 months. In each session, participants will perform a few speaking tasks, while their facial muscle activities, mouth movements, and speech will be recorded.

Where: Speech Science & Disorders Lab at KU-Lawrence

Criteria:

• Speak American English as your primary language
• Are diagnosed with a neurologic disease such as Parkinson's Disease
• Have no history of other neurologic or cognitive disorders 

Compensation: $20 compensation for each study session

For more information, please contact: 785-864-0053 or MSDProject.SSDLab@gmail.com 

Purpose: Researchers are studying mild exercises for sleep quality in people with Parkinson's disease.  

Who: Ages 40-75 years, Diagnosis of idiopathic PD

What:

• Mild exercise at home for 12 weeks
• 15 to 20 minutes per exercise session
• Online group meeting once per week

Compensation: Up to $100 ($50 per visit to the research lab)

Contact: Elvira O'Neal, eoneal@kumc.edu, 913-251-7822 or Wen Liu, wliu@kumc.edu, 913-588-4565