Parkinson's Disease Research & Clinical Trials

RESTORE-1 Clinical Study – An Experimental, One-Time Gene Therapy for Parkinson’s Disease

The main purpose of this clinical study is to evaluate the safety and efficacy of an experimental gene therapy on Parkinson’s disease motor function. A surgical procedure will be used to administer the experimental gene therapy, which involves placing the AADC gene into a specific part of the brain where it is needed to convert levodopa into dopamine. This is intended to program cells in the brain to produce AADC enzyme needed and could improve motor function. This gene therapy is investigational and has not been approved for Parkinson’s disease.

If your response to medications has become unpredictable or isn’t what it used to be, this clinical study may be an option that you should discuss with your doctor.

Inclusion Criteria

• Age 40 to 75 years
• Have been diagnosed with Parkinson’s disease for at least four years
• Able and willing to travel and take part in extended study visits, including off-medication visits
• Have noticed your motor symptoms are unpredictable despite medication

Exclusion Criteria

• Atypical or secondary Parkinsonism
• Prior brain surgery or infusion therapies that could complicate the study procedure
• Inability to comply with procedures of the protocol including frequent and prolonged visits

Contact: Stephanie Burrows, email or call (617) 667-9885

The purpose of this project is to understand the natural progression of patients diagnosed with synucleinopathies. Synucleinopathies are a group of rare diseases associated with worsening neurological deficits and the abnormal accumulation of the protein α-synuclein in the nervous system. These diseases include Parkinson’s disease, Pure autonomic failure, Dementia with Lewy bodies and Multiple system atrophy. As a part of this study, we are also investigating whether small skin biopsies can be used as a novel method of detecting disease and monitoring for progression in the disease over time.

Inclusion Criteria

• Male and female participants aged 18 or over
• Has a diagnosis of neurogenic Orthostatic Hypotension, Pure autonomic failure, Dementia with Lewy bodies or Multiple system atrophy

Exclusion Criteria

• Drug-induced orthostatic hypotension
• Isolated vasovagal syncope
• Individuals with sensory or motor features consistent with a peripheral neuropathy
• Patients on warfarin, acetylsalicylic acid and clopidogrel simultaneously, or other combinations of agents that would impair wound healing).

The Principal Investigator for this study at Beth Israel Deaconess Medical Center is Dr. Roy Freeman, MD

Contact: Sharika Rajan, srajan@bidmc.harvard.edu

Multiple system atrophy (MSA) is a fatal, adult-onset disease of the nervous system associated with α-synuclein protein deposition. BHV-3241 is an irreversible inhibitor of myeloperoxidase enzyme which is a key driver of oxidative and neuroinflammatory process that underlie neurodegeneration inpatients with MSA. The purpose of this Phase 3 study is to demonstrate the efficacy of BHV-3241 in the treatment of MSA and characterize its safety and tolerability profile. For this study, we will enroll patients diagnosed with MSA in the early stages of the disease. The study will be conducted in several closely monitored visits over a period of 48 weeks. Monetary compensation will be provided.

Inclusion Criteria

• Male and female subjects between the ages of >40 to <75 years at time of Screening.
• Diagnosis of probable or possible MSA according to consensus clinical criteria (Gilman et al 2008), including subjects with MSA of either subtype (MSA-P or MSA-C).
• Able to ambulate without the assistance of another person, defined as the ability to take at least 10 steps. Use of assistive devices (e.g., walker or cane) is allowed.
• Anticipated survival of at least 3 years at the time of Screening, as judged by the Investigator.
• A brain MRI scan (conducted within 14 days of Baseline/Day 1) that does not rule out a diagnosis of MSA.

Exclusion Criteria

• Diagnosis of neurological disorders, other than MSA
• History or presence of clinically significant thyroid disease
• History of brain surgery for Parkinsonism
• History of stem-cell treatment
• Dopamine antagonists, including metoclopramide and antipsychotic medications

The Principal Investigator for this study at Beth Israel Deaconess Medical Center is Dr. Roy Freeman, MD

Contact: Sharika Rajan, srajan@bidmc.harvard.edu

A sustained drop in blood pressure upon change in posture with disorders of the autonomic nervous system is defined as neurogenic orthostatic hypotension (nOH). A profound reduction in or failure of norepinephrine (NE) neurotransmission is seen in patients with nOH and they may experience lightheadedness, dizziness or syncope, amongst other symptoms. TD-9855 (ampreloxetine hydrochloride) is a new drug being developed for various medical conditions including nOH. The primary objective of this trial is to study the safety, tolerability and efficacy of TD-9855 in patients with symptomatic nOH in comparison with a placebo over a double-blind, randomized period of 6 weeks following an open label (OL) treatment of 16 weeks. We will enroll patients diagnosed with primary autonomic failures (MSA, PD, or PAF) and symptomatic nOH. The study will be conducted in several closely monitored visits. Monetary compensation will be provided.

Inclusion Criteria

• Male or female and at least 30 years old
• Must meet the diagnostic criteria of nOH, as demonstrated by a sustained reduction in BP of ≥20 mmHg (systolic) or ≥10 mmHg (diastolic) within 3 min of being tilted up
• Must score at least a 4 on the orthostatic hypotension questionnaire question 1
• Plasma NE levels ≥ 100 pg/mL after being in seated position for 30 minutes

Exclusion Criteria

• A known intolerance to other NRIs or serotonin norepinephrine reuptake inhibitors (SNRIs).
• Current use of concomitant antihypertensive medication for the treatment of essential hypertension
• A Montreal Cognitive Assessment (MoCA) score ≤23

The Principal Investigator for this study at Beth Israel Deaconess Medical Center is Dr. Roy Freeman, MD

Contact: Sharika Rajan, srajan@bidmc.harvard.edu

The purpose of this project is to look at how mitochondria (a part of the cells that make up your body) work specifically in blood cells of people who have Parkinson disease compared to people who do not have PD. The mitochondria are the part of the cells that provide energy, and measuring mitochondria dysfunction in people with PD may provide information about which patients would respond to treatments and drugs in future trials. The Principal Investigator for this study at Beth Israel Deaconess Medical Center is David K. Simon, MD, PhD. If you are interested, this study would involve a one-time blood draw, with the possibility of coming in for a second blood draw.
For this research study, we are asking people to participate who have Parkinson’s Disease and a known genetic mutation in the “GBA” or “Gaucher’s” gene identified by genetic testing. People who participate will be compensated with a $100 visa gift card to cover transportation costs and as a thank you for participating.

Inclusion Criteria

1. Diagnosis of idiopathic PD by a neurologist with expertise in movement disorders.
2. Diagnosis with a known a genetic mutation in the GBA gene.

Exclusion Criteria

1. Diagnosis of diabetes
2. Neuroleptic treatment within the past 90 days
3. Clinically significant orthostatic hypotension
4. Inability to provide informed consent
5. Diagnosis of idiopathic PD without a known genetic mutation of the GBA gene or without having the risk of a genetic mutation in the GBA gene.

Contact: Stephanie Burrows, email or 617-667-9885

The purpose of this study is to learn more about walking problems in people with Huntington’s disease. The study will involve participating in a single study visit with an optional follow up at Beth Israel Deaconess Medical Center. Researchers will collect a neurological history and exam from participants and measure walking using a special mat equipped with electronic sensors. Parking is compensated and $25 is paid for the completion of each study visit.

Inclusion Criteria:

Enrolling patients with Huntington’s disease, between the ages of 18 and 85.

Exclusion Criteria:

Participants should be otherwise healthy, not recently hospitalized, and without heart or lung conditions that would prevent walking for ten minutes.

Contact: Brennan Cook, bcook1@bidmc.harvard.edu or 617-667-4746

The Dystonia Coalition is an international collaboration of medical researchers and patient advocacy groups with a mission to advance the pace of clinical and translational research in the dystonias to find better treatments and a cure. To participate you must be at least 18 years old and be diagnosed with one of the following primary dystonias:

• Focal dystonia, including cranial dystonia/Meige Syndrome (including blepharospasm)
• Jaw or tongue dystonia
• Laryngeal dystonia (including spasmodic dysphonia) with diagnosis confirmed by nasolaryngoscopy
• Cervical dystonia (spasmodic torticollis)
• Limb dystonia (including writer’s cramp and musician’s dystonia)
• Segmental dystonia
• Generalized dystonia
• Hemi-dystonia

Your last injection of botulinum toxin should be at least two months prior to study visit. You must not have evidence of a secondary cause for dystonia. During the study visit, you will answer questions about your medical and family history, have a neurological exam that will be videotaped, and donate about four tablespoons of blood. The entire visit takes about 60 to 90 minutes. Parking will be compensated.

Contact: Stephanie Burrows, sburrows@bidmc.harvard.edu or 617-667-9885

We encourage individuals with Parkinson's disease who have experienced episodes of freezing, or the feeling of being "stuck" before or during walking or turning, to participate in this study. Participants will be asked to complete a baseline assessment of walking, balance and mental function. They will then be randomized to receive ten sessions of either real or sham (i.e., placebo) transcranial direct current stimulation (tDCS) over the course of two weeks. Three follow-up assessments will then be completed over the next ten weeks to measure the effects of the tDCS intervention. The entire study will take approximately 14 weeks to complete and a stipend of up to $500 will be provided. Parking is provided and transportation may be arranged if needed.

Inclusion Criteria:

• Between the ages of 40 and 89
• Have experienced freezing episodes as a result of Parkinson's disease

Exclusion Criteria:

• History of brain surgery (including that for deep brain stimulation), stroke or other neurodegenerative disease
• Severe depression or other major psychiatric diagnosis
• Inability to walk without assistance
• Uncontrolled hypertension or Diabetes Mellitus
• Certain medications, including benzodiazepines

Contact: Rachel Harrison, RachelHarrison@hsl.harvard.edu

People with Parkinson's Disease (PD) often have problems controlling shaking, stiffness and slowness of movement. Researchers have found that a protein called alpha-synuclein collects in the brain and increases symptoms of PD. Nilotinib may reduce the build-up of alpha-synuclein and may help to relieve PD symptoms. The drug involved in the study, Nilotinib, is investigational for use in PD. The purpose of this study is to determine if Nilotinib is safe and tolerable in patients with PD and to learn if the drug has the possibility to treat PD symptoms.

Note: This study is now fully enrolled. We are no longer recruiting new participants for this trial. Thank you for your interest.

Contact: Stephanie Burrows, 617-667-9885, or sburrows@bidmc.harvard.edu

This is a study for people recently diagnosed with Parkinson’s disease. It is a two-year study being conducted to look at how well a new IV drug, RO7046015, works versus placebo. The drug works by binding to a protein called alpha-synuclein, with the hope of slowing the progression of early Parkinson’s Disease.

Note: This study is now fully enrolled. We are no longer recruiting new participants for this trial. Thank you for your interest.

This is a study for people recently diagnosed with Huntington’s disease. It is a 2-year study being conducted to look at how well a new IV drug, VX15, works versus placebo. The drug works by binding to and blocking a molecule that may cause brain inflammation that has been shown to affect the thinking, movement, and behaviors that impact the daily activities of individuals with Huntington’s disease.

Inclusion Criteria:

• Age 21 years or older thought to be in the early stages of HD
• Clinically diagnosed with HD
• Able to undergo brain scans (MRI and PET)
• Able to commit to once/month study visits

Exclusion Criteria:

• Are currently or have recently (within last 30 days) participated in an investigational drug or device study
• Have had previous neurosurgery for HD or other movement disorders
• If female, are pregnant or breastfeeding
• Body weight above 348 lbs

Note: This study is now fully enrolled. We are no longer recruiting new participants for this trial. Thank you for your interest.

Contact: Jaqueline Fung, jfung@bidmc.harvard.edu or (617) 975-7636