Clinical Trials

You may be eligible to take part in the research component of the NBTIDB if you are either a new or returning patient, over the age of 18, who is being seen by one of the clinicians at the NBTI and are or will be entered into the NBTIDB, or a patient who is not coming to the NBTI for evaluation, but would still like to participate in the NBTIDB.

• Diagnosis of a movement disorder • Male or female 18 years of age or older when diagnosed • Ability to provide informed consent

At least 18 years of age

- Had a stroke more than 6 months ago

- No large impairment in vision (glasses), memory, language or concentration

- Not currently participating in another research study on the arm

• Age ≥ 45 years.
• Clinical diagnosis of ischemic stroke + brain imaging to rule out hemorrhagic stroke.
• Modified Rankin Scale (MRS) score ≤ 4.
• Ability to be randomized within 3 to 120 days after stroke onset.
• ESUS (embolic stroke of underdetermined source).

• History of atrial fibrillation
• Clear indication for treatment-dose anticoagulant therapy, such as venous thromboembolism or a mechanical heart valve.
• Need for antiplatelet agent other than aspirin, such as clopidogrel after implantation of a coronary artery stent.
• History of spontaneous intracranial hemorrhage.
• Chronic kidney disease with serum creatinine ≥2.5 mg/dL.

Inclusion Criteria:

• Aged ≥ 21 years at the date of consent.
• History of chronic, stable heart failure with reduced ejection fraction with left ventricular ejection fraction (LVEF) ≤ 45% determined by echocardiography, radionuclide angiography, left ventriculography, or cardiac magnetic resonance imaging, within the year prior to enrollment.
• Central sleep apnea, defined using as an apnea-hypopnea index (AHI) > 15/h with ≥ 50% central events (apnea and hypopneas) and a central AHI ≥ 10/h or obstructive apnea index (OAI) < 20%
• New York Heart Association (NYHA) Class III or IV, or NYHA Class II with ≥ 1 hospitalization for HF in the last 24 months.
• Treatment with stable, optimized guideline-directed medical therapies (GDMT) according to applicable guidelines in the U.S. and Canada, where stable is defined as the addition of no new class of disease-modifying drug for ≥ 30 days prior to randomization.
• In the investigator's opinion, willing and able to comply with all study requirements
• Able to fully understand study information and sign an Institutional Review Board (IRB) approved informed consent

Exclusion Criteria:

• Current positive airway pressure use of diagnosis of Obstructive Sleep Apnea (OSA).
• Oxygen saturation < 90% at rest during the day.
• Oxygen saturation < 88% for > 5 continuous minutes during sleep unaccompanied by respiratory events.
• Chronic daytime or nighttime use of supplemental oxygen.
• Current smoker or bed partner that smokes in the bedroom.
• Severe pulmonary disease requiring continuous home oxygen therapy or the continuous or frequent intermittent use of oral steroids or documented severe chronic obstructive pulmonary disease (COPD) with forced expiratory volume in 1 second (FEV1) < 50%.
• Body mass index (BMI) > 35 kg/m2.
• Cardiac surgery, percutaneous coronary intervention, myocardial infarction or unstable angina within the previous 3 months.
• Transient ischemic attack or stroke within the previous 3 months.
• Cardiac resynchronization therapy implantation scheduled or performed within 3 months prior to randomization.
• Primary hemodynamically-significant uncorrected valvular heart disease (obstructive or regurgitant) or any valvular disease expected to require surgery during the trial
• Acute myocarditis/pericarditis or other cause of potentially reversible cardiomyopathy (e.g., post-partum cardiomyopathy, tachycardia-induced cardiomyopathy), within the previous 6 months.
• End-stage (Stage D) heart failure (HF) requiring continuous outpatient intravenous (IV) inotropic therapy, placement of ventricular assist device, listing for cardiac transplantation, or end-of-life care (e.g. hospice care).
• Pregnancy or of child bearing potential without a negative pregnancy test within 10 days prior to enrollment.
• Life expectancy < 1 year for diseases unrelated to chronic HF.
• Enrolled or planning to enroll in another study that may conflict with protocol requirements or confound subject results in this trial.

2. Decision made to prescribe Edaravone prior to screening

3. Participant will likely be able to obtain commercial Edaravone and likely to complete 6 cycles of treatment, per site investigator estimation

4. Participant either naïve to Edaravone or who did not receive any Edaravone dose within 28 days prior to screening

5. Participant with a contraindication to Edaravone may not participate

6. Participant is participating in an interventional clinical trial may not participate

1. A clinical diagnosis of El Escorial of suspected, possible, probable, or definite ALS.

2. Other motor neuron disorders, including but not limited to spinobulbar muscular atrophy (SBMA, Kennedy’s disease), Spinal Muscular Atrophy (SMA), Primary Lateral Sclerosis (PLS), Progressive Muscular Atrophy (PMA), and Progressive Bulbar Palsy (PBP).

3. Excluded are any disease that does not meet criteria for any motor neuron disorder

Primary Inclusion Criteria:

• Male or female subjects ≥18 years of age
• Clinical diagnosis of ischemic stroke involving cerebral cortex
• Onset of stroke symptoms must have occurred 18 to 36 hours prior to the planned start of administration of the investigational product
• Occurrence of a moderate to moderately severe stroke with a persistent neurologic deficit documented by a NIHSS score of 8 to 20 (inclusive) that does not change by ≥4 points during the initial screening period
• A mRS score of 0 or 1 prior to the onset of symptoms of the current stroke

Primary Exclusion Criteria:

• Presence of a lacunar or a brainstem infarct
• Comatose state
• Brain hemorrhage
• Major neurological event such as stroke or clinically significant head trauma within 6 months of enrollment into the study

2. Diagnosis of PD w/in 7yrs without motor fluctuationsor dyskinesia.

3. Not on any medication for PD or on stable therapy for 8weeks prior to screening.

-Diagnosis of Parkinson's disease for less than 3 years

-Cannot be treated with any PD medication

Inclusion:

Either possible, probable, or definite ALS,predominantly lower motor neuron disease Predominantly upper motor neuron disease, orbulbar

With or without cognitive involvement

Willing to participate

On no experimental treatment

Ages 18 - 85

No prior exposure to Radicava

On a stable dose of riluzole for 30 days or offriluzole

Male or female

Females of childbearing age must usecontraception

Exclusion:

Unstable medical illness

Abnormal liver function (>2x ULN)

Unlikely to survive for at least 26 weeks

1. 40-75 years of age.

2. Diagnosis of PD with H&Y 3-4.

3. On stable PD therapy for 8 weeks prior to baseline.

4. At least 1 GBA gene mutation.

The basic eligibility criteria include:

2. FVC (breathing test) > 50%

3. If on riluzole, must be on a stable dose for 30 days. Must not start riluzole during the study.

4. If on radicava, must be on a stable dose for 30 days. Must not start riluzole during the study.

5. Must be able to swallow for the next 6 months

6. No history stem cell treatment

7. No history of cancer within the last 5 years

Inclusion Criteria:

• Meet 4 of 11 American College of Rheumatology (ACR) criteria for systemic lupus (SLE) or meet 3 of 11 ACR criteria for SLE and meet SLICC criteria
• 18 years or older
• Positive ANA or positive dsDNA

Exclusion Criteria:

• Currently pregnant or nursing
• Active or chronic infections (such as hepatitis, pneumonia, pyelonephritis, HIV, sepsis)
• Diagnosed with another chronic autoimmune disease or chronic neurodegenerative condition
• CKD > 2 and/or GFR < 60
• AST and/or ALT ≥ 3 times the upper limit of normal
• Current electrolyte disturbance (hyponatremia or hypernatremia)
• Current signs or symptoms of acute systemic vascultis

2. Being referred for evaluation for glenohumeral shoulder injections for management of shoulder pain

3. Able to utilize written, typed or verbal communication, independently or with the assistance of a caregiver

4. Able to understand study procedures, answer questionnaires, and provide informed consent

1. >25yrs of age.

2. Diagnosis of HD with CAG repeat > 36

• Inclusion Criteria:

  • Ability and acceptance to provide written informed consent of the participant or legal guardian. Participants ≥ 16 and < 18 years of age will also need to provide written assent.
  • A confirmed clinical diagnosis of Delayed Sleep-Wake Phase Disorder (DSWPD).
  • Men or women between 16 - 65 years, inclusive.
  • Body Mass Index (BMI) of ≥ 18 and ≤ 30 kg/m^2.

• Exclusion Criteria:
• History of psychiatric disorders within 12 months.
• Major surgery, trauma, illness, general anesthesia, or immobility for 3 or more days within the last 30 days.
• Pregnancy, recent pregnancy (within 6 weeks), or women who are breastfeeding.
• A positive test for substances of abuse

You might be a candidate for this study if:

• You are 40 - 75 years old
• You have been diagnosed with Parkinson disease within the last two years
• You have only mild symptoms of PD, like slowness of movement, muscle stiffness / rigidity, or tremor / shaking

There are additional eligibility criteria that will be discussed with you.

Some of the BASIC, but not full, list of eligibility criteria are below:

2. Symptom onset was no more than 15 years prior to baseline

3. Ability to independently walk with or without an assistive device (e.g., walker) at the baseline evaluation

4. Some bulbar symptoms (dysarthria, dysphagia, drooling or pseudobulbar affect)

5. UMN symptoms and signs in a region other than the legs

• Age of 40 - 80 years
• Ability to have a brain MRI scan
• Clinical diagnosis of Parkinson disease at least 5 years ago, or
• Clinical diagnosis of multiple systems atrophy (parkinsonian type), or
• Clinical diagnosis of progressive supranuclear palsy

The BASIC, but not full, list of eligibility criteria are below:

2. Age 18 years or older

3. Participant must be unable to participate in the HEALEY ALS Platform Trial

4. Participants have established care with a physician at the specialized ALS center involved in the protocol and will maintain this clinical care throughout the duration of the EAP

5. Participants must have the ability to swallow whole pills without crushing or chewing

6. Participants who are enrolled in another EAP or a Clinical Trial

1. 18 years or older

2. Must have one of the following SOD1 mutations confirmed by a central reader. Please contact the study coordinator for a complete list.

3. If a SOD1 mutation is not listed, your mutation will be adjudicated by a Mutation Adjudication Committee.

4. Plasma neurofilament (NfL) level less than 44 pg/mL during Screening

5. Clinically pre-symptomatic for ALS (i.e., must not have clinically manifested ALS) at Part A Screening Visit

6. History or positive test result at Screening for HIV, Hep-B, or Hep-C

2. Able to ambulate short distances (at least 3 m) with or without walking aid.

3. Able to grasp a pen and able to write

4. For the dedicated C9orf72 group only (10-15 participants) presence of a C9orf72 repeat expansion.

5. Having a study partner who agrees to participate in the study and who is intellectually, visually, and verbally capable, and fluent in, and able to read, the language in which study assessments are administered.

• Diagnosis of PD for at least 6 months to maximum 3 years at screening and between 50-85 years of age

• On symptomatic PD medication for at least 6 months, with a stable dose for 3 months prior to baseline

• No dyskinesisa or motor fluctuations (i.e. MDS-UPDRS Part IV = 0)

1.≥39 to ≤70 years of age at signing of informed consent

2. Diagnosis of clinically established PD

3. Marked levodopa responsiveness at screening per investigator’s judgment

4. A minimum of 3 years and a maximum of 18 years from time of PD diagnosis to the date of screening

5. Receiving optimized and stable PD medical therapy for ≥1 month prior to screening or demonstrated intolerance to PD medications per investigator’s judgment in agreement with the medical monitor

6. ≥3 hours of average daily OFF-time assessed within 3 months of screening by PD diary or per investigator’s judgment

- The subject is ≥35 and ≤80 years of age at the time of informed consent.

- The subject has a clinical diagnosis of PD for at least 1 year and for no longer than

7 years before initiation of screening

- The subject has a modified Hoehn and Yahr score ≤2.5.

- The subject is receiving symptomatic treatment for PD.

- The subject has a known GBA-PD risk-associated variant