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

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

-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

Age between 18 and 90 years old

Have had a positive COVID test within the last two years

Does not have any symptoms lasting more than 6 weeks post infection

1. >25yrs of age.

2. Diagnosis of HD with CAG repeat > 36

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

1.Individuals ≥ 18 years of age at the time of informed consent.

2. Confirmed diagnosis of Wilson disease.

3. Stable Wilson Disease with no medication or dose changes for at least 6 months at Screening.

4. Ongoing restriction of high copper containing foods for at least 6 months at Screening and continued through study participation.

• 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

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

• 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

Participants diagnosed with Parkinson’s Disease (PD) and previously implanted with a subthalamic nucleus deep brain stimulation (STN-DBS) System.

Subject must have moderate/severe freezing of gait and/or tremor considered as a significant source of impairment in the management of their Parkinson's disease.

People 55 years and older with 1) chronic insomnia (trouble falling asleep or staying asleep) or 2) good sleepers.

Exclusion criteria include:

1) Sleep disorders other than insomnia;

2) Habitual bedtime before 9pm or morning awakening before 5am;

3) History of neurological disorders;

4) History of psychiatric disorders (e.g., depression, anxiety);

5) Cognitive impairment;

6) Diabetes;

7) Current, or use within the past month, of psychoactive, hypnotic, stimulant or analgesic medications (except occasionally);

8) Use of Beta-blockers

9) Hormone replacement therapy;

10) Shift work or other types of self-imposed irregular sleep schedules.