神经科

以下为当前的临床试验。

按院区、状态和其他条件筛选该研究列表。

1. The Neurobiology of Two Distinct Types of Progressive Apraxia of Speech

   Rochester, Minn.

   The purpose of this study is to identify and distinguish two different types of Progressive Apraxia of Speech through clinical imaging and testing.

2. A Study to Evaluate Newly-diagnosed Idiopathic Generalized Epilepsy

   Rochester, Minn.

   The purpose of this study is to identify clinical characteristics and biomarkers predictive of disease outcome, progression, and treatment response in participants with idiopathic generalized epilepsy (IGE).

3. Posterior Fossa Decompression with or without Duraplasty for Chiari Type I Malformation with Syringomyelia

   Rochester, Minn.

   The purpose of this study is to determine whether a posterior fossa decompression or a posterior fossa decompression with duraplasty results in better patient outcomes with fewer complications and improved quality of life in those who have Chiari malformation type I and syringomyelia.

4. A Study to Investigate the Safety and Tolerability of Single and Multiple Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome

   Rochester, Minn.

   The purpose of this study is to evaluate the safety and tolerability of single-ascending doses of STK-001 in patients with Dravet Syndrome.

5. A Study to Evaluate Traditional Versus Early Aggressive Therapy for Multiple Sclerosis Patients

   Rochester, Minn.

   The purpose of this study is to evaluate, jointly and independently among patients deemed at higher risk vs. lower risk for disability accumulation, whether an "early aggressive" therapy approach, versus starting with a traditional, first-line therapy, influences the intermediate-term risk of disability, and to evaluate if, among patients deemed at lower risk for disability who start on first-line MS therapies but experience breakthrough disease, those who switch to a higher-efficacy versus a new first-line therapy have different intermediate-term risk of disability.FDA-approved multiple sclerosis (MS) disease-modifying therapies (DMTs) target the relapsing phase of MS but have minimal impact once the progressive phase has begun. It is unclear if, in the relapsing phase, there is an advantage of early aggressive therapy with respect to preventing long-term disability. The infectious risks and other complications associated with higher-efficacy treatments highlight the need to quantify their effectiveness in preventing disability. The TRaditional versus Early Aggressive Therapy for MS (TREAT-MS) trial is a pragmatic, randomized controlled trial that has two primary aims:

6. Natural History Study Protocol in PMM2-CDG (CDG-Ia)

   Rochester, Minn.

   Clinical and Basic Investigations into Phosphomannomutase deficiency (PMM2-CDG) This is a natural history (observational) protocol designed to collect clinical and biological information in patients with PMM2-CDG (CDG-Ia). Data from this natural history study will be used to determine a set of clinical and biological parameters that will be used for primary and secondary endpoints in a later clinical trial with a new chemical entity, Lipo-M1P.

7. HEALEY ALS Platform Trial

   Rochester, Minn., Jacksonville, Fla., Scottsdale/Phoenix, Ariz.

   The purpose of this study is to evaluate the safety and effectiveness of investigational products for the treatment of ALS.

8. A Study of Mood Symptoms in Epilepsy Patients

   Rochester, Minn.

   The purpose of this study is to track neuropsychiatric symptoms in patients with epilepsy.

9. Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma

   Rochester, Minn.

   This phase II trial studies how well reduced doses of radiation therapy to the brain and spine (craniospinal) and chemotherapy work in treating patients with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma. Recent studies using chemotherapy and radiation therapy have been shown to be effective in treating patients with WNT-driven medulloblastoma. However, there is a concern about the late side effects of treatment, such as learning difficulties, lower amounts of hormones, or other problems in performing daily activities. Radiotherapy uses high-energy radiation from x-rays to kill cancer cells and shrink tumors. Drugs used in chemotherapy, such as cisplatin, vincristine sulfate, cyclophosphamide and lomustine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving reduced craniospinal radiation therapy and chemotherapy may kill tumor cells and may also reduce the late side effects of treatment.

10. Submandibular Gland Needle Core Biopsy and Skin Punch Biopsy as Tissue Biomarkers for the Diagnosis and Monitoring of Disease Progression of Parkinson’s Disease and REM Sleep Behavior Disorder

    Scottsdale/Phoenix, Ariz.

    Determine 1) the magnitude of SMG and skin a-syn density change over time, 2) the

    improvement in adequate tissue acquisition doing bilateral SMG biopsies, 3) any laterality differences between

    SMG gland a-syn density, 4) correlate a-syn density and changes in density between SMG and skin biopsies

    Specific Aim 1. Determine, with bilateral submandibular gland (SMG) biopsies in 30 clinically

    diagnosed subjects with Parkinson’s disease (PD) and 20 clinically probable REM sleep behavior

    disorder (RBD) subjects whether bilateral biopsies improve the rate of acquisition of adequate

    glandular tissue, whether alpha-synuclein (a-syn) SMG pathology correlates with clinical severity

    and/or disease duration, and whether there is a left-right difference in the density of a-syn pathology.

    Specific Aim 2. Determine, with a second SMG biopsy in previously biopsied PD and RBD subjects,

    whether a-syn pathology density increases, decreases, or remains the same after a 1.5-2 year time

    interval and whether there is a correlation between a-syn SMG pathology and clinical and/or disease

    progression.

    Specific Aim 3. Determine whether a-syn pathology density in skin punch biopsies correlates

    with clinical measures and increases, decreases, or remains the same after a 1.5-2 year time

    interval, and whether the density and changes over time correlate with those found in SMG

    biopsies.

以下为当前的临床试验。

按院区、状态和其他条件筛选该研究列表。

1. The Neurobiology of Two Distinct Types of Progressive Apraxia of Speech

   Rochester, Minn.

   The purpose of this study is to identify and distinguish two different types of Progressive Apraxia of Speech through clinical imaging and testing.

2. A Study to Evaluate Newly-diagnosed Idiopathic Generalized Epilepsy

   Rochester, Minn.

   The purpose of this study is to identify clinical characteristics and biomarkers predictive of disease outcome, progression, and treatment response in participants with idiopathic generalized epilepsy (IGE).

3. Posterior Fossa Decompression with or without Duraplasty for Chiari Type I Malformation with Syringomyelia

   Rochester, Minn.

   The purpose of this study is to determine whether a posterior fossa decompression or a posterior fossa decompression with duraplasty results in better patient outcomes with fewer complications and improved quality of life in those who have Chiari malformation type I and syringomyelia.

4. A Study to Investigate the Safety and Tolerability of Single and Multiple Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome

   Rochester, Minn.

   The purpose of this study is to evaluate the safety and tolerability of single-ascending doses of STK-001 in patients with Dravet Syndrome.

5. A Study to Evaluate Traditional Versus Early Aggressive Therapy for Multiple Sclerosis Patients

   Rochester, Minn.

   The purpose of this study is to evaluate, jointly and independently among patients deemed at higher risk vs. lower risk for disability accumulation, whether an "early aggressive" therapy approach, versus starting with a traditional, first-line therapy, influences the intermediate-term risk of disability, and to evaluate if, among patients deemed at lower risk for disability who start on first-line MS therapies but experience breakthrough disease, those who switch to a higher-efficacy versus a new first-line therapy have different intermediate-term risk of disability.FDA-approved multiple sclerosis (MS) disease-modifying therapies (DMTs) target the relapsing phase of MS but have minimal impact once the progressive phase has begun. It is unclear if, in the relapsing phase, there is an advantage of early aggressive therapy with respect to preventing long-term disability. The infectious risks and other complications associated with higher-efficacy treatments highlight the need to quantify their effectiveness in preventing disability. The TRaditional versus Early Aggressive Therapy for MS (TREAT-MS) trial is a pragmatic, randomized controlled trial that has two primary aims:

6. Natural History Study Protocol in PMM2-CDG (CDG-Ia)

   Rochester, Minn.

   Clinical and Basic Investigations into Phosphomannomutase deficiency (PMM2-CDG) This is a natural history (observational) protocol designed to collect clinical and biological information in patients with PMM2-CDG (CDG-Ia). Data from this natural history study will be used to determine a set of clinical and biological parameters that will be used for primary and secondary endpoints in a later clinical trial with a new chemical entity, Lipo-M1P.

7. HEALEY ALS Platform Trial

   Rochester, Minn., Jacksonville, Fla., Scottsdale/Phoenix, Ariz.

   The purpose of this study is to evaluate the safety and effectiveness of investigational products for the treatment of ALS.

8. A Study of Mood Symptoms in Epilepsy Patients

   Rochester, Minn.

   The purpose of this study is to track neuropsychiatric symptoms in patients with epilepsy.

9. Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma

   Rochester, Minn.

   This phase II trial studies how well reduced doses of radiation therapy to the brain and spine (craniospinal) and chemotherapy work in treating patients with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma. Recent studies using chemotherapy and radiation therapy have been shown to be effective in treating patients with WNT-driven medulloblastoma. However, there is a concern about the late side effects of treatment, such as learning difficulties, lower amounts of hormones, or other problems in performing daily activities. Radiotherapy uses high-energy radiation from x-rays to kill cancer cells and shrink tumors. Drugs used in chemotherapy, such as cisplatin, vincristine sulfate, cyclophosphamide and lomustine, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving reduced craniospinal radiation therapy and chemotherapy may kill tumor cells and may also reduce the late side effects of treatment.

10. Submandibular Gland Needle Core Biopsy and Skin Punch Biopsy as Tissue Biomarkers for the Diagnosis and Monitoring of Disease Progression of Parkinson’s Disease and REM Sleep Behavior Disorder

    Scottsdale/Phoenix, Ariz.

    Determine 1) the magnitude of SMG and skin a-syn density change over time, 2) the

    improvement in adequate tissue acquisition doing bilateral SMG biopsies, 3) any laterality differences between

    SMG gland a-syn density, 4) correlate a-syn density and changes in density between SMG and skin biopsies

    Specific Aim 1. Determine, with bilateral submandibular gland (SMG) biopsies in 30 clinically

    diagnosed subjects with Parkinson’s disease (PD) and 20 clinically probable REM sleep behavior

    disorder (RBD) subjects whether bilateral biopsies improve the rate of acquisition of adequate

    glandular tissue, whether alpha-synuclein (a-syn) SMG pathology correlates with clinical severity

    and/or disease duration, and whether there is a left-right difference in the density of a-syn pathology.

    Specific Aim 2. Determine, with a second SMG biopsy in previously biopsied PD and RBD subjects,

    whether a-syn pathology density increases, decreases, or remains the same after a 1.5-2 year time

    interval and whether there is a correlation between a-syn SMG pathology and clinical and/or disease

    progression.

    Specific Aim 3. Determine whether a-syn pathology density in skin punch biopsies correlates

    with clinical measures and increases, decreases, or remains the same after a 1.5-2 year time

    interval, and whether the density and changes over time correlate with those found in SMG

    biopsies.