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بالأسفل التجارب السريرية الحالية.

غربل قائمة الدراسات هذه بالموقع والحالة وغيرها.

1. 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.

2. 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.

3. 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.

4. 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:

5. 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).

6. 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.

7. 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.

8. A Study to Evaluate Caregiver Connections via Technology for Patients with Alzheimer's and Other Types of Dementia

   Rochester, Minn.

   The purpose of this study is to optimize profile questionnaire and matching algorithm developed in Phase I and implement in final website design, and to determine if algorithmically matched participants have statistically significant increase in match satisfaction and self-reported sense of resiliency and quality of life over randomly matched caregivers.

9. A Study of Mood Symptoms in Epilepsy Patients

   Rochester, Minn.

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

10. Phase 2 Study of TAK-341 for Multiple System Atrophy

    Rochester, Minn.

    The purpose of this study is to evaluate the effectiveness of TAK-341 versus placebo, as measured by the change from baseline to Week 52 on l Unified Multiple System Atrophy Rating Scale (UMSARS) Part I, minus the sexual function item, with collapse of the normal and mild ratings on each item.