| Division of Neurology |
Clinical Trials
Clinical trials projects being conducted in the Division of Pediatric Neurology:
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An Open-Label, Multicenter Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Three Dosing Regimens of Oral AT2220 in Patients with Pompe Disease. PI: Priya Kishnani, MD. Co-Investigator: Edward Smith, MD.
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Is Autologous Umbilical Cord Blood Infusion Beneficial in Children with Cerebral Palsy: A Randomized, Blinded, Placebo-Controlled, Crossover Study. PI: Joanne Kurtzberg, MD. Co-Investigator: Mohamad Mikati, MD.
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A placebo-controlled, escalating dose, multiple dose study to evaluate the safety, tolerability, and pharmacokinetics of Pregabalin in pediatric patients with partial onset seizures. PI: Mohamad Mikati, MD. Co-Investigators: William Gallentine, DO; Edward Smith, MD.
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A 12-month open label extension study evaluating the safety and tolerability of flexible doses of Pregabalin in pediatric patients with partial onset seizures. PI: Mohamad Mikati, MD. Co-Investigators: William Gallentine, DO; Edward Smith, MD.
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A Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Lacosamide (LCM) Oral Solution (Syrup) as Adjunctive Therapy in Children with Partial-Onset Seizures. PI: Mohamad Mikati, MD. Co-Investigators: William Gallentine, DO; Edward Smith, MD.
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An Open-Label Extension Study to Determine Safety, Tolerability, and Efficacy of Long-TermOral Lacosamide (LCM) as Adjunctive Therapy in Children with Partial-Onset Seizures. PI: Mohamad Mikati, MD. Co-Investigators: William Gallentine, DO; Edward Smith, MD.
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Possible role of Costamere integrity and Ankyrins in muscular dystrophy. PI: Edward Smith, MD.
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Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I). PI: Swoboda. Co-Investigator: Edward Smith, MD.
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A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy. PI: Stephanie Wechsler, MD. Co-Investigator: Edward Smith, MD.




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