Ataluren in Patients with Duchenne Muscular Dystrophy (PTC124-GD-041-DMD) – NOW ENROLLING

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension (PTC124-GD-041-DMD)

Protocol, purpose and description: This study is a long-term study of ataluren in patients with nonsense mutation Duchenne muscular dystrophy

CHKD physicians involved: Crystal Proud, MD,  Thomas Enlow, MD,  L. Matthew Frank, MD,  Svinder Toor, MD, Sarah Chagnon, MD,  Ryan Williams, MD,  Matthew Warren, MPA, PA-C,  Jennifer Kalb, MPA, PA-C

Learn more about this study.

Or contact Terrie Conklin at (757) 668-9356 or Terrie.Conklin@chkd.org

Intravenous Lacosamide (EP0060) – NOW ENROLLING

A multicenter, open-label study to investigate the safety and tolerability of intravenous lacosamide in children (≥4 to <17 years of age) with epilepsy

Protocol, purpose and description: Study to Investigate Safety and Tolerability of Intravenous Lacosamide in Children

CHKD physicians involved:  Crystal Proud, MD,  Thomas Enlow, MD,  L. Matthew Frank, MD,  Svinder Toor, MD,  Ryan Williams, MD,  Matthew Warren, MPA, PA-C,  Jennifer Kalb, MPA, PA-C

Learn more about this study.

Or contact Terrie Conklin at (757) 668-9356 or Terrie.Conklin@chkd.org

A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy (BRAVE) – NOW ENROLLING

A Multi-center, Randomized, Parallel-group, Double-blind, Multiple-dose, Placebo-controlled study to assess the efficacy and safety of MNK‑1411 in male subjects 4 to 8 years of age with Duchenne Muscular Dystrophy (MNK14112096)

Protocol, purpose and description: This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD).

CHKD physicians involved:Crystal Proud, MD, L. Matthew Frank, MD, Svinder Toor, MD, Sarah Chagnon, MD, Ryan Williams, MD, Thomas Enlow, MD, Matthew Warren, MPA, PA-C, Jennifer Kalb, MPA, PA-C

Learn more about this clinical trial.

Or contact Terrie Conklin at (757) 668-9356 or Terrie.Conklin@chkd.org

Expanded Access Program to Provide Treatment With Pitolisant to Adult Patients in the U.S. With Excessive Daytime Sleepiness Associated With Narcolepsy With or Without Cataplexy – LIMITED ENROLLMENT

An Open Label Expanded Access Program Intended to Provide Treatment with HBS-101 (Pitolisant) to Adult Patients in the U.S. with Excessive Daytime Sleepiness Associated with Narcolepsy with or without Cataplexy (HBS-101-CL-001)

Protocol, purpose and description: This EAP will be open to provide access to treatment with pitolisant while a U.S. New Drug Application (NDA) is being prepared and submitted for review for marketing approval. This program will be open to adult patients in the U.S. with Excessive Daytime Sleepiness (EDS) associated with narcolepsy, with or without cataplexy. Pitolisant will be made available through treating physicians participating in the program.

CHKD physicians involved:Michael Strunc, MD

Learn more about this clinical trial.

Or contact Jennifer Parrott at (757) 668-8244 or Jennifer.Parrott@chkd.org

Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures –NOW ENROLLING  

A Phase 2, Open-label, Dose-finding Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Patients with Treatment-Resistant Childhood Absence Seizures (INS011-17-103)

Protocol, purpose and description: The primary purpose of this study is to assess the efficacy of Cannabidiol Oral Solution in the treatment of pediatric participants with treatment-resistant childhood absence seizures. This study will also assess safety, tolerability and pharmacokinetics of Cannabidiol Oral Solution, and any improvement in qualitative assessments of participant status over the duration of the study in pediatric participants with treatment-resistant childhood absence seizures. The study will include a 4-week Screening Period, a 5 or 10 day Titration Period (depending study Cohort), a 4-week Treatment Period followed by 5-day Tapering for doses >20 mg/kg/day and a 4-week Follow-up Period.

CHKD physicians involved:L. Matthew Frank, MD; Crystal Proud, MD; Svinder Toor, MD; Sarah Chagnon, MD; Ryan Williams, MD, Thomas Enlow, MD; Matthew Warren, MPA, PA-C; Jennifer Kalb, MPA, PA-C

Learn more about this clinical trial.

Or contact Terrie Conklin at (757) 668-9356 or Terrie.Conklin@chkd.org

Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Participants With Treatment-Resistant Childhood Absence Seizures

A Multicenter, Open-Label, Flexible Dose Study to Assess the Long-Term Safety of Pharmaceutical Grade Synthetic Cannabidiol Oral Solution in Pediatric Patients with Treatment-Resistant Childhood Absence Seizures (INS011-17-113)

Protocol, purpose and description: The primary purpose of this study is to assess the long-term safety and tolerability of Cannabidiol Oral Solution (CBD) in pediatric participants with treatment-resistant childhood absence seizures.

CHKD physicians involved:L. Matthew Frank, MD; Crystal Proud, MD; Svinder Toor, MD; Sarah Chagnon, MD; Ryan Williams, MD; Thomas Enlow, MD; Matthew Warren, MPA, PA-C; Jennifer Kalb, MPA, PA-C

Learn more about this clinical trial.

Or contact Terrie Conklin at (757) 668-9356 or Terrie.Conklin@chkd.org