81
Participants
Start Date
May 31, 2023
Primary Completion Date
July 31, 2027
Study Completion Date
July 31, 2027
BBP-418 (ribitol)
The BBP-418 drug product is provided as Granules for Oral solution consisting of BBP-418 drug substance and silicon dioxide in a multilaminate sachet with a foil barrier. Silicon dioxide is generally regarded as safe and listed in the FDA IIAD. Silicon dioxide is a compendial excipient and is commonly used in pharmaceutical dosage forms. The BBP-418 Granules for Oral Solution are provided in sachets.The BBP-418 Granules for Oral Solution are reconstituted in water for oral administration.
Placebo
A placebo matched for similar taste and appearance was compounded at the clinical pharmacy with sucralose as a 0.35 mg/mL oral solution in purified water (USP) in a glass container. Sucralose is similar in taste to the compounded drug product.
The Children's Hospital of Philadelphia, Philadelphia
University of Pennsylvania, Philadelphia
Kennedy Krieger Institute, Baltimore
Children's Hospital of the Kings Daughters, Norfolk
University of Florida, Gainsville
University of Iowa, Iowa City
University of Minnesota, Twin Cities, Minneapolis
Washington University School of Medicine, St Louis
University of Kansas Medical Center, Kansas City
Arkansas Children's Hospital, Little Rock
University of Colorado Anschutz Medical Campus, Aurora
University of California Irvine, Irvine
Oregon Health & Science University (OHSU) - Neurology Clinic - South Waterfront, Portland
Royal Brisbane and Women's Hospital,, Brisbane
Rigshospitalet, Neuromuscular Clinic and Research Unit, Copenhagen
Charité Universitätsmedizin Berlin and Max Delbrück Center, Berlin
IRCCS Ca' Granda Ospedale, Milan
Leiden University Medical Center, Leiden
Universitetssykehuset Nord-Norge, Department of Neurology, Tromsø
Great Ormond Street Hospital for Children, London
International Centre for Life, Newcastle upon Tyne
Lead Sponsor
ML Bio Solutions, Inc.
INDUSTRY