A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301
Status: Recruiting
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY
The purpose of this study is to evaluate the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with achondroplasia with open growth plates.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 10
Healthy Volunteers: f
View:
• Aged 3 to 10 years old (inclusive) at the time of consent.
• Informed consent provided by parent(s) or legal guardian(s). As study participants are less than 18 years old, participants are willing and able to provide written assent (where applicable and required).
• Molecular diagnosis of achondroplasia (FGFR3 G380R).
• Radiographically confirmed open growth plates at Screening, as determined by bone age X-ray.
• Able to stand and ambulate independently.
• Able to take oral medication.
• Sentinel Safety Cohort only: aged 5 to 10 years old (inclusive).
• Cohort 1 only: aged 3 to 10 years old (inclusive) and are naive to prior growth accelerating therapy.
• Cohort 2 only: aged 3 to 10 years old (inclusive) and have received prior growth accelerating therapy.
Locations
United States
California
Lundquist Institute for Biomedical Innovation
RECRUITING
Torrance
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Delaware
Nemours Alfred I duPont Hospital for Children
RECRUITING
Wilmington
Maryland
Johns Hopkins University School of Medicine
RECRUITING
Baltimore
Uncommon Cures
RECRUITING
Chevy Chase
Missouri
University of Missouri
RECRUITING
Columbia
Washington University
RECRUITING
St Louis
Tennessee
Vanderbilt University Medical Center
RECRUITING
Nashville
Texas
Children's Medical Center, Dallas
RECRUITING
Dallas
Wisconsin
University of Wisconsin-Madison
RECRUITING
Madison
Other Locations
Australia
Royal Children's Hospital
RECRUITING
Parkville
The Children's Hospital at Westmead
RECRUITING
Westmead
Canada
The Hospital for Sick Children
RECRUITING
Toronto
Contact Information
Primary
Sinette Heys
ACH@tyra.bio
(619) 728-4805
Time Frame
Start Date:2025-03-04
Estimated Completion Date:2030-06
Participants
Target number of participants:92
Treatments
Experimental: TYRA-300 0.125 mg/kg
TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
Experimental: TYRA-300 0.25 mg/kg
TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
Experimental: TYRA-300 0.375 mg/kg
TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.
Experimental: TYRA-300 0.50 mg/kg
TYRA-300 is provided as sprinkle capsules/mini-tablets. The total dose will be calculated based on the participant's weight. Weight adjustments will be made every 3 months.