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 (4) 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
Colorado
Children's Hospital Colorado
RECRUITING
Aurora
Delaware
Nemours Alfred I duPont Hospital for Children
RECRUITING
Wilmington
Missouri
Washington University
RECRUITING
St Louis
Other Locations
Australia
Royal Children's Hospital
RECRUITING
Parkville
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.