A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Purpose
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.
Condition
- Achondroplasia
Eligibility
- Eligible Ages
- Between 3 Years and 10 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- 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.
Exclusion Criteria
- Presence or history of any concurrent disease or condition that would interfere with study participation, safety evaluations, or any uncontrolled or untreated condition that could impact pediatric growth. - Diagnosis of endocrine condition that alters calcium/phosphate homeostasis. - Prior limb lengthening surgery or planned or expected to have limb lengthening surgery while enrolled in the study. - Taking medications that are strong inhibitors or inducers of cytochrome P450 (Cyp) 3A4. - History or current evidence of corneal or retinal disorder/keratopathy. - Presence of guided growth hardware/8 plates. Planned or anticipated orthopedic surgeries.
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Sequential Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
Recruiting Locations
Vanderbilt University Medical Center
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Tyra Biosciences, Inc
Detailed Description
This is a Phase 2, multicenter, open-label, dose-escalation study to determine the safety, tolerability, and identify potentially effective dose(s) of TYRA-300, a fibroblast growth factor receptor (FGFR)-3 selective tyrosine kinase inhibitor, in children 3 to 10 years of age with achondroplasia with open growth plates that will examine three cohorts of children: the Sentinel Safety Cohort, Cohort 1, and Cohort 2.