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
Lundquist Institute for Biomedical Innovation
Torrance 5403022, California 5332921 90502
Torrance 5403022, California 5332921 90502
Children's Hospital Colorado
Aurora 5412347, Colorado 5417618 80045
Aurora 5412347, Colorado 5417618 80045
Nemours Alfred I duPont Hospital for Children
Wilmington 4145381, Delaware 4142224 19803
Wilmington 4145381, Delaware 4142224 19803
Uncommon Cures
Chevy Chase 4351335, Maryland 4361885 20815
Chevy Chase 4351335, Maryland 4361885 20815
University of Missouri
Columbia 4381982, Missouri 4398678 65201
Columbia 4381982, Missouri 4398678 65201
Washington University
St Louis 4407066, Missouri 4398678 63130
St Louis 4407066, Missouri 4398678 63130
Children's Medical Center, Dallas
Dallas 4684888, Texas 4736286 75235
Dallas 4684888, Texas 4736286 75235
University of Wisconsin-Madison
Madison 5261457, Wisconsin 5279468 53715
Madison 5261457, Wisconsin 5279468 53715
More Details
- NCT ID
- NCT06842355
- 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.