A Study of TYRA-300 in Children With Achondroplasia: BEACH301
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.
Study details:
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.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
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.
Eligibility
Age eligible for study : NA
Healthy volunteers accepted : No
Gender eligible for study: ALL
Things to know
Study dates
Study start:
Primary completion:
Study completion finish:
Study type
TREATMENT
Phase
- PHASE2
Study ID
NCT06842355
Intervention or treatment
DRUG: TYRA-300 0.125 mg/kg
DRUG: TYRA-300 0.25 mg/kg
DRUG: TYRA-300 0.375 mg/kg
DRUG: TYRA-300 0.50 mg/kg
Conditions
- Achondroplasia
You may be eligible to participate in this trial based on your search. Check eligibility by answering some questions.
Research sites nearby
Closest Location:
Royal Children's Hospital
Select from list below to view details:
Royal Children's Hospital
Parkville, Victoria, Australia
Loading map...
Who is running the clinical trial?
Tyra Biosciences, Inc
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: TYRA-300 0.125 mg/kg
| Drug: TYRA-300 0.125 mg/kg |
EXPERIMENTAL: TYRA-300 0.25 mg/kg
| Drug: TYRA-300 0.25 mg/kg |
EXPERIMENTAL: TYRA-300 0.375 mg/kg
| Drug: TYRA-300 0.375 mg/kg |
EXPERIMENTAL: TYRA-300 0.50 mg/kg
| Drug: TYRA-300 0.50 mg/kg |
EXPERIMENTAL: TYRA-300 0.625 mg/kg
| Drug: TYRA-300 0.625 mg/kg |
What is the study measuring?
Primary Outcome
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-related adverse events as assessed by CTCAE v5.0 | Not Specified | Up to 12 months |
| Change from baseline in annualized growth velocity (Cohort 1) | Not Specified | 12 months |
Secondary Outcome
| Measure | Description | Time Frame |
|---|---|---|
| Change from baseline in annualized growth velocity (Cohort 1) | Not Specified | 6 months |
| Change from baseline in height z-score (Cohort 1) | Not Specified | 6 and 12 months |
| Pharmacokinetics: maximum plasma concentration (Cmax) | Not Specified | 15 days |
| Pharmacokinetics: time to reach maximum plasma concentration (Tmax) | Not Specified | 15 days |
| Pharmacokinetics: area under the plasma concentration-time curve (AUC) | Not Specified | 15 days |
| Pharmacokinetics: half-life of TYRA-300 (t1/2) | Not Specified | 15 days |
| Pharmacokinetics: apparent total clearance (CL/F) | Not Specified | 15 days |
| Pharmacokinetics: apparent volume of distribution (Vd/F) | Not Specified | 15 days |
| Change from baseline in annualized growth velocity (Cohort 2) | Not Specified | 6 and 12 months |
| Change from baseline in height z-score (Cohort 2) | Not Specified | 6 and 12 months |
| Change from baseline in standing height (cm) | Not Specified | 6 and 12 months |
| Change from baseline in sitting height (cm) | Not Specified | 6 and 12 months |
| Change from baseline in upper and lower arm length (cm) | Not Specified | 6 and 12 months |
| Change from baseline in tibial length (cm) | Not Specified | 6 and 12 months |
| Change from baseline in femur length (cm) | Not Specified | 6 and 12 months |
| Change from baseline in arm span proportionality (arm span/height ratio) | Not Specified | 6 and 12 months |
| Change from baseline in upper segment/lower segment ratio | Not Specified | 6 and 12 months |
| Change from baseline in elbow extension | Not Specified | 6 and 12 months |
References
Clinical Trials Gov: A Study of TYRA-300 in Children With Achondroplasia: BEACH301Other trials to consider
Loading suggested trials...
