BridgeMD
New treatment option for Achondroplasia
Official title A Study of TYRA-300 in Children With Achondroplasia: BEACH301
ClinicalTrials.gov ID: NCT06842355
What this study is testing
What is TYRA-300 0.125 mg/kg?
TYRA-300 0.125 mg/kg is an investigational medicine, being studied as a potential treatment for achondroplasia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 10
You may be able to join if
- 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...
- 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.
You likely can't join if
- Presence or history of any concurrent disease or condition that would interfere with study participation, safety evaluations, or any uncontrolled or...
- 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.
See the full eligibility 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.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Torrance, California, United States
- Aurora, Colorado, United States
- Wilmington, Delaware, United States
- Baltimore, Maryland, United States
- Chevy Chase, Maryland, United States
- Columbia, Missouri, United States
- St Louis, Missouri, United States
- Durham, North Carolina, United States
- Nashville, Tennessee, United States
- Dallas, Texas, United States
- Houston, Texas, United States
- Madison, Wisconsin, United States
- Westmead, New South Wales, Australia
- Parkville, Victoria, Australia
- Edmonton, Alberta, Canada
- Ottawa, Ontario, Canada
- Toronto, Ontario, Canada
- Vitoria-Gasteiz, Alava, Spain
- Stockholm, Sweden
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 10 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Torrance, California, United States; Aurora, Colorado, United States; Wilmington, Delaware, United States; Baltimore, Maryland, United States; Chevy Chase, Maryland, United States; Columbia, Missouri, United States and 13 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.