Comparing 5 approaches for achondroplasia
Official title: A Study of TYRA-300 in Children With Achondroplasia: BEACH301
A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301
- Phase 2
- 5 groups
- Sites in Edmonton, Ottawa and 1 more city
- Recruiting
Interventions (5)
- Medication
TYRA-300 0.125 mg/kg
Initial dose level of TYRA-300 per protocol, subsequent dose level escalations will occur based on criteria outlined in the protocol.
- Medication
TYRA-300 0.25 mg/kg
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
- Medication
TYRA-300 0.375 mg/kg
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
- Medication
TYRA-300 0.50 mg/kg
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
- Medication
TYRA-300 0.625 mg/kg
Subsequent dose level escalations may occur based on criteria outlined in the protocol.
Canadian Sites (3)
3 of 3 recruiting
- Recruiting
Women's and Children's Health Research Institute, University of Alberta
Edmonton, Alberta
- Recruiting
Children's Hospital of Eastern Ontario
Ottawa, Ontario
- Recruiting
The Hospital for Sick Children
Toronto, Ontario
Eligibility Criteria
See who this study is looking for15 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Aged 3 to 10 years old (inclusive) at the time of consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →.
- +Sentinel Safety CohortCohortA group of participants sharing a characteristic, followed together.Read more → 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.
- +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 ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more →, as determined by bone age X-ray.
- +Able to stand and ambulate independently.
- +Able to take oral medication.
Exclusion
- −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.
- −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.
- −Prior limb lengthening surgery or planned or expected to have limb lengthening surgery while enrolledEnrolmentThe number of participants a study plans to include, or has included.Read more → in the study.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more → — a middle-stage study in a few hundred people at most, looking at what the treatment does and watching for side effectsSide effectAn unwanted effect thought to be caused by the treatment itself.Read more →.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 5 groups in this study.
Group A receives TYRA-300 0.125 mg/kg.
Registry label: A: TYRA-300 0.125 mg/kg
Group B receives TYRA-300 0.25 mg/kg.
Registry label: B: TYRA-300 0.25 mg/kg
Group C receives TYRA-300 0.375 mg/kg.
Registry label: C: TYRA-300 0.375 mg/kg
Group D receives TYRA-300 0.50 mg/kg.
Registry label: D: TYRA-300 0.50 mg/kg
Group E receives TYRA-300 0.625 mg/kg.
Registry label: E: TYRA-300 0.625 mg/kg
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Groups are assigned by the study team using set rules, rather than by chance.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 3 years to 10 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 92 people.
The study is currently expected to finish around June 2030.
The main measurement is taken over: Up to 12 months.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Incidence of treatment-related adverse eventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → as assessed by CTCAE v5.0 — measured over Up to 12 months.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06842355 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About TYRA-300 0.125 mg/kgDrug
Initial dose level of TYRA-300 per protocol, subsequent dose level escalations will occur based on criteria outlined in the protocol.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About TYRA-300 0.25 mg/kgDrug
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About TYRA-300 0.375 mg/kgDrug
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About TYRA-300 0.50 mg/kgDrug
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About TYRA-300 0.625 mg/kgDrug
Subsequent dose level escalations may occur based on criteria outlined in the protocol.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 3 years to 10 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Groups are assigned by the study team using set rules, rather than by chance.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 3 years to 10 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Up to 12 months.
The study as a whole is currently expected to finish around 2030-06.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 92 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 3 locations, including: Edmonton, Alberta, Canada; Ottawa, Ontario, Canada; Toronto, Ontario, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.