AchondroplasiaPhase 2NCT06842355
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
A Phase 2 study testing TYRA-300 0.125 mg/kg, TYRA-300 0.25 mg/kg and TYRA-300 0.375 mg/kg in people with Achondroplasia, run by Tyra Biosciences, Inc at 25 sites in 8 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 10 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- TYRA-300 0.125 mg/kg, TYRA-300 0.25 mg/kg and TYRA-300 0.375 mg/kg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 92 participants
- Where
- 25 sitesin 8 countries
- Study dates
- 4 Mar 2025to Jun 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 10 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Show all 9Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
25 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
25 sites in 8 countries
United States12 sites, 12 recruiting
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Lauren Samz, 720-777-1975, Lauren.Samz@childrenscolorado.org
- Recruiting
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205
Plamena Simeonova, MD, 410-614-0977, psimeon1@jh.edu
- Recruiting
Uncommon Cures
Chevy Chase, Maryland, 20815
Tamanna Roshan Lal, 240 858 4938, troshanlal@uncommoncures.com
- Recruiting
University of Missouri
Columbia, Missouri, 65201
Taylor Matthews, 573-882-4758, mathewsta@health.missouri.edu
- Recruiting
Children's Medical Center, Dallas
Dallas, Texas, 75235
Colton Youngblood, (214) 456-3163, Colten.Youngblood@childrens.com
- Recruiting
Duke University Hospital
Durham, North Carolina, 27710
Stephanie Bryant, (919) 668-4777, stephanie.l.bryant@duke.edu
- Recruiting
University of Texas Health Science Center Medical School at Houston
Houston, Texas, 77030
Heather Saavedra, (713) 500-7098, heather.saavedra@uth.tmc.edu
- Recruiting
University of Wisconsin-Madison
Madison, Wisconsin, 53715
Sarah M Heitmeier, (608) 263-8863, sheitmeier@wisc.edu
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
LeeAnna Melton, (615) 322-7601, leeanna.melton@vumc.org
- Recruiting
Washington University
St Louis, Missouri, 63130
Michelle Ritter, (314) 362-7306, michelle.ritter@wustl.edu
- Recruiting
Lundquist Institute for Biomedical Innovation
Torrance, California, 90502
Adolfo Morales Garval, (310) 222-3472, adolfo.morales@lundquist.org
- Recruiting
Nemours Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803
Judith Feinson, (302) 651-5928, judith.feinson@nemours.org
Canada3 sites, 3 recruiting
- Recruiting
Women's and Children's Health Research Institute, University of Alberta
Edmonton, Alberta, T6G 2R3
Lee-Ann Carroll, (780) 248-5616, lee-ann.carroll@albertahealthservices.ca
- Recruiting
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Nasrin Khan, (613) 737-7600 ext 4119, NaKhan@cheo.on.ca
- Recruiting
The Hospital for Sick Children
Toronto, Ontario, M5G1X8
Tony Tan, (416) 813 7654 ext. x203575, tony.tan@sickkids.ca
United Kingdom3 sites, 3 recruiting
- Recruiting
Guy's & St Thomas NHS Foundation Trust 2
London, Greater London, SE1 7EH
Ana Pereira, 07796938123, ana.pereira20@nhs.net
- Recruiting
Great Ormond Street Hospital
London, Greater London, WC1N 3JH
Rachel Turek, 44 2079052271, rachel.turek@gosh.nhs.uk
- Recruiting
Sheffield Children's Hospital
Sheffield, South Yorkshire, S10 2TH
Jayne Evans, 44 11402761111, jayne.evans17@nhs.net
Australia2 sites, 2 recruiting
- Recruiting
Royal Children's Hospital
Parkville, Victoria, 3052
Raj Supriya, supriya.raj@mcri.edu.au
- Recruiting
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
Abani Surendran, 61294631868, abani.surendran@health.nsw.gov.au
Spain2 sites, 2 recruiting
- Recruiting
Hospital Univeristario La Paz
Madrid, Madrid, 28046
Ana Ortega Pita, 34 91 2071876, ana.ucicec@gmail.com
- Recruiting
Unidad de Cirugía Artroscópica (MIKS Hospital)
Vitoria-Gasteiz, Alava, 1008
Irene Gimeno Lluch, 34610984309, irene.gimeno@ucatrauma.com
France1 site, 1 recruiting
- Recruiting
Imagine Institute
Paris, 75015
Khue Le Folcvalvez, 33 171196473, khue.lefolcalvez-ext@aphp.fr
Netherlands1 site, 1 recruiting
- Recruiting
UMC Utrecht
Utrecht, 3584 CX
Anne Spaans, +31 88 75 545 76, a.j.spaans@umcutrecht.nl
Sweden1 site, 1 recruiting
- Recruiting
Astrid Lindgren Children's Hospital (Karolinska University Hospital)
Stockholm
Ola Nilsson, +46 8 5248 2425, ola.nilsson@ki.se
About this study
Official title: A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: 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.
Read the 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.
- Conditions
- Achondroplasia
- Being tested
- TYRA-300 0.125 mg/kg, TYRA-300 0.25 mg/kg, TYRA-300 0.375 mg/kg, TYRA-300 0.50 mg/kg, TYRA-300 0.625 mg/kg
- Medical subject headings
- Achondroplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
TYRA-300 0.25 mg/kgDrug
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
TYRA-300 0.375 mg/kgDrug
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
TYRA-300 0.50 mg/kgDrug
Subsequent dose level escalations will occur based on criteria outlined in the protocol.
TYRA-300 0.625 mg/kgDrug
Subsequent dose level escalations may occur based on criteria outlined in the protocol.
| Study group | Role | What they receive |
|---|---|---|
| TYRA-300 0.125 mg/kg | Experimental | 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. Receives: Drug: TYRA-300 0.125 mg/kg |
| TYRA-300 0.25 mg/kg | Experimental | 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. Receives: Drug: TYRA-300 0.25 mg/kg |
| TYRA-300 0.375 mg/kg | Experimental | 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. Receives: Drug: TYRA-300 0.375 mg/kg |
| TYRA-300 0.50 mg/kg | Experimental | 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. Receives: Drug: TYRA-300 0.50 mg/kg |
| TYRA-300 0.625 mg/kg | Experimental | 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. Receives: Drug: TYRA-300 0.625 mg/kg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-related adverse events as assessed by CTCAE v5.0
Measured: Up to 12 months
Change from baseline in annualized growth velocity (Cohort 1)
Measured: 12 months
Secondary outcomes (18)
Change from baseline in annualized growth velocity (Cohort 1)
Measured: 6 months
Change from baseline in height z-score (Cohort 1)
Measured: 6 and 12 months
Pharmacokinetics: maximum plasma concentration (Cmax)
Measured: 15 days
Pharmacokinetics: time to reach maximum plasma concentration (Tmax)
Measured: 15 days
Pharmacokinetics: area under the plasma concentration-time curve (AUC)
Measured: 15 days
Pharmacokinetics: half-life of TYRA-300 (t1/2)
Measured: 15 days
Pharmacokinetics: apparent total clearance (CL/F)
Measured: 15 days
Pharmacokinetics: apparent volume of distribution (Vd/F)
Measured: 15 days
Change from baseline in annualized growth velocity (Cohort 2)
Measured: 6 and 12 months
Change from baseline in height z-score (Cohort 2)
Measured: 6 and 12 months
Change from baseline in standing height (cm)
Measured: 6 and 12 months
Change from baseline in sitting height (cm)
Measured: 6 and 12 months
Change from baseline in upper and lower arm length (cm)
Measured: 6 and 12 months
Change from baseline in tibial length (cm)
Measured: 6 and 12 months
Change from baseline in femur length (cm)
Measured: 6 and 12 months
Change from baseline in arm span proportionality (arm span/height ratio)
Measured: 6 and 12 months
Change from baseline in upper segment/lower segment ratio
Measured: 6 and 12 months
Change from baseline in elbow extension
Measured: 6 and 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Feb 2025
- Study start4 Mar 2025
- Primary completionJan 2030 (estimated)
- Study completionJun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 92 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tyra Biosciences, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Doug Warner, MD, Study chair, Tyra Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06842355
- Sponsor's protocol ID
- TYR300-201
- Status last verified
- Sep 2026
- First submitted
- 29 Jan 2025
- Last updated
- 1 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children With Achondroplasia With Open Growth Plates: BEACH301
- ClinicalTrials.gov ID
- NCT06842355
- Acronym / sponsor ID
- TYR300-201
- Conditions
- Achondroplasia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 92 participants (estimated)
- Lead sponsor
- Tyra Biosciences, IncIndustry (pharma, biotech, devices)
- Study start
- 4 Mar 2025 (actual)
- Primary completion
- Jan 2030 (estimated)
- Study completion
- Jun 2030 (estimated)
- First posted
- 24 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| TYRA-300 0.125 mg/kg | Experimental | Drug: TYRA-300 0.125 mg/kgTYRA-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. |
| TYRA-300 0.25 mg/kg | Experimental | Drug: TYRA-300 0.25 mg/kgTYRA-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. |
| TYRA-300 0.375 mg/kg | Experimental | Drug: TYRA-300 0.375 mg/kgTYRA-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. |
| TYRA-300 0.50 mg/kg | Experimental | Drug: TYRA-300 0.50 mg/kgTYRA-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. |
| TYRA-300 0.625 mg/kg | Experimental | Drug: TYRA-300 0.625 mg/kgTYRA-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. |
Who may qualify
- Ages
- 3 to 10 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TYRA-300 0.125 mg/kgInitial dose level of TYRA-300 per protocol, subsequent dose level escalations will occur based on criteria outlined in the protocol. | Drug | TYRA-300 0.125 mg/kg |
| TYRA-300 0.25 mg/kgSubsequent dose level escalations will occur based on criteria outlined in the protocol. | Drug | TYRA-300 0.25 mg/kg |
| TYRA-300 0.375 mg/kgSubsequent dose level escalations will occur based on criteria outlined in the protocol. | Drug | TYRA-300 0.375 mg/kg |
| TYRA-300 0.50 mg/kgSubsequent dose level escalations will occur based on criteria outlined in the protocol. | Drug | TYRA-300 0.50 mg/kg |
| TYRA-300 0.625 mg/kgSubsequent dose level escalations may occur based on criteria outlined in the protocol. | Drug | TYRA-300 0.625 mg/kg |
Primary outcome
Incidence of treatment-related adverse events as assessed by CTCAE v5.0
Time frame: Up to 12 months
Change from baseline in annualized growth velocity (Cohort 1)
Time frame: 12 months
18 secondary outcomes
Change from baseline in annualized growth velocity (Cohort 1)
Time frame: 6 months
Change from baseline in height z-score (Cohort 1)
Time frame: 6 and 12 months
Pharmacokinetics: maximum plasma concentration (Cmax)
Time frame: 15 days
Pharmacokinetics: time to reach maximum plasma concentration (Tmax)
Time frame: 15 days
Pharmacokinetics: area under the plasma concentration-time curve (AUC)
Time frame: 15 days
Pharmacokinetics: half-life of TYRA-300 (t1/2)
Time frame: 15 days
Pharmacokinetics: apparent total clearance (CL/F)
Time frame: 15 days
Pharmacokinetics: apparent volume of distribution (Vd/F)
Time frame: 15 days
Change from baseline in annualized growth velocity (Cohort 2)
Time frame: 6 and 12 months
Change from baseline in height z-score (Cohort 2)
Time frame: 6 and 12 months
Change from baseline in standing height (cm)
Time frame: 6 and 12 months
Change from baseline in sitting height (cm)
Time frame: 6 and 12 months
Change from baseline in upper and lower arm length (cm)
Time frame: 6 and 12 months
Change from baseline in tibial length (cm)
Time frame: 6 and 12 months
Change from baseline in femur length (cm)
Time frame: 6 and 12 months
Change from baseline in arm span proportionality (arm span/height ratio)
Time frame: 6 and 12 months
Change from baseline in upper segment/lower segment ratio
Time frame: 6 and 12 months
Change from baseline in elbow extension
Time frame: 6 and 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2026.
Related trials for Achondroplasia
Enrolling by invitationPhase 2
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
Achondroplasia
RecruitingPhase 2/3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Achondroplasia
Active, not recruitingPhase 2
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Achondroplasia
Active, not recruitingPhase 3
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
Achondroplasia
RecruitingObservational study
Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
Achondroplasia, Hypochondroplasia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Doug Warner, MD study chair
Tyra Biosciences
25 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing TYRA-300 0.125 mg/kg, TYRA-300 0.25 mg/kg and TYRA-300 0.375 mg/kg in people with Achondroplasia, run by Tyra Biosciences, Inc at 25 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 1 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, Canada, United Kingdom, Australia, Spain and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Lara Balick ((929) 792-5633, tyraclinicaltrials@tyra.bio), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-related adverse events as assessed by CTCAE v5.0 (time frame: Up to 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2026.