BEACH301 Study:

Advancing Achondroplasia Research, Partnership, and a Healthier Future

About the Study

The purpose of this study is to evaluate the safety and efficacy of TYRA-300, an investigational drug, in children ages 3 to 10 with achondroplasia.

What to Expect

TYRA-300 is under development for once-daily oral administration. The study includes up to 3 parts over a 4-year period.

ACH clinical trial locations

Study Locations

BEACH301 is enrolling at clinics worldwide. Click here to see a map and find a location near you.

Who can join the
BEACH301 study?

Your child may be eligible to participate in the BEACH301 study if they are aged 3 to 10 years old with achondroplasia and have open growth plates.

  • 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.

About the BEACH301 Study

The BEACH301 study is a Phase 2 clinical trial evaluating TYRA-300, an investigational drug, in children aged 3 to 10 years old with achondroplasia and open growth plates. It is a multicenter, open-label, dose-escalation and dose-expansion trial.

Open-label means that all children who participate in the clinical trial will receive TYRA-300.

The BEACH301 study is testing an oral (by mouth) investigational medication called TYRA-300 to test its safety and how it is tolerated in children with achondroplasia.

  • The main goal is to test the safety of this investigational drug in children and to evaluate any side effects.

  • Researchers will study how the investigational medication affects growth over time to help decide the best dose for future studies.

  • Researchers will also study how the investigational medication affects functionality, spine health, pain, and quality of life.

What is TYRA-300?

TYRA-300 is an investigational oral drug under development to help children with achondroplasia by targeting the FGFR3 gene, which affects bone growth. TYRA-300 is designed to work selectively on FGFR3.

What to Expect During
the BEACH301 Study

BEACH301 study participants will visit a study site and take part in various activities as part of the clinical trial. To participate, parents or caregivers will be asked to read and sign an Informed Consent Form (ICF). These forms explain important information, such as the purpose of the study, required study visits and procedures, and potential risks and benefits of participation and taking TYRA-300.

After signing the consent form, there is a Screening Visit, wherein a study doctor will complete tests to assess your child’s overall health to see if they are able to be included in the study.

Who is eligible?

Children ages 3-10 with achondroplasia and open growth plates may be eligible to participate, whether they have never received or have previously received a growth-accelerating treatment.

  • Group 1: Children who have never had growth-accelerating medication.
  • Group 2: Children who have had growth-accelerating medication, either as part of medical care or a clinical trial.

About 90 children will take part in the BEACH301 study in several different countries.

Study Overview

Part 1: Natural History Lead-In: In part 1, your child will not receive treatment with the investigational study medication. The purpose is to screen your child and assess over a 6-month time period how your child grows, reaches, walks and experiences their everyday life.

Part 2: Dose-Expansion: In Dose-Expansion, participants will receive the investigational study medication at one of up to four doses in order to help identify the best dose of TYRA-300 to use in future studies. In total, this part will last 12 months.

Part 3: Treatment Extension: After completing the Dose-Expansion, all children will have the opportunity to participate in a 36-month treatment extension and continue to receive TYRA-300.

Key study procedures include a physical exam, blood tests, MRI, X-rays, body measurements,
and a home gait and reach assessment. The study site will provide more details.

For Physicians and Healthcare Providers

Are you a physician looking to refer a patient to the BEACH301 study?
Please contact a study representative at ACH@tyra.bio for more information.

Study Site Locations

Current study locations are listed below and more may be added at any time. If you or someone you know may be interested in participating in the BEACH301 study, please contact advocacy@tyra.bio.

Rare Disease Research

Hillsborough, North Carolina, United States

Rare Disease Research

Kissimmee, Florida, United States

Rare Disease Research

Atlanta, Georgia, United States

Safety is our Top Priority

At Tyra Biosciences, safety is our top priority in the BEACH301 clinical trial for children with achondroplasia. We are committed to carefully monitoring participants at every step, following safety guidelines.

Tyra Biosciences is thankful for the achondroplasia community’s help in designing this study. Families, caregivers, advocates, and medical experts all shared their input to make sure the study reflects the needs of the community.

What is Achondroplasia?

Achondroplasia is the most common condition that affects how bones grow. It happens because of a change in a gene called FGFR3, which stands for “Fibroblast Growth Factor Receptor-3.” Most people with achondroplasia have the same specific change in this gene, called G380R.

How Does Achondroplasia Affect the Body?

The FGFR3 gene helps make a protein that is important for bones. This protein works in special parts of the bones called growth plates. Growth plates are areas in the long bones, the spine, and the base of the skull that help bones grow and develop.

In achondroplasia, the change in the FGFR3 gene causes the protein to work differently. It tells the body to slow down bone growth. This can lead to clinical features of achondroplasia. The features of achondroplasia include disproportionate short stature with shortened limbs.

Frequently Asked Questions

Clinical trials help advance research and bring potential new treatments to children with achondroplasia. By participating, your child may gain access to an investigational treatment and contribute to the development of future therapies for the community.

An investigational treatment has not been approved by regulatory authorities but is allowed to be administered to people for research purposes to understand if it might be a safe and effective treatment for a specific disease or condition.

Taking part in this research study is voluntary. Voluntary means it is your choice whether or not you allow your child to participate. You do not have to allow your child to participate in this study if you do not want to. If you decide to allow your child to participate in the study, you can change your mind at any time. Depending on how old your child is, your child may be required to agree to the study and sign a separate form (called an assent form) to participate in this study.

Yes, the participation is voluntary and you can take your child out of the study at any time. Any data generated up to the point of withdrawal may continue to be used, as described more fully in the informed consent form.

No, the study covers the cost of all trial-related procedures, tests, and medication. Travel support is provided to help families get to the study site. The research team at the study site can provide you more information.

About Our Sponsor

Tyra Biosciences, Inc is a clinical-stage biotechnology company based in Carlsbad, CA. At Tyra, we are focused on applying our accelerated small molecule drug discovery engine to develop therapies in targeted oncology and genetically defined conditions.

Working together with scientists, physicians, advocacy groups and patient families, we’re committed to drug development with the aim to help children with achondroplasia enjoy a healthier future and improved functional ability with fewer painful surgeries.

Let’s Stay Connected!

We are working to advance BEACH301, our clinical study of TYRA-300 for children with achondroplasia. Please complete this form to receive updates.

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