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A Study of TYRA-300 in Children With Achondroplasia: BEACH301

The BEACH301 study is exploring a new medication called TYRA-300 for children aged 3 to 10 who have achondroplasia. Achondroplasia is a condition that affects how bones grow, leading to shorter limbs. This study aims to understand if TYRA-300 is safe for children, how well their bodies handle it, and to identify the most effective dose. The children taking part still have 'open growth plates', which are areas in their bones where growth happens. This is a Phase 2 study, meaning it's an early step to gather more information after initial safety tests. The goal is to see if this medicine could help children with achondroplasia grow more effectively.

Överblick

Status
Rekrytering
Fas
FAS2
Sponsor
Tyra Biosciences, Inc
Inskrivningsmål
92
Starta
04 Mar 2025
Beräknad slutförande
01 Jun 2030
Akondroplasi

Vad handlar denna studie om?

This study, known as BEACH301, is testing a new medicine called TYRA-300 for children who have a condition called achondroplasia. Achondroplasia is a genetic condition that affects how bones grow, particularly in the arms and legs, leading to shorter limbs. The study includes children aged 3 to 10 years old who still have 'open growth plates' – these are parts of their bones that are still growing.

The main goals of this study are to see if TYRA-300 is safe for children to take, how well their bodies tolerate it (meaning if they have uncomfortable side effects), and to figure out the right amount of medicine (the dose) that might be helpful. This is a 'Phase 2' study, which means it’s an important step in researching new medicines. It helps us learn more about the medicine's effects in people after initial safety checks.

Researchers are looking at different doses of TYRA-300 in this study. They hope to find a dose that could potentially help children with achondroplasia grow. The study is designed to carefully observe how children respond to the medicine and to gather crucial information for its future development.

Viktiga punkter

  • This study is for children aged 3-10 with achondroplasia.
  • It tests a new medicine, TYRA-300, taken by mouth.
  • The main goals are to check safety, tolerability, and find the right dose.
  • Children must have 'open growth plates' to join.
  • Parents and children must agree to participate.
  • Deltagande innebär regelbundna klinikbesök och övervakning.

Vem kan vara berättigad?

To join this study, children need to be between 3 and 10 years old. They must have a confirmed diagnosis of achondroplasia and still have 'open growth plates,' which are the areas in their bones that allow them to grow. It's also important that they can stand and walk by themselves and can take medicine by mouth.

There are a few specific groups within the study. Some children (aged 5-10) will be in a 'safety group' first. Others (aged 3-10) will either not have had any previous growth-boosting treatments, or they will have had such treatments in the past. Parents or legal guardians must give their permission for their child to take part, and if old enough, the child will also be asked if they want to participate.

Children cannot join the study if they have other serious health conditions that might interfere with the study or their growth, or if they have certain eye problems. Those who have had or plan to have limb-lengthening surgery, or who are taking certain other medications, also won't be able to participate.

Kan denna studie passa dig?

Svara på dessa snabba frågor för att se om du kan vara berättigad. Detta är endast en vägledning — forskarteamet fattar det slutgiltiga beslutet.

  1. Is your child between 3 and 10 years old?
  2. Does your child have a confirmed diagnosis of achondroplasia?
  3. Does your child's doctor confirm they have 'open growth plates'?
  4. Can your child stand and walk on their own?
  5. Can your child take medicine by mouth?
  6. Has your child not had limb-lengthening surgery or planned for one soon?
Svara på varje fråga för att se ditt resultat.

Vad innebär deltagande?

If your child takes part in this study, they will receive the study medicine, TYRA-300, which is taken by mouth. The study involves several visits to a clinic where doctors and nurses will check your child's health, measure their growth, and take blood samples to see how the medicine is working and to monitor for any side effects. These visits will help the researchers understand how safe the medicine is and what dose might be most effective.

Your child will be carefully monitored throughout the study. The study duration is not specified in detail, but it will involve regular check-ups and follow-up appointments. The medical team will explain the full schedule of visits and assessments before you decide to participate.

Potentiella risker och fördelar

Taking part in any study has potential benefits and risks. A potential benefit of joining this study could be that your child receives a new medicine that might help with their growth due to achondroplasia. However, there's no guarantee the medicine will work for every child, and it might not work at all. Potential risks include experiencing side effects from the medicine, which could be mild or more serious, though the study aims to find a safe dose. There are also risks associated with clinic visits and procedures like blood tests. You and your child have the right to withdraw from the study at any time, for any reason, without it affecting your child's future medical care.

Platser (25)

  • Lundquist Institute for Biomedical Innovation
    Verifierat postnummer
    Torrance, USA· Rekrytering
  • Barnsjukhuset Colorado
    Verifierat postnummer
    Aurora, USA· Rekrytering
  • Nemours Alfred I duPont Hospital for Children
    Verifierat postnummer
    Wilmington, USA· Rekrytering
  • Johns Hopkins University School of Medicine
    Verifierat postnummer
    Baltimore, USA· Rekrytering
  • Uncommon Cures
    Verifierat postnummer
    Chevy Chase, USA· Rekrytering
  • University of Missouri
    Verifierat postnummer
    Columbia, United States· Rekrytering
  • Washington Universitet
    Verifierat postnummer
    St Louis, USA· Rekrytering
  • Duke University Hospital
    Verifierat postnummer
    Durham, USA· Rekrytering
  • Vanderbilt University Medical Center
    Verifierat postnummer
    Nashville, USA· Rekrytering
  • Children's Medical Center, Dallas
    Verifierat postnummer
    Dallas, USA· Rekrytering
  • University of Texas Health Science Center Medical School at Houston
    Verifierat postnummer
    Houston, USA· Rekrytering
  • University of Wisconsin-Madison
    Verifierat postnummer
    Madison, USA· Rekrytering

Vanliga frågor

What is achondroplasia?

Achondroplasia is a genetic condition that affects bone growth, leading to shorter arms and legs.

What does 'open growth plates' mean?

It means the areas in your child's bones where growth normally happens are still active and open.

Is TYRA-300 a pill?

Yes, TYRA-300 is a medicine that children will take by mouth.

Vad är en 'Fas 2'-studie?

It's an early stage of testing a new medicine in people to see if it's safe and if it works.

Will my child definitely grow taller in this study?

There's no guarantee the medicine will work for every child, as this study is still trying to find out if it is effective.

Hur du får reda på mer

Lara Balick

tyraclinicaltrials@tyra.bio (929) 792-5633 Officiell rättegångsprotokoll

Prata alltid med din läkare eller specialist innan du bestämmer dig för att delta i en studie.

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