Evaluating TYRA-300 for children with achondroplasia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 2, multicenter, open-label study aims to assess the safety and tolerability of TYRA-300, a selective FGFR-3 tyrosine kinase inhibitor, in children aged 3 to 10 years with achondroplasia and open growth plates. The study will involve a dose-escalation approach across three cohorts: a Sentinel Safety Cohort, Cohort 1 for treatment-naive participants, and Cohort 2 for those who have previously received growth accelerating therapy. Participants will receive varying doses of TYRA-300 to identify potentially effective dosages.

Why it matters

Who can join: the full rules

Inclusion 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.

Exclusion Criteria:

* 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.

Where it's running

• Lundquist Institute for Biomedical Innovation
  Torrance, California, United States Recruiting
• Children's Hospital Colorado
  Aurora, Colorado, United States Recruiting
• Nemours Alfred I duPont Hospital for Children
  Wilmington, Delaware, United States Recruiting
• Johns Hopkins University School of Medicine
  Baltimore, Maryland, United States Recruiting
• Uncommon Cures
  Chevy Chase, Maryland, United States Recruiting
• University of Missouri
  Columbia, Missouri, United States Recruiting

Study contacts

• Study coordinator: Lara Balick
• Email: tyraclinicaltrials@tyra.bio
• Phone: (929) 792-5633

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Evaluating a new treatment for infants with achondroplasia

  This study is testing a new treatment for infants with achondroplasia to see if it helps them grow better compared to a placebo.

  Recruiting at 18 sites Phase 2 Ascendis Pharma A/S · Saint Paul, Minnesota and 17 other locations · NCT06079398

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.