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

ConditionAchondroplasia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age3-10
SponsorTyra Biosciences, Inc

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • TYRA-300 0.125 mg/kg

    Drug

    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/kg

    Drug

    Subsequent dose level escalations will occur based on criteria outlined in the protocol.

  • TYRA-300 0.375 mg/kg

    Drug

    Subsequent dose level escalations will occur based on criteria outlined in the protocol.

  • TYRA-300 0.50 mg/kg

    Drug

    Subsequent dose level escalations will occur based on criteria outlined in the protocol.

  • TYRA-300 0.625 mg/kg

    Drug

    Subsequent dose level escalations may occur based on criteria outlined in the protocol.

Treatment groups

92 Participants
are divided into 5 treatment groups
Group A: TYRA-300 0.125 mg/kgExperimental treatment 1 intervention
Group B: TYRA-300 0.25 mg/kgExperimental treatment 1 intervention
Group C: TYRA-300 0.375 mg/kgExperimental treatment 1 intervention
Group D: TYRA-300 0.50 mg/kgExperimental treatment 1 intervention
Group E: TYRA-300 0.625 mg/kgExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of treatment-related adverse events as assessed by CTCAE v5.0

Time frame
Up to 12 months
2

Change from baseline in annualized growth velocity (Cohort 1)

Time frame
12 months

Secondary outcomes

1

Change from baseline in annualized growth velocity (Cohort 1)

Time frame
6 months
2

Change from baseline in height z-score (Cohort 1)

Time frame
6 and 12 months
3

Pharmacokinetics: maximum plasma concentration (Cmax)

Time frame
15 days
4

Pharmacokinetics: time to reach maximum plasma concentration (Tmax)

Time frame
15 days

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Tyra Biosciences, Inc

Lead sponsor

Source ClinicalTrials.gov
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