92 Participants Needed

TYRA-300 for Achondroplasia

Recruiting at 2 trial locations
SH
KH
Overseen ByKate Hogg Call
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

Will I have to stop taking my current medications?

The trial requires that participants do not take medications that strongly affect a specific liver enzyme (cytochrome P450 3A4). If your current medications do not fall into this category, you may not need to stop them.

How does the drug TYRA-300 differ from other treatments for achondroplasia?

TYRA-300 is unique because it is a tyrosine kinase inhibitor (a type of drug that blocks certain enzymes) targeting the FGFR3 gene mutation, which is the cause of achondroplasia. This approach is different from other treatments like vosoritide, which aims to increase growth velocity, as TYRA-300 directly addresses the genetic mutation responsible for the condition.12345

What is the purpose of 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.

Research Team

DW

Doug Warner, MD

Principal Investigator

Tyra Biosciences

Eligibility Criteria

This trial is for children aged 3 to 10 with a confirmed diagnosis of achondroplasia, which is a bone growth disorder. They must have open growth plates, be able to walk and take oral medication independently. Consent from parents or guardians is required, and the child's willingness to assent if applicable.

Inclusion Criteria

I am between 3 and 10 years old.
I can stand and walk on my own.
I am between 3 and 10 years old and have not had treatment to speed up growth.
See 6 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose-Escalation

Participants receive TYRA-300 in a dose-escalation format to determine safety and tolerability

12 weeks
Weight adjustments every 3 months

Dose-Expansion

Participants continue to receive TYRA-300 to identify potentially effective dose(s)

12 weeks
Weight adjustments every 3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • TYRA-300
Trial Overview The study tests different doses of TYRA-300 (0.125 mg/kg up to 0.50 mg/kg) in children with achondroplasia to assess safety, tolerability, and effective dosing for promoting bone growth.
Participant Groups
4Treatment groups
Experimental Treatment
Group I: TYRA-300 0.50 mg/kgExperimental Treatment1 Intervention
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.
Group II: TYRA-300 0.375 mg/kgExperimental Treatment1 Intervention
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.
Group III: TYRA-300 0.25 mg/kgExperimental Treatment1 Intervention
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.
Group IV: TYRA-300 0.125 mg/kgExperimental Treatment1 Intervention
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.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Tyra Biosciences, Inc

Lead Sponsor

Trials
4
Recruited
500+

Findings from Research

A spinal orthosis was tested on 19 achondroplastic children aged 11 to 52 months, resulting in a significant mean reduction of 23 degrees in the thoracolumbar curve and 15 degrees in the lumbosacral curve, indicating effective correction of spinal deformities.
In a larger group of 31 achondroplastic children and adults, the orthosis not only reduced spinal deformities but also provided symptom relief for back pain and neurological deficits, demonstrating its efficacy as a treatment option.
Achondroplasia: effectiveness of an orthosis in reducing deformity of the spine.Siebens, AA., Hungerford, DS., Kirby, NA.[2007]
In a study of 6 patients with achondroplasia, surgical treatment involving posterior osteotomy and decompression effectively corrected thoracolumbar kyphosis from an average of 53.3° to 13.3°, achieving a 75% correction rate.
Patients also showed significant improvement in neurological function, with average JOA scores increasing from 4.5 to 8.5 points after surgery, indicating the procedure's efficacy in addressing both spinal deformity and associated neurological deficits.
[Posterior osteotomy and decompression for spinal deformity in patients with achondroplasia].Sun, W., Zhang, JG., Qiu, GX., et al.[2011]
In a study of 22 children with achondroplasia and genu varum, tension band plate (TBP) surgery effectively corrected knee deformities, with significant improvements in radiographic measurements over an average follow-up of 24.5 months.
Younger patients (average age 6.5 years) had better outcomes, highlighting the importance of early intervention for successful correction of varus deformities.
Growth Modulation by Tension Band Plate in Achondroplasia With Varus Knee Deformity: Comparison of Gait Analysis Measurements.Ulusaloglu, AC., Asma, A., Silva, LC., et al.[2023]

References

Achondroplasia: effectiveness of an orthosis in reducing deformity of the spine. [2007]
[Posterior osteotomy and decompression for spinal deformity in patients with achondroplasia]. [2011]
Growth Modulation by Tension Band Plate in Achondroplasia With Varus Knee Deformity: Comparison of Gait Analysis Measurements. [2023]
Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. [2020]
Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model. [2020]
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