A Phase 2 Dose Escalation/Dose Expansion Study of TYRA-300 in Children with Achondroplasia
Research type
Research Study
Full title
A Multicenter, Phase 2, Dose-Escalation/Dose-Expansion Study of TYRA-300 in Children with Achondroplasia with Open Growth Plates: BEACH301
IRAS ID
1011484
Contact name
Will Charlton
Contact email
Sponsor organisation
Tyra Biosciences, Inc
Eudract number
2024-520331-33
Clinicaltrials.gov Identifier
Research summary
The present study is to determine the safety, tolerability, and identify potentially effective dose(s) of TYRA-300 in children with ACH with open growth plates of children: the Sentinel Safety Cohort, Cohort 1, and Cohort 2. The Sentinel Safety Cohort will participate in a dose-escalation of up to four dose levels of TYRA-300 and be comprised of children with ACH aged 5-10 years old (inclusive) who are naïve to treatment with prior growth accelerating therapy. Participants enrolled in the Sentinel Safety Cohort will not be required to complete a natural history study lead-in prior to enrolling in the dose-escalation. Following the dose-escalation, sentinel participants will continue into 12 months of open-label treatment at their assigned dose level.
Cohorts 1 and 2 will participate in a natural history study lead-in for a minimum of 6 months prior to rollover into an open label dose-expansion of dose levels cleared by the Sentinel Safety Cohort. Cohort 1 will be comprised of children with ACH aged 3-10 years old (inclusive) who are naïve to treatment with prior growth accelerating therapy. Cohort 2 will be an exploratory cohort comprised of children with ACH aged 3-10 years old (inclusive) who have received prior growth accelerating therapy.
Within this study all participants will have the opportunity to participate in a 36-month treatment extension. Additionally, all participants will have the opportunity to continue therapy with TYRA-300 in a long-term treatment extension study within a separate protocol.REC name
London - Westminster Research Ethics Committee
REC reference
25/LO/0490
Date of REC Opinion
14 Aug 2025
REC opinion
Further Information Favourable Opinion