Phase
Condition
Severe Short Stature
Treatment
TYRA-300 0.50 mg/kg
TYRA-300 0.25 mg/kg
TYRA-300 0.375 mg/kg
Clinical Study ID
Ages 3-10 All Genders
Study Summary
Eligibility Criteria
Inclusion
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 participantsare less than 18 years old, participants are willing and able to provide writtenassent (where applicable and required).
Molecular diagnosis of achondroplasia (FGFR3 G380R).
Radiographically confirmed open growth plates at Screening, as determined by boneage 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 growthaccelerating therapy.
Cohort 2 only: aged 3 to 10 years old (inclusive) and have received prior growthaccelerating therapy.
Exclusion
Exclusion Criteria:
Presence or history of any concurrent disease or condition that would interfere withstudy participation, safety evaluations, or any uncontrolled or untreated conditionthat could impact pediatric growth.
Diagnosis of endocrine condition that alters calcium/phosphate homeostasis.
Prior limb lengthening surgery or planned or expected to have limb lengtheningsurgery 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 orthopedicsurgeries.
Study Design
Study Description
Connect with a study center
The Children's Hospital at Westmead
Westmead, New South Wales 2145
AustraliaActive - Recruiting
Royal Children's Hospital
Parkville, Victoria 3052
AustraliaActive - Recruiting
Women's and Children's Health Research Institute, University of Alberta
Edmonton, Alberta T6G 2R3
CanadaActive - Recruiting
Children's Hospital of Eastern Ontario
Ottawa, Ontario K1H 8L1
CanadaActive - Recruiting
The Hospital for Sick Children
Toronto, Ontario M5G1X8
CanadaActive - Recruiting
Unidad de Cirugía Artroscópica (MIKS Hospital)
Vitoria-Gasteiz, Alava 1008
SpainActive - Recruiting
Astrid Lindgren Children's Hospital (Karolinska University Hospital)
Stockholm,
SwedenActive - Recruiting
Lundquist Institute for Biomedical Innovation
Torrance, California 90502
United StatesActive - Recruiting
Children's Hospital Colorado
Aurora, Colorado 80045
United StatesActive - Recruiting
Nemours Alfred I duPont Hospital for Children
Wilmington, Delaware 19803
United StatesActive - Recruiting
Johns Hopkins University School of Medicine
Baltimore, Maryland 21205
United StatesActive - Recruiting
Uncommon Cures
Chevy Chase, Maryland 20815
United StatesActive - Recruiting
University of Missouri
Columbia, Missouri 65201
United StatesActive - Recruiting
Washington University
St Louis, Missouri 63130
United StatesActive - Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee 37232
United StatesActive - Recruiting
Children's Medical Center, Dallas
Dallas, Texas 75235
United StatesActive - Recruiting
University of Texas Health Science Center Medical School at Houston
Houston, Texas 77030
United StatesActive - Recruiting
University of Wisconsin-Madison
Madison, Wisconsin 53715
United StatesActive - Recruiting

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