Phase
Condition
Severe Short Stature
Treatment
N/AClinical Study ID
Ages 30-17 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Signed informed consent by study participant or parent(s) or legally authorizedrepresentative (LAR) and signed informed assent by the study participant (whenapplicable)
Aged 2.5 to <17 years at study entry
Diagnosis of ACH
Study participants and parent(s) or LAR(s) are willing and able to comply with studyvisits and study procedures
Exclusion
Key Exclusion Criteria:
Have hypochondroplasia or short stature condition other than ACH (e.g. trisomy 21,pseudoachondroplasia, psychosocial short stature)
In females, having had their menarche
Height < -2 or > +2 standard deviations for age and sex based on reference tables ongrowth in children with ACH
Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior toscreening
Current evidence of corneal or retinal disorder/keratopathy
Current evidence of endocrine alterations of calcium/phosphorus homeostasis
Have a concurrent disease or condition that in the view of the Investigator and/orSponsor, may impact growth or where the treatment is known to impact growth.
Significant abnormality in screening laboratory results.
Have been treated with growth hormone, insulin-like growth factor 1 (IGF 1), oranabolic steroids in the previous 6 months or long-term treatment (>3 months) at anytime
Have had regular long-term treatment (>1 month) with oral corticosteroids (low-doseongoing inhaled steroid for asthma is acceptable)
Have had previous guided growth surgery or limb-lengthening surgery within 12 monthsprior to screening.
Study Design
Connect with a study center
Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
Buenos Aires,
ArgentinaSite Not Available
Murdoch Children's Research Institute
Parkville,
AustraliaSite Not Available
Stollery Children's Hospital
Edmonton, Alberta T6G 2H7
CanadaSite Not Available
Children's Hospital - London Health Sciences Center
London, Ontario N6A 5W9
CanadaSite Not Available
University of Ottawa
Ottawa, Ontario K1N 6N5
CanadaSite Not Available
SickKids - The Hospital for Sick Children
Toronto, Ontario M5G1X8
CanadaSite Not Available
University of Montreal
Montréal, Quebec H3C 3J7
CanadaSite Not Available
Hopital Femme Mere Enfant
Lyon,
FranceSite Not Available
Hopital Necker-Enfants Malades
Paris,
FranceSite Not Available
Hopital des Enfants
Toulouse,
FranceSite Not Available
Otto-von-Guericke-University Magdeburg Medical Fakulty
Magdeburg, 39120
GermanySite Not Available
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlino
Milan, 20122
ItalySite Not Available
Rare Disease Unit Fondazione Policlinico A Gemelli IRCCS
Rome, 00168
ItalySite Not Available
Haukeland Universitetssjukehus
Bergen, 5009
NorwaySite Not Available
Oslo Universitetssykehus
Oslo, 0372
NorwaySite Not Available
KK Women's and Children's Hospital
Singapore, 229899
SingaporeSite Not Available
Vithas Hospital San Jose
Barcelona, 08305
SpainSite Not Available
Hospital Universitario La Paz
Madrid, 24086
SpainSite Not Available
Hospital Universitario Virgen de la Victoria
Málaga,
SpainSite Not Available
Bristol Royal Hospital for Children
Bristol, England
United KingdomSite Not Available
Birmingham Children's Hospital
Birmingham,
United KingdomSite Not Available
Queen Elizabeth University Hospital
Glasgow,
United KingdomSite Not Available
St. Thomas' Hospital
London,
United KingdomSite Not Available
Manchester University Children's Hospital
Manchester,
United KingdomSite Not Available
Sheffield Children's Hospital
Sheffield,
United KingdomSite Not Available
Benioff Children's Hospital Oakland
Oakland, California 94609
United StatesSite Not Available
Children's Hospital Colorado
Aurora, Colorado 80045
United StatesSite Not Available
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware 19803
United StatesSite Not Available
Johns Hopkins School of Medicine
Baltimore, Maryland 21211
United StatesSite Not Available
University of Missouri
Columbia, Missouri 65211
United StatesSite Not Available
Cincinnati Children's Hospital
Cincinnati, Ohio 45229
United StatesSite Not Available
Vanderbilt University Medical Center
Nashville, Tennessee 37232
United StatesSite Not Available
Cook's Children Medical Center
Dallas, Texas 75207
United StatesSite Not Available
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin 53705
United StatesSite Not Available
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