Phase
Condition
Severe Short Stature
Treatment
Infigratinib 0.064 mg/kg
Infigratinib 0.128 mg/kg
Infigratinib 0.25 mg/kg
Clinical Study ID
Ages 3-11 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Signed informed consent by participant or parent(s) or legally authorizedrepresentative (LAR) and signed informed assent by the participant (whenapplicable).
Diagnosis of ACH, documented clinically and confirmed by genetic testing.
At least a 6-month period of growth assessment in the PROPEL study (ProtocolQBGJ398-001) before study entry.
Ambulatory and able to stand without assistance
Able to swallow oral medication.
Exclusion
Exclusion Criteria:
Hypochondroplasia or short stature condition other than ACH.
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.
Significant concurrent disease or condition that, in the view of the Investigatorand/or Sponsor, would confound assessment of efficacy or safety of infigratinib.
Current evidence of corneal or retinal disorder/keratopathy.
History of malignancy.
Currently receiving treatment with agents that are known strong inducers orinhibitors of CYP3A4 and medications which increase serum phosphorus and/or calciumconcentration.
Treatment with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolicsteroids in the previous 6 months or long-term treatment (>3 months) at any time.
Treatment with a C-type natriuretic peptide (CNP) analog, fibroblast growth factor (FGF) ligand trap, or treatment targeting FGFR inhibition at any time.
Regular long-term treatment (>3 weeks) with oral corticosteroids (low-dose ongoinginhaled steroid for asthma is acceptable).
Treatment with any other investigational product or investigational medical devicefor the treatment of ACH or short stature.
Previous limb-lengthening surgery or guided growth surgery.
Fracture within 12 months of screening.
Study Design
Connect with a study center
Murdoch Children's Hospital
Parkville, Victoria 3052
AustraliaSite Not Available
Stollery Children's Hospital
Edmonton, Alberta T6G 2H7
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
Vithas Hospital San José
Vitoria-Gasteiz, Álava 01012
SpainSite Not Available
Hospital Universitario La Paz
Madrid, 24086
SpainSite Not Available
Hospital Universitario Virgen de la Victoria
Málaga,
SpainSite Not Available
Sheffield Children's Hospital
Sheffield, England S10 2TH
United KingdomSite Not Available
Birmingham Children's Hospital
Birmingham,
United KingdomSite Not Available
University Hospitals Bristol and Weston NHS Foundation Trust
Bristol, BS1 3NU
United KingdomSite Not Available
Queen Elizabeth University Hospital
Glasgow,
United KingdomSite Not Available
Evelina London Children's Hospital
London,
United KingdomSite Not Available
Manchester University Children's Hospital
Manchester,
United KingdomSite Not Available
UCSF Benioff Children's Hospital
Oakland, California 94618
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
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United StatesSite Not Available
Vanderbilt University Medical Center
Nashville, Tennessee 37232
United StatesSite Not Available
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