A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
NCT ID: NCT06164951
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
ACTIVE_NOT_RECRUITING
PHASE3
110 participants
INTERVENTIONAL
2023-11-10
2026-04-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Keywords
Explore important study keywords that can help with search, categorization, and topic discovery.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Infigratinib 0.25 mg/kg/day
Infigratinib at 2, 3.5, 5, 7, 10 mg
Infigratinib 0.25 mg/kg/day
Daily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
Placebo 0.25 mg/kg/day
Placebo Comparator at 2, 3.5, 5, 7, 10 mg
Placebo Comparator 0.25 mg/kg/day
Daily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Infigratinib 0.25 mg/kg/day
Daily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
Placebo Comparator 0.25 mg/kg/day
Daily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
Type of Subject and Disease Characteristics
2. Subjects who have a diagnosis of ACH that has been documented clinically and confirmed by genetic testing.
3. Subjects must have completed at least 26 weeks in the PROPEL (QBGJ398-001) study before screening.
4. Subjects are able to swallow oral medication.
5. Subjects and parent(s), legal guardian(s), or caregivers are willing and able to comply with study visits and study procedures.
6. Subjects are ambulatory and able to stand without assistance.
Sex and Contraceptive/Barrier Requirements
7. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
8. If sexually active, subjects, whether male or female, must be willing to use a highly effective method of contraception while taking study drug and for 3 months after the last dose of study drug.
Informed Consent
9. Signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol, must be obtained for each subject from their parent(s) or legal guardian and signed informed consent/assent must be obtained from the subject (when applicable)
Exclusion Criteria
1. Subjects who have hypochondroplasia or short stature condition other than ACH.
2. Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
3. Current evidence of clinically significant corneal or retinal disorder/keratopathy -confirmed by ophthalmic examination.
4. Concurrent circumstance, disease or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations and/or would require treatment with a prohibited medication, and/or would place the subject at high risk for poor treatment compliance or for not completing the study.
5. History and/or current evidence of extensive ectopic tissue calcification.
6. History of malignancy.
Prior/Concomitant Therapy
7. Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature.
8. Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid therapy (ie, \>15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (ie, 2.5-10 mg/kg/day of hydrocortisone or equivalent) for over 3 weeks within 6 months of the screening visit (low-dose local preparations including inhaled steroid for asthma, intranasal sprays for allergies, and topical steroids are allowed).
9. Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening surgery or guided growth surgery during the study period. Guided growth surgery with plates removed at least 12 months prior to screening is allowed.
10. Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 or prolonged treatment (\>1 week) with medications that alter the pH of the gastrointestinal tract including antacids, H2 antagonists (eg, ranitidine, famotidine), and proton-pump inhibitors (eg, omeprazole).
11. Current evidence of endocrine alterations of calcium/phosphorus homeostasis.
Diagnostic assessments
12. Subjects who have significant abnormality in screening laboratory results.
Other Exclusions
13. Having had a fracture of the long bones (ie, extremities) or spine within 12 months prior to screening.
14. Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study.
15. Allergy or hypersensitivity to any components of the study drug.
3 Years
17 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
QED Therapeutics, a BridgeBio company
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
QED Therapeutics, Inc. Medical Director, Clinical Development
Role: STUDY_DIRECTOR
QED Therapeutics
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
QED Investigative Site
San Francisco, California, United States
QED Investigative Site
Aurora, Colorado, United States
QED Investigative Site
Baltimore, Maryland, United States
QED Investigative Site
Columbia, Missouri, United States
QED Investigative Site
Cincinnati, Ohio, United States
QED Investigative Site
Nashville, Tennessee, United States
QED Investigative Site
Madison, Wisconsin, United States
QED Investigative Site
Buenos Aires, Buenos Aires F.D., Argentina
QED Investigative Site
Parkville, Victoria, Australia
QED Investigative Site
Edmonton, Alberta, Canada
QED Investigative Site
London, Ontario, Canada
QED Investigative Site
Ottawa, Ontario, Canada
QED Investigative Site
Montreal, Quebec, Canada
QED Investigative Site
Bron, , France
QED Investigative Site
Paris, , France
QED Investigative Site
Toulouse, , France
QED Investigative Site
Rome, , Italy
QED Investigative Site
Bergen, , Norway
QED Investigative Site
Oslo, , Norway
QED Investigative Site
Singapore, , Singapore
QED Investigative Site
Málaga, , Spain
QED Investigative Site
Vitoria-Gasteiz, , Spain
QED Investigative Site
Bristol, , United Kingdom
QED Investigative Site
Glasgow, , United Kingdom
QED Investigative Site
London, , United Kingdom
QED Investigative Site
Manchester, , United Kingdom
QED Investigative Site
Sheffield, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
QBGJ398-303
Identifier Type: -
Identifier Source: org_study_id