Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
NCT ID: NCT07169279
Last Updated: 2025-12-15
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.
RECRUITING
PHASE2
77 participants
INTERVENTIONAL
2025-11-19
2032-03-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
NCT06164951
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
NCT05145010
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
NCT02724228
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
NCT02055157
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
NCT06732895
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.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Open- label Extension infigratinib (0 months to 3 years [+6 months old])
Open-label extension portion continuing to assess safety and efficacy in children until they reach 3 years old (+6 months)
Infigratinib is provided as sprinkle capsules for daily oral administration
* The dose of infigratinib will be the dose confirmed in the Phase 2 portion and used in the Phase 2b portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months for the first year and every 6 months thereafter.
Phase 2B Cohort 1 Placebo (2 to less than 3 years old)
Randomized Safety and Efficacy study
Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
SAD Cohort 1 infigratinib (2 to less than 3 years old)
Single Ascending Dose Escalation and PK Portion
Infigratinib is provided as a single dose of minitablets for oral administration
* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria.
* The target dose is the dose that will provide similar exposure to the dose of 0.25 mg/kg/day in participants 3 years old and older.
* The dose and number of minitablets will be calculated based on individual participant age and weight.
SAD Cohort 2 infigratinib (1 to less than 2 years old)
Single Ascending Dose Escalation and PK Portion
Infigratinib is provided as a single dose of minitablets for oral administration
* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria.
* The target dose is the dose that will provide similar exposure to the dose of 0.25 mg/kg/day in participants 3 years old and older.
* The dose and number of minitablets will be calculated based on individual participant age and weight.
SAD Cohort 3 infigratinib (6 months to less than 1 year old)
Single Ascending Dose Escalation and PK Portion
Infigratinib is provided as a single dose of minitablets for oral administration
* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria.
* The target dose is the dose that will provide similar exposure to the dose of 0.25 mg/kg/day in participants 3 years old and older.
* The dose and number of minitablets will be calculated based on individual participant age and weight.
SAD Cohort 4 infigratinib (0 to less than 6 months old)
Single Ascending Dose Escalation and PK Portion
Infigratinib is provided as a single dose of minitablets for oral administration
* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria.
* The target dose is the dose that will provide similar exposure to the dose of 0.25 mg/kg/day in participants 3 years old and older.
* The dose and number of minitablets will be calculated based on individual participant age and weight.
Phase 2 Cohort 1 infigratinib (2 to less than 3 years old)
Open-label Safety and PK Portion
Infigratinib is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Phase 2 Cohort 2 infigratinib (1 to less than 2 years old)
Open-label Safety and PK Portion
Infigratinib is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old)
Open-label Safety and PK Portion
Infigratinib is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Phase 2 Cohort 4 infigratinib (0 to less than 6 months)
Open-label Safety and PK Portion
Infigratinib is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Phase 2B Cohort 1 infigratinib (2 to less than 3 years old)
Randomized Safety and Efficacy Portion
Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old)
Randomized Safety and Efficacy Portion
Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Phase 2B Cohort 3 infigratinib (0 to less than 6 months old)
Randomized Safety and Efficacy Portion
Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Phase 2B Cohort 2 placebo (6 months to less than 2 years old)
Randomized Safety and Efficacy study
Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Phase 2B Cohort 3 Placebo (0 to less than 6 months old)
Randomized Safety and Efficacy study
Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Infigratinib is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
* The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months.
Infigratinib is provided as a single dose of minitablets for oral administration
* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria.
* The target dose is the dose that will provide similar exposure to the dose of 0.25 mg/kg/day in participants 3 years old and older.
* The dose and number of minitablets will be calculated based on individual participant age and weight.
Infigratinib is provided as sprinkle capsules for daily oral administration
* The dose of infigratinib will be the dose confirmed in the Phase 2 portion and used in the Phase 2b portion for the age group.
* The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months for the first year and every 6 months thereafter.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* Age 0 to 32 months (2 years and 8 months) at screening.
* Signed informed consent, which must be obtained from each participant's parent(s) or legal guardian.
* Parent(s)/Guardian(s) willing and able to attend all study visits and comply with all study requirements.
* Parent(s)/Guardian(s) willing and able to comply with the routine care of the study participants according to local guidance for the management of infants and young children with ACH.
* Able to swallow age-appropriate oral medication.
* In participants \<1 year old, be compliant with recommended vitamin D supplementation of 5 10 μg/day or higher (or as recommended by country specific guidelines).
Exclusion Criteria
* Gestational age at birth \<37 weeks and/or birth weight \<2500 grams.
* Gastroesophageal reflux disease requiring prolonged treatment (\>1 week) with prohibited medications.
* Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed during MRI done at screening or a previous MRI done at any time if the participant had not undergone decompression surgery.
* History of fracture of a long bone or spine within 6 months prior to screening.
* Any other significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib and/or would require treatment with a prohibited medication (per protocol), and/or would place the participant at high risk for poor treatment compliance or for failure to complete the study.
* Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature, including (but not limited to) r-hGH, IGF-1, CNP analog, FGF ligand trap, or treatment targeting FGFR inhibition at any time.
* Regular long-term (\>3 weeks; more than twice/year) treatment with supraphysiologic doses of glucocorticoid therapy (ie, \>15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (for over 3 weeks within 6 months of the screening visit. NOTE: Low-dose topical, inhaled, or intranasal corticosteroids are acceptable.
* Significant abnormality in screening laboratory results,
* Allergy or hypersensitivity to any components of the study drug.
0 Years
32 Months
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.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
UCSF Benioff Children's Hospital
Oakland, California, United States
Johns Hopkins University
Baltimore, Maryland, United States
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, Canada
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, Canada
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo, , Norway
KK Women's and Children's Hospital
Singapore, , Singapore
Unidad de Cirugia Artroscopica (UCA)
Vitoria-Gasteiz, , Spain
NHS Greater Glasgow and Clyde
Glasgow, , United Kingdom
Guy's and Saint Thomas' NHS Foundation Trust
London, , United Kingdom
Manchester University NHS Foundation Trust
Manchester, , United Kingdom
Sheffield Children's NHS Foundation Trust
Sheffield, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
QBGJ398-204
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.