Testing Infigratinib is provided as a single against a placebo for achondroplasia
Official title: Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children With Achondroplasia
- Phase 2
- 15 groups
- Sites in Ottawa and Montreal
- Recruiting
Interventions (4)
- Medication
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 dose and number of minitablets will be calculated based on individual participant age and weight.
- Medication
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.
- Medication
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.
- Medication
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.
Canadian Sites (2)
2 of 2 recruiting
- Recruiting
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario
- Recruiting
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec
Eligibility Criteria
See who this study is looking for17 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Age 0 to 32 months (2 years and 8 months) at screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- +Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report from a certified laboratory, documenting the specific mutation.
- +Signed informed consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more →, 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
- −Allergy or hypersensitivity to any components of the study drug.
- −Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition that can affect growth.
- −Gestational age at birth \<37 weeks and/or birth weight \<2500 grams.
- −Gastroesophageal reflux disease requiring prolonged treatment (\>1 week) with prohibited medications.
- −History of fracture of a long bone or spine within 6 months prior to screeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
- −Any other significant concurrent disease or condition that, in the view of the investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more → and/or sponsorSponsorThe organisation responsible for the study overall.Read more →, would confound assessment of efficacy or safety of infigratinib and/or would require treatment with a prohibited medication (per protocolProtocolThe detailed plan a study must follow.Read more →), 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,
- −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.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more → — a middle-stage study in a few hundred people at most, looking at what the treatment does and watching for side effectsSide effectAn unwanted effect thought to be caused by the treatment itself.Read more →.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 15 groups in this study.
Groups A, B, C and D receive Infigratinib is provided as a single dose of minitablets for oral administration.
Registry label: A: SAD Cohort 1 infigratinib (2 to less than 3 years old) · B: SAD Cohort 2 infigratinib (1 to less than 2 years old) · C: SAD Cohort 3 infigratinib (6 months to less than 1 year old) · D: SAD Cohort 4 infigratinib (0 to less than 6 months old)
Groups E, F, G, H and L receive Infigratinib is provided as sprinkle capsules for daily oral administration.
Registry label: E: Phase 2 Cohort 1 infigratinib (2 to less than 3 years old) · F: Phase 2 Cohort 2 infigratinib (1 to less than 2 years old) · G: Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old) · H: Phase 2 Cohort 4 infigratinib (0 to less than 6 months) · L: Open- label Extension infigratinib (0 months to 3 years [+6 months old])
Groups I, J and K receive Infigratinib or placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → comparatorControl groupThe group a new treatment is measured against.Read more → is provided as sprinkle capsules for daily oral administration.
Registry label: I: Phase 2B Cohort 1 infigratinib (2 to less than 3 years old) · J: Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old) · K: Phase 2B Cohort 3 infigratinib (0 to less than 6 months old)
Groups M, N and O, the placebo group, receive Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration.
Registry label: M: Phase 2B Cohort 1 Placebo (2 to less than 3 years old) · N: Phase 2B Cohort 2 placebo (6 months to less than 2 years old) · O: Phase 2B Cohort 3 Placebo (0 to less than 6 months old)
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
Neither you nor the study team would know which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 0 years to 32 months.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 77 people.
The study is currently expected to finish around March 2032.
The main measurement is taken over: 2 weeks.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Single Ascending Dose Portion: Identify the dose of infigratinib to be used in each age cohortCohortA group of participants sharing a characteristic, followed together.Read more → of the Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more → potion of the study (by assessing safety and PKPharmacokineticsThe study of how the body absorbs, distributes, and clears a treatment.Read more → of infigratinib and its active metabolites) — measured over 2 weeks.
Phase 2 Portion: Confirm the doses to be used in each age cohort in the Phase 2b portion of the study (by assessing safety and PK of infigratinib and its active metabolites) — measured over 52 weeks.
Phase 2b Portion: Evaluate the safety and efficacy of infigratinib in infants and children < 3 years old with ACH (by assessing AEAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more →'s & SAESerious adverse eventAn adverse event meeting a formal severity threshold, such as requiring hospital admission.Read more →'s) — measured over 52 weeks.
Extension Portion: Evaluate the safety and efficacy of infigratinib in participants who completed the Phase 2 or Phase 2b portion of the study until they have reached 3 years old (+6 months) (by assessing AE's and SAE's) — measured over 3 years and 6 months.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT07169279 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About Infigratinib is provided as a single dose of minitablets for oral administrationDrug
* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria. * The dose and number of minitablets will be calculated based on individual participant age and weight.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Infigratinib is provided as sprinkle capsules for daily oral administrationDrug
* 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.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administrationDrug
* 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.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Infigratinib is provided as sprinkle capsules for daily oral administrationDrug
* 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.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 0 years to 32 months.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
Neither you nor the study team would know which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 0 years to 32 months.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: 2 weeks.
The study as a whole is currently expected to finish around 2032-03.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 77 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 2 locations, including: Ottawa, Ontario, Canada; Montreal, Quebec, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \< 3 years old with ACH at the selected dose.
Think this trial might be right for you?
Complete a quick intake form and we will match you with this and other relevant trials based on your medical profile.
See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.