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New treatment option for Achondroplasia
Official title Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
ClinicalTrials.gov ID: NCT07169279
What this study is testing
What is Infigratinib is provided as a single dose of minitablets for oral administration?
Infigratinib is provided as a single dose of minitablets for oral administration is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for achondroplasia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 32
You may be able to join if
- Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report...
- 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...
You likely can't join if
- 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.
- Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed...
- 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 how well it...
See the full eligibility criteria
- 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.
- 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).
- 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.
- 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...
- 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 how well it works or safety of infigratinib and/or would require treatment with a...
- 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...
- 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...
- Significant abnormality in screening laboratory results,
- Allergy or hypersensitivity to any components of the study drug.
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Baltimore, Maryland, United States
- Madison, Wisconsin, United States
- Parkville, Victoria, Australia
- Ottawa, Ontario, Canada
- Montreal, Quebec, Canada
- Oslo, Norway
- Singapore, Singapore
- Vitoria-Gasteiz, Spain
- Glasgow, United Kingdom
- Manchester, United Kingdom
- Sheffield, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 32 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Baltimore, Maryland, United States; Madison, Wisconsin, United States; Parkville, Victoria, Australia; Ottawa, Ontario, Canada; Montreal, Quebec, Canada and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.