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A study to investigate the safety and efficacy of infigratinib compared with placebo in infants and young children with achondroplasia
EU CTIS ID: 2024-518072-31-00
What this study is testing
SAD: To identify the dose of infigratinib to be used in the Phase 2 portion of the study, based on safety and exposure of single ascending doses of infigratinib. PH2: To confirm the doses to be used in each age cohort based on safety and PK. Ph2b: To evaluate the safety and efficacy of infigratinib in infant and young children <3 years old with ACH. Extension: To 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)
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
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 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.
- In breastfeeding infants/young children, the mother must be willing and able to discontinue treatment with a drug that can be harmful to the participant, as this could confound the assessment of safety (ie, risk of developing an ADR to medication exposure via breast milk) or could impact the PK of infigratinib (a noncomprehensive list of prohibited medications is included in Appendix 7 [Section 10.6]). If discontinuation of treatment is not possible, the mother must be willing and able to stop the nursing of the participant.
You likely can't join if
- 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 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, and/or would place the participant at high risk for poor treatment compliance or for failure to complete the study.
- Gestational age at birth <37 weeks and/or birth weight <2500 grams.
- 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.
- Current evidence of endocrine alterations of calcium/phosphorus homeostasis, including serum calcium and/or phosphorus outside of the normal range for age at screening.
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.
- In breastfeeding infants/young children, the mother must be willing and able to discontinue treatment with a drug that can be harmful to the participant, as this could confound the assessment of safety (ie, risk of developing an ADR to medication exposure via breast milk) or could impact the PK of infigratinib (a noncomprehensive list of prohibited medications is included in Appendix 7 [Section 10.6]). If discontinuation of treatment is not possible, the mother must be willing and able to stop the nursing of the participant.
- 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).
- 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 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, and/or would place the participant at high risk for poor treatment compliance or for failure to complete the study.
- Gestational age at birth <37 weeks and/or birth weight <2500 grams.
- 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.
- Current evidence of endocrine alterations of calcium/phosphorus homeostasis, including serum calcium and/or phosphorus outside of the normal range for age at screening.
- Allergy or hypersensitivity to any components of the study drug.
- History or presence of ectopic tissue X (based on participant medical history).
- History or presence of malignancy (based on participant medical history).
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- Singapore
- United States
- Australia
- 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, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; Singapore; United States; Australia; United Kingdom. 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 EU CTIS; the study team decides eligibility.