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Clinical Trials in the UK / NCT07169279
Recruiting Phase 2

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

NCT07169279 · tracked via the Priya Life Science UK tracker
Sponsor
QED Therapeutics, a BridgeBio company
Phase
Phase 2
Started
2025-11-19
Last updated
2026-07-13

Condition(s) studied

Achondroplasia

Investigational drug(s) / intervention(s)

Infigratinib is provided as a single dose of minitablets for oral administrationInfigratinib is provided as sprinkle capsules for daily oral administrationInfigratinib or placebo comparator is provided as sprinkle capsules for daily oral administrationInfigratinib is provided as sprinkle capsules for daily oral administration

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.

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 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.

Study summary

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.

Eligibility

Sex
ALL
Min age
0 Years
Max age
32 Months
Healthy volunteers
No
Inclusion 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). Exclusion Criteria: * 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 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.

Primary outcome measure(s)

  • Single Ascending Dose Portion: Identify the dose of infigratinib to be used in each age cohort of the Phase 2 potion of the study (by assessing safety and PK of infigratinib and its active metabolites) — 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). — 52 weeks
  • Phase 2b Portion: Evaluate the safety and efficacy of infigratinib in infants and children < 3 years old with ACH (by assessing AE's & SAE's) — 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) — 3 years and 6 months

Trial sites (12)

FacilityCityRegionStatus
UCSF Benioff Children's Hospital Oakland California Recruiting
Johns Hopkins University Baltimore Maryland Recruiting
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic Madison Wisconsin Recruiting
Murdoch Children's Research Institute Parkville Victoria Recruiting
Children's Hospital of Eastern Ontario Research Institute Ottawa Ontario Recruiting
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine Montreal Quebec Recruiting
Paediatric Clinical Research Unit at Oslo University Hospital Oslo Norway Recruiting
KK Women's and Children's Hospital Singapore Singapore Recruiting
Unidad de Cirugía Artroscópica (UCA) Vitoria-Gasteiz Spain Recruiting
NHS Greater Glasgow and Clyde Glasgow United Kingdom Recruiting
Manchester University NHS Foundation Trust Manchester United Kingdom Recruiting
Sheffield Children's NHS Foundation Trust Sheffield United Kingdom Recruiting

More QED Therapeutics, a BridgeBio company trials in the UK

Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia Enrolling by invitation
An Interventional Study of Infigratinib in Children With Hypochondroplasia Enrolling by invitation
Extension Study of Infigratinib in Children With Achondroplasia (ACH) Enrolling by invitation
Prospective Clinical Assessment Study in Children With Hypochondroplasia Recruiting

Other trials for the same condition

A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) Active, not recruiting
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia Active, not recruiting
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia Recruiting
A Phase 2 Clinical Trial to Evaluate Efficacy, Safety, and Tolerability of Navepegritide in Combination With Lonapegsomatropin in Children With Achondroplasia Active, not recruiting
Extension Study of Infigratinib in Children With Achondroplasia (ACH) Enrolling by invitation
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07169279 on ClinicalTrials.gov ↗ ← All trials in the UK