Recruiting
Phase 2

Infigratinib

Sponsor:

QED Therapeutics, a BridgeBio company

Code:

NCT07169279

Conditions

Achondroplasia

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

Interventions

Infigratinib is provided as a single dose of minitablets for oral administration

Infigratinib is provided as sprinkle capsules for daily oral administration

Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration

Infigratinib is provided as sprinkle capsules for daily oral administration

Study Details

Brief 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.

Conditions

Achondroplasia

Study ID

NCT07169279

Start date

Nov 19, 2025

Status verified date

Jul, 2026

Completion date

Mar, 2032

Anticipated

Primary completion date

Mar, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 2

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

77 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: SAD Cohort 1 infigratinib (2 to less than 3 years old)

Single Ascending Dose Escalation and PK Portion

experimental: SAD Cohort 2 infigratinib (1 to less than 2 years old)

Single Ascending Dose Escalation and PK Portion

experimental: SAD Cohort 3 infigratinib (6 months to less than 1 year old)

Single Ascending Dose Escalation and PK Portion

experimental: SAD Cohort 4 infigratinib (0 to less than 6 months old)

Single Ascending Dose Escalation and PK Portion

experimental: Phase 2 Cohort 1 infigratinib (2 to less than 3 years old)

Open-label Safety and PK Portion

experimental: Phase 2 Cohort 2 infigratinib (1 to less than 2 years old)

Open-label Safety and PK Portion

experimental: Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old)

Open-label Safety and PK Portion

experimental: Phase 2 Cohort 4 infigratinib (0 to less than 6 months)

Open-label Safety and PK Portion

experimental: Phase 2B Cohort 1 infigratinib (2 to less than 3 years old)

Randomized Safety and Efficacy Portion

experimental: Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old)

Randomized Safety and Efficacy Portion

experimental: Phase 2B Cohort 3 infigratinib (0 to less than 6 months old)

Randomized Safety and Efficacy Portion

experimental: 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)

placebo comparator: Phase 2B Cohort 1 Placebo (2 to less than 3 years old)

Randomized Safety and Efficacy study

placebo comparator: Phase 2B Cohort 2 placebo (6 months to less than 2 years old)

Randomized Safety and Efficacy study

placebo comparator: Phase 2B Cohort 3 Placebo (0 to less than 6 months old)

Randomized Safety and Efficacy study

Interventions

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.

Primary outcome measure

  • 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) [ Time Frame: 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). [ Time Frame: 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) [ Time Frame: 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) [ Time Frame: 3 years and 6 months ]

Central Contacts and Locations

Central contacts

QED Therapeutics Inc.

18772805655medinfo@qedtx.com

Locations

UCSF Benioff Children's Hospital

Recruiting

Oakland, California, United States, 94609

Johns Hopkins University

Recruiting

Baltimore, Maryland, United States, 21218

University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

Recruiting

Madison, Wisconsin, United States, 53705

Children's Hospital of Eastern Ontario Research Institute

Recruiting

Ottawa, Ontario, Canada, K1H 8L1

Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine

Recruiting

Montreal, Quebec, Canada, H3T 1C5

More Information

Sponsor

QED Therapeutics, a BridgeBio company

Last update posted

Jul 13, 2026

Last verified

Jul, 2026

Keywords

  • skeletal dysplasia
  • endochondral ossification
  • achondroplasia
  • ACH
  • Shortened proximal limbs
  • fibroblast growth factor receptor 3
  • FGFR3
  • disproportionate short stature
  • quality of life
  • dwarfism
  • bone diseases
  • musculoskeletal diseases
  • osteochondrodysplasia
  • functional abilities
  • annualized growth velocity
  • annualized height velocity
  • growth
  • growth disorder
  • congenital
  • AHV
  • AGV
  • infant and toddler
  • endochondral bone formation

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by QED Therapeutics, a BridgeBio company on 2026-07-13.