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
Status: Recruiting
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Maximum Age: 2
Healthy Volunteers: f
View:
• 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).
Locations
United States
California
UCSF Benioff Children's Hospital
RECRUITING
Oakland
Maryland
Johns Hopkins University
RECRUITING
Baltimore
Wisconsin
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
RECRUITING
Madison
Other Locations
Australia
Murdoch Children's Research Institute
RECRUITING
Parkville
Canada
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
RECRUITING
Montreal
Children's Hospital of Eastern Ontario Research Institute
RECRUITING
Ottawa
Norway
Paediatric Clinical Research Unit at Oslo University Hospital
RECRUITING
Oslo
Singapore
KK Women's and Children's Hospital
RECRUITING
Singapore
Spain
Unidad de Cirugia Artroscopica (UCA)
NOT_YET_RECRUITING
Vitoria-gasteiz
United Kingdom
NHS Greater Glasgow and Clyde
NOT_YET_RECRUITING
Glasgow
Guy's and Saint Thomas' NHS Foundation Trust
NOT_YET_RECRUITING
London
Manchester University NHS Foundation Trust
NOT_YET_RECRUITING
Manchester
Sheffield Children's NHS Foundation Trust
NOT_YET_RECRUITING
Sheffield
Contact Information
Primary
QED Therapeutics Inc.
medinfo@qedtx.com
18772805655
Time Frame
Start Date:2025-11-19
Estimated Completion Date:2032-03
Participants
Target number of participants:77
Treatments
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)