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
NOT_YET_RECRUITING
Baltimore
Wisconsin
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
NOT_YET_RECRUITING
Madison
Other Locations
Australia
Murdoch Children's Research Institute
RECRUITING
Parkville
Canada
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
NOT_YET_RECRUITING
Montreal
Children's Hospital of Eastern Ontario Research Institute
RECRUITING
Ottawa
Norway
Paediatric Clinical Research Unit at Oslo University Hospital
NOT_YET_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)