Long-Term Safety and Efficacy of Infigratinib in Children with Hypochondroplasia
This study aims to evaluate the safety and effectiveness of Infigratinib treatment in children with Hypochondroplasia, focusing on changes in standing height and monitoring any treatment-related adverse events.
Infigratinib
Bone Diseases+15
+ Bone Diseases, Developmental
+ Carbohydrate Metabolism, Inborn Errors
Treatment Study
Summary
Study start date: April 21, 2026
Actual date on which the first participant was enrolled.This study focuses on a treatment for children with Hypochondroplasia (HCH), a condition that affects bone growth and results in short stature. The treatment being studied is a drug called Infigratinib, which is a selective tyrosine kinase inhibitor that targets fibroblast growth factor receptors 1-3. The main goal is to evaluate the long-term safety, tolerability, and effectiveness of this drug in children with HCH who have previously participated in other related studies. During this study, participants will receive Infigratinib and their progress will be closely monitored. The changes in their standing height will be measured over time and compared to both HCH and non-HCH growth charts. Additionally, any adverse events or side effects that occur during the treatment will be recorded. The aim is to understand the potential benefits and risks of using Infigratinib for the treatment of HCH.
Protocol
This section provides details of the study plan, including how the study is designed and what the study is measuring.135 patients to be enrolled
Total number of participants that the clinical trial aims to recruit.Treatment Study
Eligibility
Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.Any sex
Biological sex of participants that are eligible to enroll.From 3 to 18 Years
Range of ages for which participants are eligible to join.Healthy volunteers not allowed
If individuals who are healthy and do not have the condition being studied can participate.Conditions
Pathology
Criteria
Inclusion Criteria: * Inclusion Criteria for Participants Rolling Over from ACCEL 2/3 1. Pediatric participants with HCH who have completed ACCEL 2/3 2. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche Exclusion Criteria: * Exclusion Criteria for Participants Rolling Over from ACCEL 2/3 1. Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations 2. Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication 3. Participants who prematurely discontinued ACCEL 2/3 4. Participants who have reached final height or near final height 5. Current participation in an ongoing clinical study with a sponsor other than QED
Study Plan
Find out more about all the medication administered in this study, their detailed description and what they involve.One single intervention group is designated in this study
This study does not include a placebo group
Treatment Groups
Group I
ExperimentalStudy Objectives
Primary Objectives
Secondary Objectives
Study Centers
These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.This study has 25 locations
UCSF Benioff Children's Hospital
Oakland, United StatesOpen UCSF Benioff Children's Hospital in Google MapsChildrens Hospital Colorado
Aurora, United StatesChildren's National Hospital
Washington D.C., United StatesJohns Hopkins School of Medicine
Baltimore, United States