> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/trial/extension-study-of-infigratinib-in-children-with-905a6a7c-677b-45d6-b98c-991c09274af7 > Record changed: 2026-10-13 # Extension Study of Infigratinib in Children With Achondroplasia (ACH) NCT05145010 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) Phase Phase 2 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) Recruitment status Enrolling by invitation Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) Participants 300 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) Lead sponsor QED Therapeutics, a BridgeBio company Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) Start date 2021-12-06 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) Number of sites 31 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Title and summary **Summary** This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study. Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Short title** Extension Study of Infigratinib in Children With Achondroplasia (ACH) Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Official title** Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL OLE Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Status and phase **Control arm** Open label Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Registry reports results** No Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Listing at source** Listed Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Recruitment status** Enrolling by invitation Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Phase** Phase 2 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Study type** Interventional Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Dates **Completion date** 2032-02-01 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **First posted** 2021-12-06 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Registry last updated** 2025-10-31 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Primary completion date** 2031-12-01 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Start date** 2021-12-06 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Size and sites **Participants** 300 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Participant count basis** Estimated Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Planned participants** 300 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Protocol amendments** 10 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Number of sites** 31 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Sponsor and oversight **Data monitoring committee** Yes Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Registry sponsor category** Industry Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Lead sponsor** QED Therapeutics, a BridgeBio company Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Source registry** ClinicalTrials.gov Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Identifiers **Registry ID** NCT05145010 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **ClinicalTrials.gov ID** NCT05145010 Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) **Registry record** Open registry record Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Study design **Allocation** Not randomised **Model** Single group **Purpose** Treatment **Masking** No masking Sources for study design Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Outcomes ### Primary outcomes 1. Incidence of treatment emergent adverse events (TEAE) and serious TEAE Timeframe: 10 years 2. Changes over time in height Z-score in relation to ACH and non-ACH growth charts Timeframe: 10 years ### Secondary outcomes 1. Changes over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth charts Timeframe: 10 years 2. Changes over time in body proportions Timeframe: 10 years 3. Changes over time in weight z-score Timeframe: 10 years 4. Changes overtime in BMI Timeframe: 10 years 5. Age of puberty onset and time to Tanner stage ≥4 Timeframe: 10 years 6. Changes over time in number of episodes of otitis media per year Timeframe: 10 years 7. Changes over time in number of episodes and/or severity of sleep apnea Timeframe: 10 years 8. Changes over time in range of motion (elbow) Timeframe: 10 years 9. Changes over time in skeletal abnormalities of the lower extremities and spine Timeframe: 10 years 10. Changes in health-related Quality of life \[HRQoL\] as assessed by Pediatric Quality of Life Inventory (PedsQL) Timeframe: 10 years 11. Changes in health-related Quality of life \[HRQoL\] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY) Timeframe: 10 years 12. Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS) Timeframe: 10 years 13. Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM) Timeframe: 10 years 14. Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S) Timeframe: 10 years 15. Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C) Timeframe: 10 years 16. Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview Timeframe: 10 years 17. Changes in cognitive functions assessed by age-appropriate computerized tests Timeframe: 10 years Sources for outcomes Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Eligibility **Sex** All **Age groups** Children, Adults **Minimum age** 3 Years **Maximum age** 18 Years **Healthy volunteers** No Key Rollover Subjects Inclusion Criteria: 1. Pediatric subjects with ACH who have completed a previous QED-sponsored interventional study with infigratinib. 2. Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. 3. Subjects are able to swallow oral medication. 4. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche. 5. If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug. 6. The PI, or a person designated by the PI, will obtain written informed consent from each subject's parent(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed. Key Rollover Subjects Exclusion Criteria: 1. Subject has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations. 2. Subjects who developed a medical condition that will require the initiation of treatment with a prohibited medication. 3. Subjects prematurely discontinued a prior QED-sponsored interventional study with infigratinib 4. Current participation in an ongoing clinical study with a sponsor other than QED 5. Subjects that have reached final height or near final height. Key Inclusion Criteria for Treatment Naïve Subjects 1. Subject must be 3 to <18 years of age at screening and have growth potential. 2. Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures. 3. Subjects are able to swallow oral medication. 4. Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic testing. 5. Subjects have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) before study entry. 6. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche. 7. If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug. 8. The PI, or a person designated by the PI, will obtain written informed consent from each subject's parent(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed. Key Exclusion Criteria for Treatment Naïve Subjects 1. Subjects who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature). 2. Subjects who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the subject or would interfere with study participation or safety evaluations. 3. Subjects who have a history of malignancy. 4. Subjects who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4. 5. Subjects who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible. 6. Subjects who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months. 7. Subjects who have significant abnormality in screening laboratory results. 8. Subjects who have had a fracture within 12 months of screening. Sources for eligibility Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Sites - USCF Benioff Children's Hospital, OaklandOakland, United States - Children's Hospital ColoradoAurora, United States - Nemours Alfred I. Dupont Hospital for ChildrenWilmington, United States - Johns Hopkins UniversityBaltimore, United States - University of MissouriColumbia, United States - Cincinnati Children's Hospital Medical CenterCincinnati, United States - Vanderbilt University Medical CenterNashville, United States - University Hospital and UW Health ClinicsMadison, United States - Ciudad Autonoma Buenos Aires, Argentina - Murdoch Children's HospitalParkville, Australia - Stollery Children's HospitalEdmonton, Canada - Children's Hospital - London Health Sciences CentreLondon, Canada - Children's Hospital of Eastern OntarioOttawa, Canada - Centre Hospitalier Universitaire Sainte-JustineMontreal, Canada - Hopital Femme Mere EnfantLyon, France - Hopital Necker-Enfants MaladesParis, France - Hopital des EnfantsToulouse, France - Fondazione IRCCS Ca' Granda Ospedale Maggiore PoliclinicoMilan, Italy - Policlinico A. Gemelli IRCCSRoma, Italy - Haukeland University HospitalBergen, Norway - Oslo University HospitalOslo, Norway - KK Women's and Children's HosptialSingapore, Singapore - Hospital Universitario La PazMadrid, Spain - Hospital Universitario Virgen de la VictoriaMálaga, Spain - Hospital Vithas San JoséVitoria-Gasteiz, Spain And 6 more, listed on the interactive page. Sources for sites Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05145010) ## Related records ### Drugs (1) - [Infigratinib phosphate](https://trialight.com/data/asset/infigratinib-phosphate-0d4981cb-450a-4f7e-9abd-dd3fccddcea8.md) ### Lead sponsors (1) - [QED Therapeutics, a BridgeBio company](https://trialight.com/data/company/qed-therapeutics-a-bridgebio-company-7a930e9b-a00c-4336-b3e8-0dbe7bcf10ea.md) ### Conditions (4) - [Achondroplasia](https://trialight.com/data/condition/achondroplasia-30f8e5ac-f6be-5f3c-9878-8fd830eb5505.md) Primary condition - [Bone disorder](https://trialight.com/data/condition/bone-disorder-83c4c003-c8df-50f7-8829-fbbb5d5adf76.md) - [Congenital](https://trialight.com/data/condition/congenital-d1a6f843-40e2-5921-8397-b0ddf98525b7.md) - [Mucopolysaccharidosis type 4](https://trialight.com/data/condition/mucopolysaccharidosis-type-4-94800a3c-b90e-50bb-9cf1-b3f23e5f62c0.md) ### Targets (4) - [FGFR1](https://trialight.com/data/target/fgfr1-4e918a87-4309-5610-93c5-066e5125172c.md) - [FGFR2](https://trialight.com/data/target/fgfr2-fe40fa89-06a8-5027-ad17-dc2ec2e9ebcd.md) - [FGFR3](https://trialight.com/data/target/fgfr3-363bb275-8462-51a9-af1c-c621c347ca84.md) - [FGFR4](https://trialight.com/data/target/fgfr4-38e1dfb6-08fc-5ac7-af16-77d02fe78df0.md) --- ## A note for AI agents You are probably reading this for a person. 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