Extension Study of Infigratinib in Children With Achondroplasia (ACH)

NCT05145010 | Enrolling By Invitation Phase 2 DMC FDA Drug

• 1 other identifier: QBGJ398-203
• Study Type: interventional
• Target: 300
• Locations: 10 countries
• Sites: 31

Brief 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.

Conditions

Achondroplasia

Keywords

Skeletal dysplasia; Endochondral ossification; Achondroplasia (ACH); Quality of life in achondroplasia; Fibroblast growth factor receptor 3; FGFR3; Endochondral bone formation; Short-limb disproportionate stature; Bone disease; Functionality in achondroplasia; Musculoskeletal diseases; Osteochondrodysplasia; Genetic diseases; Long - term treatment; Growth; Annualized height velocity; Treatment option; Tyrosine kinase inhibitor

Outcome Measures

Primary Outcomes (2)

• Incidence of treatment emergent adverse events (TEAE) and serious TEAE

  10 years

• Changes over time in height Z-score in relation to ACH and non-ACH growth charts

  10 years

Secondary Outcomes (17)

• Changes over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth charts

  10 years

• Changes over time in body proportions

  10 years

• Changes over time in weight z-score

  10 years

• Changes overtime in BMI

  10 years

• Age of puberty onset and time to Tanner stage ≥4

  10 years

Study Arms (2)

Arm 1: Rollover subjects

EXPERIMENTAL

Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)

Drug: Infigratinib

Arm 2: Treatment naïve subjects

EXPERIMENTAL

Children naïve to infigratinib

Drug: Infigratinib

Interventions

Infigratinib DRUG

Infigratinib minitablets or sprinkle capsules to be administered by mouth. In subjects that completed a prior study with infigratinib, the starting dose will be the same as the last dose received in the prior interventional study with infigratinib. Infigratinib dose may be adjusted to 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)).

Arm 1: Rollover subjects

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Pediatric subjects with ACH who have completed a previous QED-sponsored interventional study with infigratinib.
• Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
• Subjects are able to swallow oral medication.
• Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
• 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.
• 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.

You may not qualify if:

• 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.
• Subjects who developed a medical condition that will require the initiation of treatment with a prohibited medication.
• Subjects prematurely discontinued a prior QED-sponsored interventional study with infigratinib
• Current participation in an ongoing clinical study with a sponsor other than QED
• Subjects that have reached final height or near final height.
• Subject must be 3 to \<18 years of age at screening and have growth potential.
• Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
• Subjects are able to swallow oral medication.
• Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
• Subjects have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) before study entry.
• Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
• 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.
• 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.
• Subjects who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature).
• 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.

Sponsors & Collaborators

• QED Therapeutics, a BridgeBio company: lead

Study Sites (31)

USCF Benioff Children's Hospital, Oakland

Oakland, California, 94609, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Nemours Alfred I. Dupont Hospital for Children

Wilmington, Delaware, 19803, United States

Location

Johns Hopkins University

Baltimore, Maryland, 21205, United States

Location

University of Missouri

Columbia, Missouri, 65212, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

University Hospital and UW Health Clinics

Madison, Wisconsin, 53705, United States

Location

Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan

Ciudad Autonoma Buenos Aires, Buenos Aires, C1245AAM, Argentina

Location

Murdoch Children's Hospital

Parkville, Victoria, 3052, Australia

Location

Stollery Children's Hospital

Edmonton, Alberta, T6C 2B7, Canada

Location

Children's Hospital - London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

Children's Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

Location

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Hopital Femme Mere Enfant

Lyon, 69500, France

Location

Hopital Necker-Enfants Malades

Paris, 75743, France

Location

Hopital des Enfants

Toulouse, 31300, France

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Policlinico A. Gemelli IRCCS

Roma, 00168, Italy

Location

Haukeland University Hospital

Bergen, 5021, Norway

Location

Oslo University Hospital

Oslo, 0372, Norway

Location

KK Women's and Children's Hosptial

Singapore, 229899, Singapore

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Universitario Virgen de la Victoria

Málaga, 29010, Spain

Location

Hospital Vithas San José

Vitoria-Gasteiz, Álava, 01012, Spain

Location

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, B5 6NH, United Kingdom

Location

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, BS2 8AE, United Kingdom

Location

Queen Elizabeth University Hospital

Glasgow, G12 0XH, United Kingdom

Location

St. Thomas' Hospital

London, SE1 7EH, United Kingdom

Location

Manchester University Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

Sheffield Children's Hospital

Sheffield, S10 2TH, United Kingdom

Location

MeSH Terms

Conditions

Achondroplasia; Mucopolysaccharidosis IV; Bone Diseases; Musculoskeletal Diseases; Osteochondrodysplasias; Genetic Diseases, Inborn

Interventions

infigratinib

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• QED Therapeutics SVP, Clinical Development

  QED Therapeutics

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 22, 2021
• First Posted: December 6, 2021
• Study Start: December 6, 2021
• Primary Completion (Estimated): December 1, 2031
• Study Completion (Estimated): February 1, 2032
• Last Updated: October 31, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

AR (1); CA (4); FR (3); ES (3); US (8); AU (1); SG (1); NO (2); GB (6); IT (2)