Extension Study of Infigratinib in Children With Achondroplasia (ACH)

NCT ID: NCT05145010

Last Updated: 2025-10-31

Basic Information

• Recruitment Status: ENROLLING_BY_INVITATION
• Clinical Phase: PHASE2
• Total Enrollment: 300 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-12-06
• Study Completion Date: 2032-02-01

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Arm 1: Rollover subjects

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

Group Type: EXPERIMENTAL

Infigratinib

Intervention Type: 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 2: Treatment naïve subjects

Children naïve to infigratinib

Group Type: EXPERIMENTAL

Infigratinib

Intervention Type: DRUG

Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the subjects naïve to Infigratinib will be 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)).

Interventions

Infigratinib

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)).

Intervention Type: DRUG

Infigratinib

Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the subjects naïve to Infigratinib will be 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)).

Intervention Type: DRUG

Eligibility Criteria

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.

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.

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.

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.

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

QED Therapeutics, Inc., a Bridgebio company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

QED Therapeutics SVP, Clinical Development

Role: STUDY_DIRECTOR

QED Therapeutics

Locations

USCF Benioff Children's Hospital, Oakland

Oakland, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nemours Alfred I. Dupont Hospital for Children

Wilmington, Delaware, United States

Johns Hopkins University

Baltimore, Maryland, United States

University of Missouri

Columbia, Missouri, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

University Hospital and UW Health Clinics

Madison, Wisconsin, United States

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

Ciudad Autonoma Buenos Aires, Buenos Aires, Argentina

Murdoch Children's Hospital

Parkville, Victoria, Australia

Stollery Children's Hospital

Edmonton, Alberta, Canada

Children's Hospital - London Health Sciences Centre

London, Ontario, Canada

Children's Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, Canada

Hopital Femme Mere Enfant

Lyon, , France

Hopital Necker-Enfants Malades

Paris, , France

Hopital des Enfants

Toulouse, , France

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Policlinico A. Gemelli IRCCS

Roma, , Italy

Haukeland University Hospital

Bergen, , Norway

Oslo University Hospital

Oslo, , Norway

KK Women's and Children's Hosptial

Singapore, , Singapore

Hospital Universitario La Paz

Madrid, , Spain

Hospital Universitario Virgen de la Victoria

Málaga, , Spain

Hospital Vithas San José

Vitoria-Gasteiz, Álava, Spain

Birmingham Women's and Children's NHS Foundation Trust

Birmingham, , United Kingdom

University Hospitals Bristol and Weston NHS Foundation Trust

Bristol, , United Kingdom

Queen Elizabeth University Hospital

Glasgow, , United Kingdom

St. Thomas' Hospital

London, , United Kingdom

Manchester University Children's Hospital

Manchester, , United Kingdom

Sheffield Children's Hospital

Sheffield, , United Kingdom

Countries

United States; Argentina; Australia; Canada; France; Italy; Norway; Singapore; Spain; United Kingdom

Other Identifiers

QBGJ398-203

Identifier Type: -

Identifier Source: org_study_id