Extension Study of Infigratinib in Children With Achondroplasia (ACH)

August 19, 2023 updated by: QED Therapeutics, Inc.

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

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 the PROPEL study (Protocol QBGJ398 001) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

280

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Recruiting
        • Murdoch Children's Hospital
        • Contact:
        • Principal Investigator:
          • Ravi Savarirayan, MD
  • France
      • Lyon, France
        • Recruiting
        • Hopital Femme Mere Enfant
        • Contact:
        • Principal Investigator:
          • Massililiano Rossi, MD
      • Paris, France
        • Recruiting
        • Hôpital Necker-Enfants Malades
      • Toulouse, France
        • Recruiting
        • Hopital des Enfants
  • Spain
      • Madrid, Spain
        • Recruiting
        • Hospital Universitario La Paz
      • Málaga, Spain
        • Recruiting
        • Hospital Universitario Virgen De La Victoria
    • Álava
      • Vitoria-Gasteiz, Álava, Spain, 01012
        • Recruiting
        • Hospital Vithas San Jose
        • Contact:
        • Principal Investigator:
          • Josep M de Bergua, MD
  • United Kingdom
      • Glasgow, United Kingdom, G12 0XH
        • Recruiting
        • Queen Elizabeth University Hospital
        • Principal Investigator:
          • Helen McDevitt, MD
        • Contact:
      • London, United Kingdom
        • Recruiting
        • St. Thomas' Hospital
      • Manchester, United Kingdom
        • Recruiting
        • Manchester University Children's Hospital
      • Sheffield, United Kingdom, S10 2TH
        • Recruiting
        • Sheffield Children's Hospital
        • Contact:
        • Principal Investigator:
          • Paul Arundel, MD
  • United States
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Completed
        • Nemours Alfred I. Dupont Hospital for Children
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Recruiting
        • Vanderbilt University Medical Center
        • Contact:
        • Principal Investigator:
          • John A Phillips, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Rollover Subjects Inclusion Criteria:

  1. Pediatric subjects with ACH who have completed study activities in a previous QED-sponsored interventional study with infigratinib.
  2. Subjects and parent(s) or legally authorized representatives (LARs) are willing and able to comply with study visits and study procedures.
  3. Subjects are able to swallow oral medication.
  4. In girls ≥10 years of age or girls of any age who have experienced menarche, having a negative pregnancy test.
  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 LAR and the subject's assent, when applicable, before any study-specific activity is performed.

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. 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 who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
  3. Subjects have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) before study entry.
  4. In girls ≥10 years of age or girls of any age who have experienced menarche, having a negative pregnancy test.
  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 LAR 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 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.
  6. Subjects who have significant abnormality in screening laboratory results.
  7. Subjects who have had a fracture within 12 months of screening.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Arm 2: Treatment naïve subjects
Children naïve to infigratinib
Drug: Infigratinib
Infigratinib minitablets 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 the dose identified from the dose escalation portion of the Phase 2 study (PROPEL 2).
Drug: Infigratinib
Infigratinib minitablets to be administered by mouth. Starting dose for the subjects naive to infigratinib will be the one identified during QBGJ398-201 (PROPEL 2).
Experimental: Arm 1: Rollover subjects
Children who have completed QED-sponsored interventional study with infigratinib
Drug: Infigratinib
Infigratinib minitablets 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 the dose identified from the dose escalation portion of the Phase 2 study (PROPEL 2).
Drug: Infigratinib
Infigratinib minitablets to be administered by mouth. Starting dose for the subjects naive to infigratinib will be the one identified during QBGJ398-201 (PROPEL 2).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Time Frame: 10 years
10 years
Changes over time in height Z-score in relation to ACH and non-ACH growth charts
Time Frame: 10 years
10 years

Secondary Outcome Measures

Outcome Measure
Time Frame
Changes over time in absolute height velocity, expressed as height velocity z-score in relation to ACH and non ACH growth chart
Time Frame: 10 years
10 years
Changes over time in body proportions
Time Frame: 10 years
10 years
Changes over time in weight z-score
Time Frame: 10 years
10 years
Changes overtime in BMI
Time Frame: 10 years
10 years
Age of puberty onset and time to Tanner stage ≥4
Time Frame: 10 years
10 years
Changes over time in number of episodes of otitis media per year
Time Frame: 10 years
10 years
Changes over time in number of episodes and/or severity of sleep apnea
Time Frame: 10 years
10 years
Changes over time in range of motion (hip, knee and elbow)
Time Frame: 10 years
10 years
Changes over time in skeletal abnormalities of the lower extremities and spine
Time Frame: 10 years
10 years
Changes in health-related qQuality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL) and Quality of Life in Short Stature Youth questionnaire (QoLISSY)
Time Frame: 10 years
10 years
Changes in overall body pain as assessed by Numeric Rating Scale for pain (Pain-NRS)
Time Frame: 10 years
10 years
Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)
Time Frame: 10 years
10 years
Changes in cognitive functions assessed by age-appropriate computerized tests
Time Frame: 10 years
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

QED Therapeutics, Inc.

Investigators

  • Study Director: QED Therapeutics SVP, Clinical Development, QED Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 6, 2021

Primary Completion (Estimated)

December 1, 2031

Study Completion (Estimated)

February 1, 2032

Study Registration Dates

First Submitted

November 22, 2021

First Submitted That Met QC Criteria

November 22, 2021

First Posted (Actual)

December 6, 2021

Study Record Updates

Last Update Posted (Actual)

August 22, 2023

Last Update Submitted That Met QC Criteria

August 19, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • QBGJ398-203

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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