ClinicalTrials.Veeva

Menu

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

Q

QED Therapeutics

Status and phase

Invitation-only
Phase 2

Conditions

Achondroplasia

Treatments

Drug: Infigratinib

Study type

Interventional

Funder types

Industry

Identifiers

NCT05145010
QBGJ398-203

Details and patient eligibility

About

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.

Enrollment

300 estimated patients

Sex

All

Ages

3 to 18 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Single Group Assignment

Masking

None (Open label)

300 participants in 2 patient groups

Arm 1: Rollover subjects
Experimental group
Description:
Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)
Treatment:
Drug: Infigratinib
Drug: Infigratinib
Arm 2: Treatment naïve subjects
Experimental group
Description:
Children naïve to infigratinib
Treatment:
Drug: Infigratinib
Drug: Infigratinib

Trial contacts and locations

18

Loading...

Central trial contact

QED Therapeutics SVP, Clinical Development

Data sourced from clinicaltrials.gov

Clinical trials

Find clinical trialsTrials by location
© Copyright 2024 Veeva Systems