Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2

Who is this study for? Child patients with Achondroplasia
What treatments are being studied? Infigratinib
Status: Completed
Location: See all (19) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Maximum Age: 11
Healthy Volunteers: f
View:

• Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable).

• Diagnosis of ACH, documented clinically and confirmed by genetic testing.

• At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.

• Ambulatory and able to stand without assistance

• Able to swallow oral medication.

Locations
United States
California
UCSF Benioff Children's Hospital
Oakland
Delaware
Nemours Alfred I. Dupont Hospital for Children
Wilmington
Maryland
Johns Hopkins School of Medicine
Baltimore
Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati
Tennessee
Vanderbilt University Medical Center
Nashville
Other Locations
Australia
Murdoch Children's Hospital
Parkville
Canada
Stollery Children's Hospital
Edmonton
France
Hopital Femme Mere Enfant
Lyon
Hopital Necker-Enfants Malades
Paris
Hopital des Enfants
Toulouse
Spain
Hospital Universitario La Paz
Madrid
Hospital Universitario Virgen de la Victoria
Málaga
Vithas Hospital San José
Vitoria-gasteiz
United Kingdom
Birmingham Children's Hospital
Birmingham
University Hospitals Bristol and Weston NHS Foundation Trust
Bristol
Queen Elizabeth University Hospital
Glasgow
Evelina London Children's Hospital
London
Manchester University Children's Hospital
Manchester
Sheffield Children's Hospital
Sheffield
Time Frame
Start Date: 2020-03-10
Completion Date: 2024-10-21
Participants
Target number of participants: 84
Treatments
Experimental: Infigratinib 0.016 mg/kg
Dose Escalation:~Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Experimental: Infigratinib 0.032 mg/kg
Dose Escalation and PK substudy:~Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Experimental: Infigratinib 0.064 mg/kg
Dose Escalation and PK substudy:~Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Experimental: Infigratinib 0.128 mg/kg
Dose Escalation and PK substudy:~Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Experimental: Infigratinib 0.25 mg/kg
Dose Escalation and PK substudy:~Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.~Dose Expansion:~Upon identification of the recommended dose from all cohorts analyzed, an expansion cohort of 20 subjects may begin enrollment to further determine safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of the selected dose.
Sponsors
Leads: QED Therapeutics, a BridgeBio company

This content was sourced from clinicaltrials.gov

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