Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
84 participants
INTERVENTIONAL
2020-03-10
2024-10-21
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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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.
Infigratinib 0.016 mg/kg
Initial cohort dose of infigratinib at the protocol-specified starting dose, with subsequent cohort escalations based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
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.
Infigratinib 0.032 mg/kg
Subsequent cohort dose escalation based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
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.
Infigratinib 0.064 mg/kg
Subsequent cohort dose escalation based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
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.
Infigratinib 0.128 mg/kg
Subsequent cohort dose escalation based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
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.
Infigratinib 0.25 mg/kg
Subsequent cohort dose escalation based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
Interventions
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Infigratinib 0.016 mg/kg
Initial cohort dose of infigratinib at the protocol-specified starting dose, with subsequent cohort escalations based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
Infigratinib 0.032 mg/kg
Subsequent cohort dose escalation based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
Infigratinib 0.064 mg/kg
Subsequent cohort dose escalation based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
Infigratinib 0.128 mg/kg
Subsequent cohort dose escalation based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
Infigratinib 0.25 mg/kg
Subsequent cohort dose escalation based on protocol-specific criteria.
Infigratinib tablets to be administered by mouth.
Eligibility Criteria
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Inclusion Criteria
2. Diagnosis of ACH, documented clinically and confirmed by genetic testing.
3. At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.
4. Ambulatory and able to stand without assistance
5. Able to swallow oral medication.
Exclusion Criteria
2. In females, having had their menarche.
3. Height \< -2 or \> +2 standard deviations for age and sex based on reference tables on growth in children with ACH.
4. Significant concurrent disease or condition that, in the view of the Investigator and/or Sponsor, would confound assessment of efficacy or safety of infigratinib.
5. Current evidence of corneal or retinal disorder/keratopathy.
6. History of malignancy.
7. Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 and medications which increase serum phosphorus and/or calcium concentration.
8. Treatment with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (\>3 months) at any time.
9. Treatment with a C-type natriuretic peptide (CNP) analog, fibroblast growth factor (FGF) ligand trap, or treatment targeting FGFR inhibition at any time.
10. Regular long-term treatment (\>3 weeks) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).
11. Treatment with any other investigational product or investigational medical device for the treatment of ACH or short stature.
12. Previous limb-lengthening surgery or guided growth surgery.
13. Fracture within 12 months of screening.
3 Years
11 Years
ALL
No
Sponsors
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QED Therapeutics, Inc., a Bridgebio company
INDUSTRY
Responsible Party
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Principal Investigators
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QED Therapeutics VP, Clinical Development
Role: STUDY_DIRECTOR
QED Therapeutics
Locations
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UCSF Benioff Children's Hospital
Oakland, California, United States
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, United States
Johns Hopkins School of Medicine
Baltimore, Maryland, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
Murdoch Children's Hospital
Parkville, Victoria, Australia
Stollery Children's Hospital
Edmonton, Alberta, Canada
Hopital Femme Mere Enfant
Lyon, , France
Hopital Necker-Enfants Malades
Paris, , France
Hopital des Enfants
Toulouse, , France
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario Virgen de la Victoria
Málaga, , Spain
Vithas Hospital San José
Vitoria-Gasteiz, Álava, Spain
Sheffield Children's Hospital
Sheffield, England, United Kingdom
Birmingham Children's Hospital
Birmingham, , United Kingdom
University Hospitals Bristol and Weston NHS Foundation Trust
Bristol, , United Kingdom
Queen Elizabeth University Hospital
Glasgow, , United Kingdom
Evelina London Children's Hospital
London, , United Kingdom
Manchester University Children's Hospital
Manchester, , United Kingdom
Countries
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References
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Savarirayan R, De Bergua JM, Arundel P, Salles JP, Saraff V, Delgado B, Leiva-Gea A, McDevitt H, Nicolino M, Rossi M, Salcedo M, Cormier-Daire V, Skae M, Kannu P, Phillips J 3rd, Saal H, Harmatz P, Candler T, Hill D, Muslimova E, Weng R, Bai Y, Raj S, Hoover-Fong J, Irving M, Rogoff D. Oral Infigratinib Therapy in Children with Achondroplasia. N Engl J Med. 2025 Feb 27;392(9):865-874. doi: 10.1056/NEJMoa2411790. Epub 2024 Nov 18.
Savarirayan R, De Bergua JM, Arundel P, McDevitt H, Cormier-Daire V, Saraff V, Skae M, Delgado B, Leiva-Gea A, Santos-Simarro F, Salles JP, Nicolino M, Rossi M, Kannu P, Bober MB, Phillips J 3rd, Saal H, Harmatz P, Burren C, Gotway G, Cho T, Muslimova E, Weng R, Rogoff D, Hoover-Fong J, Irving M. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. Ther Adv Musculoskelet Dis. 2022 Mar 21;14:1759720X221084848. doi: 10.1177/1759720X221084848. eCollection 2022.
Other Identifiers
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QBGJ398-201
Identifier Type: -
Identifier Source: org_study_id
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