Clinical trial: Study of Infigratinib in Children With Achondroplasia (NCT04265651)

Eligibility criteria:
Inclusion Criteria:

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

          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:

          1. Hypochondroplasia or short stature condition other than ACH.

          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 6 months of screening.
No condition on gender to be admitted to the trial.
No healthy subjects accepted to join the trial.
Subject must be at least 3 Years old.
Subject must be at most 11 Years

Patient description (medical language): A 3-year-old girl is brought to the clinic by her parents for assessment of her short stature. Physical examination reveals short limbs and a relatively large head. She has a flat nasal bridge and a small midface. The girl's father exhibits similar physical features; however, her mother looks normal. The genetic testing reveals an autosomal dominant point mutation in the fibroblast growth factor receptor 3 (FGFR3) gene consistent with achondroplasia in both father and the child. The girl has not received any treatment yet, and it is her first visit after immigration to the US. The other mental and developmental examinations are unremarkable.

Patient description (patient's own words): I just brought my little 3-year-old girl to the clinic to have her checked up by the doctor. I was worried sick because she was smaller than her classmates. The lovely doctor performed a physical exam and she told me that she has short limbs and a larger head. My girl also has a flat nasal bridge and a small midface. My baby just has the face of her father, unlike me! They performed some genetic testing on my child and my husband and found out that both my husband and her have a mutation of the FGFR3 gene, which led the doctor to diagnose them with dwarfism. My baby never received any treatment and it was our first visit to the doctor since we arrived in the US. The other tests regarding her mental and physical development were perfectly fine.

Does the patient match the trial criteria? 0