A study to evaluate long-term Infigratinib in children with Achondroplasia
- Conditions
- Achondroplasia in ChildrenMedDRA version: 20.0Level: LLTClassification code 10000452Term: AchondroplasiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2021-001855-15-ES
- Lead Sponsor
- QED Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 280
Inclusion Criteria for Rollover Subjects
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. In girls =10 years of age or girls of any age who have experienced menarche, having a negative pregnancy test.
4. 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.
5. The PI, or a person designated by the PI, will obtain written informed consent from each subject’s Legally Authorised Representative and the subject’s assent, when applicable, before any study-specific activity is performed.
Inclusion Criteria for Treatment Naïve Subjects
1. Subject must be 3 to <18 years of age (inclusive) at screening and have growth potential (as defined by HV =1.5 cm/year, Tanner stage =4 of pubertal development, and bone age of =13 years in females and =15 years in males.
2. Subjects and parent(s) or LARs 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. In girls =10 years of age or girls of any age who have experienced menarche, having a negative pregnancy test.
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 Legally Authorised Representative and the subject’s assent, when applicable, before any study-specific activity is performed.
Are the trial subjects under 18? yes
Number of subjects for this age range: 280
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion Criteria for Rollover Subjects
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 that prematurely discontinued a prior QED-sponsored interventional study with infigratinib.
4. Subjects that have reached final height or near final height (as defined by HV <1.5cm/year and Tanner stage =4).
Exclusion Criteria for Treatment Naïve Subjects
Medical Conditions
1. Subjects who have hypochondroplasia or short stature condition other than ACH (eg, 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, including but not limited to cardiac or vascular disease; hyperthyroidism; abnormal thyroid levels or recently diagnosed hypothyroidism that has not been stable on therapy for at least 3 months; uncontrolled diabetes mellitus (hemoglobin A1c [HbA1c] >9%); adrenal insufficiency; autoimmune inflammatory disease; inflammatory bowel disease; diagnosis of severe sleep apnea that will require surgery or the initiation of continuous positive airway pressure (CPAP) machine use. Subjects with previously diagnosed obstructive sleep apnea (OSA) who are already using a CPAP machine at Screening and whose OSA is stable are eligible provided they continue to be compliant with CPAP use.
3. Subjects who have a history and/or current evidence of extensive ectopic tissue calcification.
4. Subjects who have a history of malignancy.
5. Subjects who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4 or medications that alter the pH of the gastrointestinal tract, including antacids, H2 antagonists (eg, ranitidine), and proton-pump inhibitors (eg, omeprazole); or enzyme-inducing anti-epileptic drugs, including carbamazepine, phenytoin, phenobarbital, and primidone.
6. Subjects who have current evidence of endocrine alterations of calcium/phosphorus homeostasis (Vitamin D supplementation or Vitamin D treatment for Vitamin D insufficiency or deficiency with normal calcium and phosphorus levels is allowed).
7. Subjects who have received regular long-term treatment (=3 weeks) with supraphysiologic doses of glucocorticoid therapy (ie, >15 mg/m2/day of hydrocortisone or equivalence) or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit (low-dose ongoing inhaled steroid for asthma is acceptable).
8. 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. Subjects that received the last dose of any of these growth-promoting agents =6 months before screening are eligible.
9. Subjects who have significant abnormality in screening laboratory results, including but not limited to the following:
a. Hemoglobin <10.0 g/dL.
b. Total bilirubin >1.5× upper limit of normal (ULN).
c. Aspartate aminotransferase/serum glutamic-oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic-pyruvic transaminase (A
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method