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Tyrosine Kinase Inhibitor
Infigratinib for Achondroplasia
Phase 2
Recruiting
Research Sponsored by QED Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Ambulatory and able to stand without assistance
Able to swallow oral medication.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 18 months
Awards & highlights
Study Summary
This trial is testing a new drug for children with achondroplasia who have participated in a previous study for at least six months. The new drug is designed to target fibroblast growth factor receptors 1-3. The trial will include dose escalation and expansion, as well as a pharmacokinetic substudy.
Who is the study for?
This trial is for children aged 3-11 with Achondroplasia who were part of the PROPEL study. They must be able to swallow pills, stand unassisted, and not have started puberty. Kids can't join if they've had certain treatments like growth hormones recently or have eye disorders, a history of cancer, or been on long-term steroids.Check my eligibility
What is being tested?
The trial tests different doses of Infigratinib (a drug that targets specific growth receptors) in kids with Achondroplasia to see which dose is safe and works best. It's an open-label study where everyone knows what treatment they're getting, involving both increasing the dose over time and expanding it to more patients.See study design
What are the potential side effects?
Infigratinib may cause side effects such as liver issues, high phosphate levels in blood leading to muscle problems or rash, finger or toe pain/swelling due to increased blood flow there; also possible are gastrointestinal symptoms like diarrhea.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can walk and stand up on my own without help.
Select...
I can take pills by mouth.
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I have been diagnosed with ACH through clinical assessment and genetic testing.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 18 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 18 months
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from baseline in annualized height velocity
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
PK parameters of infigratinib (AUC24- PK substudy only)
+8 moreSecondary outcome measures
Absolute and change from baseline in arm span (cm)
Absolute and change from baseline in head circumference (cm)
Absolute and change from baseline in knee height (cm)
+12 moreTrial Design
5Treatment groups
Experimental Treatment
Group I: Infigratinib 0.25 mg/kgExperimental Treatment1 Intervention
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.
Group II: Infigratinib 0.128 mg/kgExperimental Treatment1 Intervention
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.
Group III: Infigratinib 0.064 mg/kgExperimental Treatment1 Intervention
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.
Group IV: Infigratinib 0.032 mg/kgExperimental Treatment1 Intervention
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.
Group V: Infigratinib 0.016 mg/kgExperimental Treatment1 Intervention
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.
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Who is running the clinical trial?
QED Therapeutics, Inc.Lead Sponsor
8 Previous Clinical Trials
874 Total Patients Enrolled
3 Trials studying Achondroplasia
640 Patients Enrolled for Achondroplasia
QED Therapeutics VP, Clinical DevelopmentStudy DirectorQED Therapeutics
2 Previous Clinical Trials
530 Total Patients Enrolled
2 Trials studying Achondroplasia
530 Patients Enrolled for Achondroplasia
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- You have another serious health condition that could make it difficult to evaluate the effectiveness or safety of infigratinib.I have had a bone break in the last year.I have a condition causing short stature, but it's not achondroplasia.I have had cancer before.I have never been treated with CNP analogs, FGF ligand traps, or FGFR inhibitors.I have started my menstrual periods.I have been on oral steroids for more than 3 weeks, but inhaled steroids for asthma are okay.I am on medication that affects liver enzymes or changes calcium/phosphorus levels.My height is much shorter or taller than average for my age and sex.I can walk and stand up on my own without help.I can take pills by mouth.I have a current eye condition affecting my cornea or retina.I have had surgery to lengthen my limbs or correct their growth.I have been diagnosed with ACH through clinical assessment and genetic testing.I haven't taken growth hormone, IGF-1, or steroids in the last 6 months.I have not used any experimental drugs or devices for ACH or growth issues.
Research Study Groups:
This trial has the following groups:- Group 1: Infigratinib 0.016 mg/kg
- Group 2: Infigratinib 0.064 mg/kg
- Group 3: Infigratinib 0.25 mg/kg
- Group 4: Infigratinib 0.128 mg/kg
- Group 5: Infigratinib 0.032 mg/kg
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Are we recruiting new participants for this research?
"This trial is still open to patients, as reported on clinicaltrials.gov. The trial was first posted on March 10, 2020, and was last edited on April 5, 2022."
Answered by AI
Does this research allow for people below 35 years of age?
"In order to take part in this clinical trial, patients must be aged between 3 and 11 years old. Out of the 25 total clinical trials, 14 are for patients aged under 18 and 11 are for patients aged 65 and over."
Answered by AI
How many individuals are consenting to participate in this research?
"In order to carry out this research 78 patients are needed, these patients must meet the specified inclusion criteria. The patients can come from multiple sites, two examples being Vanderbilt University Medical Center which is located in Nashville, Tennessee and Stollery Children's Hosptial situated in Edmonton, Alberta."
Answered by AI
Has the FDA cleared Infigratinib 0.128 mg/kg for patient use?
"Infigratinib 0.128 mg/kg is in Phase 2 of clinical trials, so there is some evidence of its safety, but its efficacy has yet to be explored. Our team at Power put its safety at a 2."
Answered by AI
What is the efficacy of Infigratinib 0.128 mg/kg in other patient populations?
"Infigratinib 0.128 mg/kg was first studied in the year 2016 at Memorial Sloan Kettering Cancer Center. So far there have been 6 completed trials. There are currently 10 live trials, with many of these studies concentrated in Nashville, Tennessee."
Answered by AI
What are the primary uses of Infigratinib 0.128 mg/kg?
"Infigratinib 0.128 mg/kg is an effective treatment for human patients with unresectable, metastatic cholangiocarcinomas who have the FGFR2 protein."
Answered by AI
What is the target population for this clinical trial?
"Children with achondroplasia, aged 3-11, who have also completed at least 6 months of growth assessment in the PROPEL study (Protocol QBGJ398-001) are eligible to participate. To be accepted, participants must also be able to walk without assistance and stand unaided."
Answered by AI
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