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Tyrosine Kinase Inhibitor

Infigratinib for Achondroplasia

Q: Is eligibility for this experiment limited to individuals over twenty years of age?

As per the conditions of this trial, minors aged 3 and above can be enrolled, while young adults up to 18 years old are eligible.

Q: How perilous is the Arm 1: Rollover study for participants?

Arm 1: Rollover subjects has been assigned a safety score of 2, as there is evidence that it can be safely used but no data to support its efficacy yet.

Q: What qualifications are necessary for prospective participants of this medical trial?

This clinical trial is enlisting a maximum of 280 patients, who must be diagnosed with achondroplasia and aged between 3 and 18 years old.

Q: What is the breadth of this investigation's deployment?

This trial is occuring across multiple French hospitals, including Hopital Femme Mere Enfant in Lyon, Hopital Necker-Enfants Malades in Paris, and Hopital des Enfants in Toulouse. In addition to these three sites there are a further 12 medical centres participating.

Q: To what conditions are the subjects in Rollover Arm 1 typically prescribed?

Subjects with advanced directives, an abundance of FGFR2 proteins, human bodies and unresectable metastatic cholangiocarcinomas can <a href="https://www.withpower.com/clinical-trials/all">all</a> receive treatment through Arm 1: Rollover.

Q: Are there any opportunities for new participants to join this experiment?

Confirmed. Clinicaltrials.gov has information that this clinical trial is now open for enrollment, which began on December 6th 2021 and was last amended on March 8th 2023. Up to 280 individuals are being accepted at twelve distinct medical sites.

Q: What is the scope of this clinical trial's subject sample?

The trial sponsor, QED Therapeutics, Inc., must recruit a total of 280 individuals who meet the criteria outlined in order to initiate this study. Recruitment will occur from two locations: Hopital Femme Mere Enfant in Lyon Álava and Hopital Necker-Enfants Malades in Paris Victoria.

Q: What other clinical trials have been conducted to determine the utility of Arm 1: Rollover participants?

Currently, there are 8 ongoing clinical trials focused on researching Arm 1: Rollover subjects; one of these investigations is in its third phase. Most studies related to this treatment take place at Stanford University, although 195 sites across the United States have been identified as conducting research for it.

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

Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic testing

Subject must be 3 to <18 years of age at screening and have growth potential

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 10 years

Awards & highlights

Study Summary

This trial studies the long-term safety, tolerability, and efficacy of a drug to treat ACH, and also assesses the quality of life of those affected.

Who is the study for?

This trial is for children with Achondroplasia (ACH) aged 3 to <18 who have growth potential and completed a previous infigratinib study or have been assessed for growth over 6 months. They must be able to swallow pills, comply with the study plan, and use contraception if applicable. Those with recent fractures, other short stature conditions, or using certain drugs can't join.Check my eligibility

What is being tested?

The trial tests the long-term safety and effectiveness of Infigratinib, an FGFR inhibitor, in kids who've taken it before or are new to it after a specific dose has been identified. It's an open-label extension study where all participants receive the drug and their quality of life is monitored.See study design

What are the potential side effects?

While not explicitly listed here, side effects may include issues related to organ function due to FGFR inhibition. As this is a tyrosine kinase inhibitor being tested on children with ACH, close monitoring will occur for any adverse reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My ACH diagnosis is confirmed by clinical assessment and genetic testing.

Select...

I am between 3 and 17 years old and still growing.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 10 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~10 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 10 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Changes over time in height Z-score in relation to ACH and non-ACH growth charts

Incidence of treatment emergent adverse events (TEAE) and serious TEAE

Secondary outcome measures

Age of puberty onset and time to Tanner stage ≥4

Changes in cognitive functions assessed by age-appropriate computerized tests

Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)

+10 more

Side effects data

From 2023 Phase 2 trial • 158 Patients • NCT02159066

23%

Hypoalbuminaemia

23%

Oedema peripheral

23%

Anaemia

15%

Blood creatine increased

15%

Visual field defect

15%

Gamma-glutamyltransferase increased

15%

Urinary tract infection

15%

Nausea

15%

Diarrhoea

15%

Blood creatine phosphokinase increased

15%

Blood creatinine increased

Colitis

Pyrexia

Cancer pain

Femur fracture

Constipation

Rash maculo-papular

Cardiac arrest

Intestinal perforation

Vomiting

Cardiac failure

Fatigue

Alanine aminotransferase increased

Hypertension

Gastrointestinal haemorrhage

Aphasia

Blood alkaline phosphatase increased

Pain in extremity

100%

80%

60%

40%

20%

Study treatment Arm

Part II: Encorafenib + Binimetinib + Capmatinib

Part I: Encorafenib + Binimetinib (Naive)

Part I: Encorafenib + Binimetinib (Non-naive)

Part II: Encorafenib + Binimetinib + Ribociclib

Part II: Encorafenib + Binimetinib + Infigratinib

Part II: Encorafenib + Binimetinib + Buparlisib

Trial Design

2Treatment groups

Experimental Treatment

Group I: Arm 2: Treatment naïve subjectsExperimental Treatment1 Intervention

Children naïve to infigratinib

Group II: Arm 1: Rollover subjectsExperimental Treatment1 Intervention

Children who have completed QED-sponsored interventional study with infigratinib

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

QED Therapeutics, Inc.Lead Sponsor

8 Previous Clinical Trials

702 Total Patients Enrolled

3 Trials studying Achondroplasia

468 Patients Enrolled for Achondroplasia

QED Therapeutics VP, Clinical DevelopmentStudy DirectorQED Therapeutics

2 Previous Clinical Trials

358 Total Patients Enrolled

2 Trials studying Achondroplasia

358 Patients Enrolled for Achondroplasia

QED Therapeutics SVP, Clinical DevelopmentStudy DirectorQED Therapeutics

Media Library

Infigratinib (Tyrosine Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT05145010 — Phase 2

Achondroplasia Research Study Groups: Arm 2: Treatment naïve subjects, Arm 1: Rollover subjects

Achondroplasia Clinical Trial 2023: Infigratinib Highlights & Side Effects. Trial Name: NCT05145010 — Phase 2

Infigratinib (Tyrosine Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05145010 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is eligibility for this experiment limited to individuals over twenty years of age?

"As per the conditions of this trial, minors aged 3 and above can be enrolled, while young adults up to 18 years old are eligible."

Answered by AI

How perilous is the Arm 1: Rollover study for participants?

"Arm 1: Rollover subjects has been assigned a safety score of 2, as there is evidence that it can be safely used but no data to support its efficacy yet."

Answered by AI

What qualifications are necessary for prospective participants of this medical trial?

"This clinical trial is enlisting a maximum of 280 patients, who must be diagnosed with achondroplasia and aged between 3 and 18 years old."

Answered by AI

What is the breadth of this investigation's deployment?

"This trial is occuring across multiple French hospitals, including Hopital Femme Mere Enfant in Lyon, Hopital Necker-Enfants Malades in Paris, and Hopital des Enfants in Toulouse. In addition to these three sites there are a further 12 medical centres participating."

Answered by AI

To what conditions are the subjects in Rollover Arm 1 typically prescribed?

"Subjects with advanced directives, an abundance of FGFR2 proteins, human bodies and unresectable metastatic cholangiocarcinomas can all receive treatment through Arm 1: Rollover."

Answered by AI

Are there any opportunities for new participants to join this experiment?

"Confirmed. Clinicaltrials.gov has information that this clinical trial is now open for enrollment, which began on December 6th 2021 and was last amended on March 8th 2023. Up to 280 individuals are being accepted at twelve distinct medical sites."

Answered by AI

What is the scope of this clinical trial's subject sample?

"The trial sponsor, QED Therapeutics, Inc., must recruit a total of 280 individuals who meet the criteria outlined in order to initiate this study. Recruitment will occur from two locations: Hopital Femme Mere Enfant in Lyon Álava and Hopital Necker-Enfants Malades in Paris Victoria."

Answered by AI

What other clinical trials have been conducted to determine the utility of Arm 1: Rollover participants?

"Currently, there are 8 ongoing clinical trials focused on researching Arm 1: Rollover subjects; one of these investigations is in its third phase. Most studies related to this treatment take place at Stanford University, although 195 sites across the United States have been identified as conducting research for it."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

2021. "Extension Study of Infigratinib in Children With Achondroplasia (ACH)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05145010.

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