INFIGRATINIB for Achondroplasia

Inclusion criteria

• {"criterion_text":"- Inclusion Criteria for Rollover Subjects 1. Pediatric subjects with ACH who have completed a previous QEDsponsored interventional study with infigratinib."}
• {"criterion_text":"- 2. Subjects and parent(s), legal guardians, or caregivers are willing and able to comply with study visits and study procedures."}
• {"criterion_text":"- 3. Subjects are able to swallow oral medication."}
• {"criterion_text":"- 4. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche."}
• {"criterion_text":"- 5. 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."}
• {"criterion_text":"- 6. The PI, or a person designated by the PI, will obtain written informed consent from each subject's parents(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed."}
• {"criterion_text":"- Inclusion Criteria for Treatment Naïve Subjects 1. Subject must be 3 to <18 years of age at screening and have growth potential"}
• {"criterion_text":"- 2. Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures."}
• {"criterion_text":"- 3. Subjects are able to swallow oral medication."}
• {"criterion_text":"- 4. Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic testing."}
• {"criterion_text":"- 5. Subjects have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry."}
• {"criterion_text":"- 6. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- 8. The PI, or a person designated by the PI, will obtain written informed consent from each subject's parent(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed."}

Exclusion criteria

• {"criterion_text":"- 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."}
• {"criterion_text":"- 2. Subjects who developed a medical condition that will require the initiation of treatment with a prohibited medication."}
• {"criterion_text":"- 3. Subjects that prematurely discontinued a prior QED-sponsored interventional study with infigratinib."}
• {"criterion_text":"- 4. Current participation in an ongoing clinical study with a sponsor other than QED."}
• {"criterion_text":"- 5. Subjects that have reached final height or near final height"}
• {"criterion_text":"- Exclusion Criteria for Treatment Naïve Subjects 1. Subjects who have hypochondroplasia or short stature condition other than ACH (eg, trisomy 21, pseudoachondroplasia, psychosocial short stature)."}
• {"criterion_text":"- 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"}
• {"criterion_text":"- 3. Subjects who have a history of malignancy."}
• {"criterion_text":"- 4. Subjects who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4"}
• {"criterion_text":"- 5. Subjects who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible."}
• {"criterion_text":"- 6. Subjects who have received treatment with growth hormone, insulinlike growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months."}
• {"criterion_text":"- 7. Subjects who have significant abnormality in screening laboratory results."}
• {"criterion_text":"- 8. Subjects who have had a fracture within 12 months of screening."}

Primary endpoints

• {"endpoint_text":"- Incidence of treatment emergent adverse events (TEAE) and serious TEAE","definition_or_measurement_approach":"Incidence and recording of TEAEs and serious TEAEs collected throughout treatment and through the Safety Follow-Up Visit (Safety Follow-Up Visit occurs 3 months after the final dose)."}
• {"endpoint_text":"- Changes over time in height Z-score in relation to ACH and non-ACH growth charts.","definition_or_measurement_approach":"Assessed as changes over time in standing height Z-score relative to ACH and non-ACH growth charts (standing height Z-score measurements collected longitudinally)."}

Secondary endpoints

• {"endpoint_text":"- Change over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non-ACH growth charts.","definition_or_measurement_approach":"Height velocity measured over time and expressed as HV Z-score relative to ACH and non-ACH growth charts."}
• {"endpoint_text":"- Changes over time in body proportions","definition_or_measurement_approach":"Anthropometric measurements over time to evaluate body proportion changes."}
• {"endpoint_text":"- Changes over time in weight Z-score","definition_or_measurement_approach":"Weight measurements converted to Z-scores and tracked over time."}
• {"endpoint_text":"- Changes over time in BMI","definition_or_measurement_approach":"Body mass index calculated and changes assessed over time."}
• {"endpoint_text":"- Age of puberty onset and time to Tanner stage ≥4.","definition_or_measurement_approach":"Assessment of pubertal onset age and time to achieve Tanner stage ≥4."}
• {"endpoint_text":"- Changes over time in number of episodes of otitis media per year.","definition_or_measurement_approach":"Recording number of otitis media episodes per year over the study period."}
• {"endpoint_text":"- Changes over time in number of episodes and/or severity of sleep apnea.","definition_or_measurement_approach":"Recording number and/or severity of sleep apnea episodes over time."}
• {"endpoint_text":"- Changes over time in range of motion (elbow).","definition_or_measurement_approach":"Range of motion measurements of the elbow taken longitudinally."}
• {"endpoint_text":"- Changes over time in skeletal abnormalities of the lower extremities and spine","definition_or_measurement_approach":"Clinical and imaging assessments of skeletal abnormalities over time."}
• {"endpoint_text":"- Changes in health-related quality of life (HRQoL) as assessed by Pediatric Quality of Life Inventory (PedsQL)","definition_or_measurement_approach":"PedsQL questionnaire administered to assess HRQoL changes."}
• {"endpoint_text":"- Changes in health-related quality of life (HRQoL) as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)","definition_or_measurement_approach":"QoLISSY questionnaire administered to assess HRQoL in short stature youth."}
• {"endpoint_text":"- Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)","definition_or_measurement_approach":"Pain-NRS scores collected to assess overall pain."}
• {"endpoint_text":"- Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)","definition_or_measurement_approach":"WeeFIM instrument used to evaluate functional abilities over time."}
• {"endpoint_text":"- Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)","definition_or_measurement_approach":"PGI-S questionnaires completed by patient/parent to assess severity."}
• {"endpoint_text":"- Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)","definition_or_measurement_approach":"PGI-C questionnaires to assess change in physical functioning."}
• {"endpoint_text":"- Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview","definition_or_measurement_approach":"Qualitative interviews of subjects and caregivers conducted to evaluate perceived treatment benefit."}
• {"endpoint_text":"- Changes in cognitive functions assessed by age-appropriate computerized tests","definition_or_measurement_approach":"Age-appropriate computerized cognitive tests (e.g., Cogstate) administered to assess cognitive function changes."}

Methods

• Rollover recruitment: enrolling pediatric subjects who completed a previous QED-sponsored interventional Phase 2 or Phase 3 study with infigratinib (rollover population).
• Direct recruitment from observational study PROPEL (QBGJ398-001) for subjects naïve to infigratinib (treatment naïve population).

France

Earliest CTIS Part Ii Submission Date

16-05-2024

Latest Decision Or Authorization Date

27-01-2026

Processing Time Days

621

Number Of Sites

3

Number Of Participants

17

Spain

Earliest CTIS Part Ii Submission Date

16-05-2024

Latest Decision Or Authorization Date

23-01-2026

Processing Time Days

617

Number Of Sites

4

Number Of Participants

38

Italy

Earliest CTIS Part Ii Submission Date

04-03-2025

Latest Decision Or Authorization Date

30-01-2026

Processing Time Days

332

Number Of Sites

2

Number Of Participants

5

Norway

Earliest CTIS Part Ii Submission Date

02-05-2025

Latest Decision Or Authorization Date

26-01-2026

Processing Time Days

269

Number Of Sites

2

Number Of Participants

13

Primary sponsor

Full Name

Qed Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

SUSAR reporting ; Site operations, inlcuding site activation, vendor management (including E-data capture through Medidata vendor, ePRO vendors), central imaging

Name

Fortrea Inc.

Responsibilities

SUSAR reporting; medical monitoring (24/7 coverage)

Name

Syneos Health Clinique Inc.

Responsibilities

Pharmacokinetic assessments

Name

Scout Clinical

Responsibilities

Subject concierge, Subject Travel Reimbursement

Third parties

• {"country":"United States","full_name":"Health Outcome Solutions","duties_or_roles":"development and implementation of patient-related outcomes (PRO)","organisation_type":"Health care"}
• {"country":"Belgium","full_name":"MEDPACE LABORATORIES","duties_or_roles":"Clinical chemistry, Clinical haematology, Analytical chemistry, urinalysis","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"SUSAR reporting ; Site operations, inlcuding site activation, vendor management (including E-data capture through Medidata vendor, ePRO vendors), central imaging","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"Electronic Data Capture (EDC)","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Switzerland","full_name":"STA Pharmaceutical Switzerland S.A.","duties_or_roles":"importer","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"Manufacturer, Importer","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Imperial Clinical Research Services International Limited","duties_or_roles":"Printing of site materials","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Cogstate Inc.","duties_or_roles":"Computerized Cognitive assessment","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Mapi Research Trust","duties_or_roles":"Questionnaire licensing","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Fisher Clinical Services GmbH (Rheinfelden)","duties_or_roles":"importer","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Signant Health Management Limited","duties_or_roles":"ePRO","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"collecting the qualitative interviews","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Subject concierge, Subject Travel Reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United Kingdom","full_name":"ATOM International Limited","duties_or_roles":"Anthropometric measurements and Range of Motion training","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"Genetic Testing","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"SUSAR reporting; medical monitoring (24/7 coverage)","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Syneos Health Clinique Inc.","duties_or_roles":"Pharmacokinetic","organisation_type":"Hospital/Clinic/Other health care facility"}