INFIGRATINIB for Hypochondroplasia

Stratification factors

• age
• sex
• pubertal development

Inclusion criteria

• {"criterion_text":"- Participants must have completed the Week 26 visit in the observational study (QBGJ398-004)."}
• {"criterion_text":"- Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche."}
• {"criterion_text":"- If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception while taking study drug and for 30 days after the last dose of study drug."}
• {"criterion_text":"- Signed informed consent."}
• {"criterion_text":"- ACCEL 2: Participants 5-11 years of age (inclusive)."}
• {"criterion_text":"- Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test."}
• {"criterion_text":"- AHV >X cm/year over a period ≥26 weeks prior to screening based on measurements obtained during observational Study QBJG398-004 (ACCEL) (Phase 2 portion only). NOTE: Visit window may be applied to the 26-week period of the observational ACCEL study as per the ACCEL SoA (ie. the Week 26 visit in ACCEL may occur slightly earlier than 26 weeks)."}
• {"criterion_text":"- Participants are able to swallow oral medication."}
• {"criterion_text":"- Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures."}
• {"criterion_text":"- Participants are ambulatory and able to stand without assistance."}

Exclusion criteria

• {"criterion_text":"- Participants who have ACH or a short stature condition other than HCH. Participants with variants in FGFR3 known to cause ACH or other FGFR3-related conditions will be excluded."}
• {"criterion_text":"- Children with epilepsy who meet certain additional criteria."}
• {"criterion_text":"- Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib. Complete list referenced in protocol."}
• {"criterion_text":"- Current evidence of clinically significant corneal or retinal disorder/keratopathy."}
• {"criterion_text":"- Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations."}
• {"criterion_text":"- History and/or current evidence of extensive ectopic tissue calcification."}
• {"criterion_text":"- History of malignancy."}
• {"criterion_text":"- Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature."}
• {"criterion_text":"- Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit."}
• {"criterion_text":"- Current participation in any other ongoing clinical study with other sponsor."}
• {"criterion_text":"- Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study; or previous guided growth surgery with plates still in place or removed within the 12 months prior to screening."}
• {"criterion_text":"- Currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP)3A or prolonged treatment (>1 week) with medications that alter the pH of the gastrointestinal tract."}
• {"criterion_text":"- Participants receiving medications which could increase serum phosphorus and/or calcium concentrations"}
• {"criterion_text":"- Clinically significant abnormality in any laboratory test result at screening as specified in the protocol."}
• {"criterion_text":"- Having had a fracture of the long bones or spine within 12 months prior to screening."}
• {"criterion_text":"- Females who have had their menarche (ACCEL 2 only)."}
• {"criterion_text":"- Pregnant or breastfeeding at the screening visit."}
• {"criterion_text":"- Allergy to any components of the study drug."}

Primary endpoints

• {"endpoint_text":"- ACCEL 2: Change from baseline (BL) in height velocity (HV) at Week 26 (annualized to cm/year). ACCEL 3: Change from BL to Week 52 in AHV compared to placebo.","definition_or_measurement_approach":"Change from baseline in height velocity (annualized to cm/year) at specified timepoints: ACCEL 2 at Week 26 (annualized to cm/year); ACCEL 3 change from baseline to Week 52 in annualized height velocity (AHV) compared to placebo."}

Secondary endpoints

• {"endpoint_text":"- ACCEL 2: Change from BL in height Z-score (in relation to both HCH and average height tables for age and sex) compared to placebo.","definition_or_measurement_approach":"Change from baseline in height Z-score using both HCH-specific and population average height tables for age and sex, compared to placebo."}
• {"endpoint_text":"- ACCEL 3: Change from BL to Week 52 in upper to lower body segment ratio, compared to placebo.","definition_or_measurement_approach":"Change from baseline to Week 52 in upper-to-lower body segment ratio compared between infigratinib and placebo arms."}

Spain

Earliest CTIS Part Ii Submission Date

16-06-2025

Latest Decision Or Authorization Date

01-12-2025

Processing Time Days

168

Number Of Sites

2

Number Of Participants

7

France

Earliest CTIS Part Ii Submission Date

27-06-2025

Latest Decision Or Authorization Date

27-11-2025

Processing Time Days

153

Number Of Sites

1

Number Of Participants

5

Norway

Earliest CTIS Part Ii Submission Date

13-06-2025

Latest Decision Or Authorization Date

27-11-2025

Processing Time Days

167

Number Of Sites

2

Number Of Participants

8

Portugal

Earliest CTIS Part Ii Submission Date

04-04-2025

Latest Decision Or Authorization Date

27-11-2025

Processing Time Days

237

Number Of Sites

1

Number Of Participants

7

Sweden

Earliest CTIS Part Ii Submission Date

13-06-2025

Latest Decision Or Authorization Date

11-03-2026

Processing Time Days

271

Number Of Sites

1

Number Of Participants

5

Primary sponsor

Full Name

Qed Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Clinical Research LLC

Responsibilities

Central Imaging

Name

Premier Research International LLC

Responsibilities

management of trial sites, Investigator recruitment, project management, data management, Q/A auditing

Name

Fortrea Inc.

Responsibilities

Pharmacovigilance

Name

Syneos Health Clinique Inc.

Responsibilities

PK Analysis laboratory

Name

Medpace Reference Laboratories LLC

Responsibilities

Storage of biosamples

Third parties

• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"Pharmacovigilance","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Icon Clinical Research LLC","duties_or_roles":"Central Imaging","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medpace Reference Laboratories LLC","duties_or_roles":"Storage of biosamples","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Belgium","full_name":"Medpace Belgium","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Central ECG","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient and Travel reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Premier Research International LLC","duties_or_roles":"management of trial sites, Investigator recruitment, project management, data management, Q/A auditing","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Canada","full_name":"Syneos Health Clinique Inc.","duties_or_roles":"PK Analysis laboratory","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Health Outcomes Solutions","duties_or_roles":"Interview Analysis","organisation_type":"Industry"}
• {"country":"United States","full_name":"Meso Scale Diagnostics LLC","duties_or_roles":"CXM Analysis","organisation_type":"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":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Cogstate Inc.","duties_or_roles":"Cognitive testing","organisation_type":"Pharmaceutical company"}