INFIGRATINIB for Hypochondroplasia

Inclusion criteria

• {"criterion_text":"- Inclusion Criteria for Participants Rolling Over from ACCEL 2/3: 1.\tPediatric participants with HCH who have completed ACCEL 2/3.\n- Inclusion Criteria for Participants Rolling Over from ACCEL 2/3: 2.\tNegative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.\n- Inclusion Criteria for Participants Rolling Over from ACCEL: 1.\tParticipant must be 3 to <18 years of age at screening and have growth potential (defined as X in females and X in males).\n- Inclusion Criteria for Participants Rolling Over from ACCEL: 2.\tDiagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.\n- Inclusion Criteria for Participants Rolling Over from ACCEL: 3.\tParticipants have at least a 6-month period of growth assessment in the ACCEL study before study entry.\n- Inclusion Criteria for Participants Rolling Over from ACCEL: 4.\tParticipants are ambulatory and able to stand without assistance.\n- Inclusion Criteria for Participants Rolling Over from ACCEL: 5.\tNegative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche."}

Exclusion criteria

• {"criterion_text":"- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 1.\tParticipant has concurrent medical condition (ie, circumstance, syndrome, symptom, sign, etc) that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.\n- Exclusion Criteria for Participants Rolling Over from ACCEL: 4.\tCurrently receiving treatment with agents that are known strong X or prolonged treatment (>1 week) with medications that alter the pH of the gastrointestinal tract.\n- Exclusion Criteria for Participants Rolling Over from ACCEL: 5.\tParticipants receiving medications which could increase X\n- Exclusion Criteria for Participants Rolling Over from ACCEL: 6.\tRegular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid therapy or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit.\n- Exclusion Criteria for Participants Rolling Over from ACCEL: 7.\tHaving had a fracture of the long bones or spine within 12 months of screening.\n- Exclusion Criteria for Participants Rolling Over from ACCEL: 8.\tCurrent participation in an ongoing clinical study with a sponsor other than QED.\n- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 2.\tCurrent evidence of clinically significant X\n- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 3.\tParticipants who developed a medical condition that requires the initiation of treatment with a prohibited medication.\n- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 4.\tParticipants who prematurely discontinued ACCEL 2/3.\n- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 5.\tParticipants who have reached final height or near final height (AHV X for a minimum 6-month observation period and X for males and X for females).\n- Exclusion Criteria for Participants Rolling Over from ACCEL 2/3: 6.\tCurrent participation in an ongoing clinical study with a sponsor other than QED.\n- Exclusion Criteria for Participants Rolling Over from ACCEL: 1.\tParticipants who have a clinically significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the participant or would interfere with study participation or safety evaluations. Complete list referenced in protocol.\n- Exclusion Criteria for Participants Rolling Over from ACCEL: 2.\tParticipants who have a history and/or current evidence of extensive ectopic tissue calcification.\n- Exclusion Criteria for Participants Rolling Over from ACCEL: 3.\tParticipants who have a history of malignancy."}

Primary endpoints

• {"endpoint_text":"- Incidence, type, severity, and causality of AEs; SAEs; AEs that require dose reduction or discontinuation per study protocol; and changes over time in vital signs, laboratory assessments and special investigations (ophthalmic and dental evaluations), and imaging (x-rays, , dual x-ray absorptiometry [DXA] scan).","definition_or_measurement_approach":"Safety assessed by recording incidence, type, severity and causality of AEs and SAEs, AEs requiring dose reduction/discontinuation; serial monitoring of vital signs, laboratory assessments, ophthalmic and dental evaluations, and imaging including x-rays and DXA scans."}
• {"endpoint_text":"- Changes over time in standing height Z-score (in relation to both HCH and average height tables for age and sex).","definition_or_measurement_approach":"Efficacy assessed by serial measurement of standing height and calculation of height Z-score over time relative to HCH-specific and average population growth tables for age and sex."}

Secondary endpoints

• {"endpoint_text":"- Changes over time in AHV and AHV Z-score in relation to average height tables for age and in body proportions including upper to lower body segment ratio, upper arm to forearm length ratio, upper leg to lower leg length ratio, arm span to standing height ratio, and head circumference to standing height ratio, in weight Z-score and BMI in relation to average height tables (and HCH tables, if and when they become available), age of puberty onset and time to X, in body composition","definition_or_measurement_approach":"Anthropometric and growth velocity assessments (AHV, AHV Z-score), body proportions and composition measured longitudinally; weight Z-score and BMI relative to reference tables; pubertal timing recorded."}
• {"endpoint_text":"- Change over time in various quality of life scales including Pediatric Quality of Life, Quality of Life in Short Stature Youth, Patient/ Parent Global Impression of Severity and Change","definition_or_measurement_approach":"Serial patient- and parent-reported outcome measures using specified QoL instruments (PedsQL, QoLISSY, PGIC/PPGIC) over time."}
• {"endpoint_text":"- Treatment benefit as reported by parent and participant during qualitative interview.","definition_or_measurement_approach":"Qualitative interviews of participants and caregivers to capture perceived treatment benefit; analysis of interview data."}
• {"endpoint_text":"- Changes over time in body composition as assessed by dual x-ray absorptiometry (DXA) scans.","definition_or_measurement_approach":"DXA scans performed longitudinally to quantify body composition changes."}
• {"endpoint_text":"- Changes over time in bone morphology/density by x-ray and DXA.","definition_or_measurement_approach":"Serial radiographs and DXA to assess bone morphology and density over time."}

Spain

Earliest CTIS Part Ii Submission Date

23-03-2026

Latest Decision Or Authorization Date

15-04-2026

Processing Time Days

23

Number Of Sites

1

Number Of Participants

13

Norway

Earliest CTIS Part Ii Submission Date

25-03-2026

Latest Decision Or Authorization Date

15-04-2026

Processing Time Days

21

Number Of Sites

2

Number Of Participants

9

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

Syneos Health Clinique Inc.

Responsibilities

PK Analysis laboratory

Name

Medpace Reference Laboratories LLC

Responsibilities

Storage of samples

Third parties

• {"country":"United States","full_name":"Icon Clinical Research LLC","duties_or_roles":"Central Imaging","organisation_type":"Pharmaceutical company"}
• {"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":"X testing","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medpace Reference Laboratories LLC","duties_or_roles":"Storage of samples","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Health Outcome Solutions","duties_or_roles":"Interview analysis","organisation_type":"SME"}
• {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Storage of Qualitative interview data","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Fortrea Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient and Travel reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Belgium","full_name":"Medpace Belgium","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"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":"Canada","full_name":"Syneos Health Clinique Inc.","duties_or_roles":"PK Analysis laboratory","organisation_type":"Hospital/Clinic/Other health care facility"}