NCT07393373

Brief Summary

Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
135

participants targeted

Target at P75+ for phase_2

Timeline
122mo left

Started Apr 2026

Longer than P75 for phase_2

Geographic Reach
10 countries

25 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 19, 2026

Completed
18 days until next milestone

First Posted

Study publicly available on registry

February 6, 2026

Completed
2 months until next milestone

Study Start

First participant enrolled

April 21, 2026

Completed
10.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 31, 2036

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 31, 2036

Last Updated

February 6, 2026

Status Verified

January 1, 2026

Enrollment Period

10.1 years

First QC Date

January 19, 2026

Last Update Submit

January 30, 2026

Conditions

Keywords

skeletal dysplasiaendochondral ossificationhypochondroplasiaHCHshortened proximal limbsfibroblast growth factor receptor 3FGFR3endochondral bone formationdisproportionate short staturequality of lifedwarfismbone diseasesmusculoskeletal diseasesosteochondrodysplasiafunctional abilitiesannualized growth velocityannualized height velocitygrowthgenetic diseasescongenitalAHVAGVPathogenic variants

Outcome Measures

Primary Outcomes (2)

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

    10 years

  • Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts

    10 years

Secondary Outcomes (17)

  • Changes over time in AHV Z-score

    10 years

  • Changes over time in body proportions

    10 years

  • Changes over time in weight Z-score

    10 years

  • Changes overtime in BMI

    10 years

  • Age of puberty onset and time to Tanner stage ≥4

    10 years

  • +12 more secondary outcomes

Study Arms (1)

Arm 1: Rollover subjects

EXPERIMENTAL

Children who have completed QED-sponsored interventional study with infigratinib

Drug: Infigratinib

Interventions

Infigratinib to be administered by mouth and initiated at the last dose level received in the ACCEL 2/3 study or at the dose selected to be further evaluated after proof-of-concept is established for Phase 2 portion of ACCEL 2/3.

Arm 1: Rollover subjects

Eligibility Criteria

Age3 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Pediatric participants with HCH who have completed ACCEL 2/3
  • Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche

You may not qualify if:

  • Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations
  • Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication
  • Participants who prematurely discontinued ACCEL 2/3
  • Participants who have reached final height or near final height
  • Current participation in an ongoing clinical study with a sponsor other than QED

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (25)

UCSF Benioff Children's Hospital

Oakland, California, 94609, United States

Location

Childrens Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

Johns Hopkins School of Medicine

Baltimore, Maryland, 21287, United States

Location

University of Missouri

Columbia, Missouri, 65201, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic

Madison, Wisconsin, 53705, United States

Location

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

Location

London Health Sciences Centre - Children's Hospital of Western Ontario

London, Ontario, N6C 2R5, Canada

Location

Children's Hospital of Eastern Ontario Research Institute

Ottawa, Ontario, K1H 8L1, Canada

Location

Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3T 1C5, Canada

Location

Hôpital Femme Mère Enfant

Bron, 69677, France

Location

Hôpital Universitaire Necker-Enfants Malades

Paris, 75015, France

Location

Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants

Toulouse, 31059, France

Location

Haukeland University Hospital

Bergen, 5021, Norway

Location

Paediatric Clinical Research Unit at Osla University Hospital

Oslo, 0372, Norway

Location

Hospital Pediátrico de Coimbra

Coimbra, 3000-602, Portugal

Location

KK Women's and Children's Hospital

Singapore, 229899, Singapore

Location

UCA Hospital MiKS

Vitoria-Gasteiz, 01010, Spain

Location

Astrid Lindgren Children's Hospital

Solna, 17164, Sweden

Location

The Portland Hospital for Women and Children

London, England, W1W 5AH, United Kingdom

Location

Manchester University

Manchester, England, M13 9WL, United Kingdom

Location

Sheffield Children's Hospital

Sheffield, England, S10 2TH, United Kingdom

Location

Glasgow Clinical Research Facility, Queen Elizabeth University Hospital

Glasgow, Scotland, G51 4TF, United Kingdom

Location

MeSH Terms

Conditions

HypochondroplasiaMucopolysaccharidosis IVDwarfismBone DiseasesMusculoskeletal DiseasesOsteochondrodysplasiasGenetic Diseases, Inborn

Interventions

infigratinib

Condition Hierarchy (Ancestors)

MucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesBone Diseases, DevelopmentalEndocrine System Diseases

Study Officials

  • QED Therapeutics SVP, Clinical Development

    QED Therapeutics, a BridgeBio company

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 19, 2026

First Posted

February 6, 2026

Study Start

April 21, 2026

Primary Completion (Estimated)

May 31, 2036

Study Completion (Estimated)

May 31, 2036

Last Updated

February 6, 2026

Record last verified: 2026-01

Data Sharing

IPD Sharing
Will not share

Locations