Clinical Trials in Sweden / NCT06410976
Recruiting
Observational
Prospective Clinical Assessment Study in Children With Hypochondroplasia
NCT06410976 · tracked via the Priya Life Science Sweden tracker
Condition(s) studied
Hypochondroplasia
Study summary
This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).
Eligibility
Inclusion Criteria:
Signed informed consent.
Aged 2.5 to \<17 years at study entry.
Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
Participants are ambulatory and able to stand without assistance.
Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
Exclusion Criteria:
Have ACH or short stature condition other than HCH.
In females, having had their menarche. Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening.
Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant.
Clinically significant abnormality in any laboratory test result at screening
Current evidence of corneal or retinal disorders.
Have used any other investigational or approved product or medical device for the treatment of HCH or short stature for ≥ 30 days or with the last dose \<6 months before screening.
Have had regular long-term treatment (\>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).
Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening.
Having had a fracture of the long bones or spine within 12 months of screening.
History and/or current evidence of extensive ectopic tissue calcification.
History of malignancy.
Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation, and/or would place the participant at high risk for poor compliance with study activities or for not completing the study.
Current participation in any other ongoing clinical study with another sponsor.
Primary outcome measure(s)
- Annualized height velocity — up to 3 years
Trial sites (25)
| Facility | City | Region | Status |
|---|---|---|---|
| UCSF Benioff Children's Hospital | Oakland | California | Recruiting |
| Childrens Hospital Colorado | Aurora | Colorado | Recruiting |
| Children's National Hospital | Washington D.C. | District of Columbia | Recruiting |
| Johns Hopkins School of Medicine | Baltimore | Maryland | Recruiting |
| University of Missouri | Columbia | Missouri | Recruiting |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | Recruiting |
| Vanderbilt University Medical Center | Nashville | Tennessee | Recruiting |
| University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic | Madison | Wisconsin | Recruiting |
| Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne | Parkville | Victoria | Recruiting |
| London Health Sciences Centre - Children's Hospital of Western Ontario | London | Ontario | Recruiting |
| Children's Hospital of Eastern Ontario Research Institute | Ottawa | Ontario | Recruiting |
| Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | Recruiting |
| Hôpital Femme Mère Enfant HCL | Bron | France | Recruiting |
| Hôpital Necker-Enfants Malades | Paris | France | Recruiting |
| Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants | Toulouse | France | Recruiting |
| Haukeland University Hospital | Bergen | Norway | Recruiting |
| Paediatric Clinical Research Unit at Oslo University Hospital | Oslo | Norway | Recruiting |
| Hospital Pediátrico de Coimbra | Coimbra | Portugal | Recruiting |
| KK Women's and Children's Hospital | Singapore | Singapore | Recruiting |
| Unidad de Cirugía Artroscopica, Hopsital MIKS | Vitoria-Gasteiz | Spain | Recruiting |
| Astrid Lindgren Children's Hospital | Solna | Sweden | Recruiting |
| The Portland Hospital for Women and Children | London | England | Recruiting |
| Manchester University | Manchester | England | Recruiting |
| Sheffield Children's NHS Foundation Trust | Sheffield | England | Recruiting |
| Glasgow Clinical Research Facility, Queen Elizabeth University Hospital | Glasgow | Scotland | Recruiting |
More QED Therapeutics, a BridgeBio company trials in Sweden
An Interventional Study of Infigratinib in Children With Hypochondroplasia
Enrolling by invitation
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Enrolling by invitation
Other trials for the same condition
An Interventional Study of Infigratinib in Children With Hypochondroplasia
Enrolling by invitation
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Enrolling by invitation
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06410976 on ClinicalTrials.gov ↗ ← All trials in Sweden