Clinical Trials in the UK / NCT07073014
Enrolling by invitation
Phase 3
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
NCT07073014 · tracked via the Priya Life Science UK tracker
Condition(s) studied
Hypochondroplasia
Investigational drug(s) / intervention(s)
Vosoritide: Open-label administration of vosoritide using weight-band dosing
Study summary
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
Eligibility
Inclusion Criteria:
* Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays
* Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure
* Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study
* If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study
* Participants are willing and able to perform all study procedures as physically possible
* Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training
Exclusion Criteria:
* Permanently discontinued study treatment in the studies 111-303 or 111-212
* Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care
* Taking or planning to take any prohibited medications
* Planned or expected to have limb-lengthening surgery during the study period
* Planned or expected bone-related surgery during the study period
* Require any investigational agent prior to completion of study period
* Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy
* Have known hypersensitivity to vosoritide or its excipients
* Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study
* Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason
Primary outcome measure(s)
- Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH) — Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
As measured by change from baseline in height Z-score
Trial sites (30)
| Facility | City | Region | Status |
|---|---|---|---|
| Phoenix Children's Hospital - Thomas Campus | Phoenix | Arizona | |
| Benioff Children's Hospital - Oakland | Oakland | California | |
| Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children) | Wilmington | Delaware | |
| Children's National Medical Center | Washington D.C. | District of Columbia | |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | |
| The Johns Hopkins University School of Medicine | Baltimore | Maryland | |
| University of Minnesota Masonic Children's Hospital | Minneapolis | Minnesota | |
| University of Missouri | Columbia | Missouri | |
| Cook Children's Endocrinology | Fort Worth | Texas | |
| Medical College of Wisconsin | Milwaukee | Wisconsin | |
| Murdoch Children's Research Institute of The Royal Children's Hospital | Parkville | Victoria | |
| University of Alberta Stollery Children's Hospital | Edmonton | Alberta | |
| SickKids - The Hospital for Sick Children | Toronto | Ontario | |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | |
| Hospices Civils de Lyon - Hopital Femme Mere Enfant | Lyon | France | |
| Hopital de la Timone | Marseille | France | |
| Hopital Necker-Enfants Malade | Paris | France | |
| CHU de Toulouse | Toulouse | France | |
| Universitätsklinikum des Saarlandes | Homburg | Saarland | |
| Uniklinik Koln | Cologne | Germany | |
| Univeristatskinderklinik Magdeburg | Magdeburg | Germany | |
| Instituto Giannina Gaslini | Genoa | Italy | |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS | Rome | Italy | |
| Ospedale Pediatrico Bambino Gesu | Rome | Italy | |
| Osaka University Hosptial | Osaka | Japan | |
| Tokushima University Hospital | Tokushima | Japan | |
| Institute of Science Tokyo Hospital | Tokyo | Japan | |
| Tottori University Hospital | Tottori | Japan | |
| Hospital MiKS - Unidad de Cirugía Artroscópica | Vitoria-Gasteiz | Spain | |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | United Kingdom |
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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 NCT07073014 on ClinicalTrials.gov ↗ ← All trials in the UK