Clinical Trial Finder

Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

Study Purpose

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

 No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

 Interventional
Eligible Ages N/A and Over
Gender All
More Inclusion & Exclusion Criteria

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

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

 NCT07073014
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

 Phase 3
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

 BioMarin Pharmaceutical
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

 N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

 Industry
Overall Status Enrolling by invitation
Countries Australia, Canada, France, Germany, Italy, Japan, Spain, United Kingdom, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

 Hypochondroplasia
Arms & Interventions

Arms

Experimental: Vosoritide

Open-label active drug

Interventions

Drug: - Vosoritide

Open-label administration of vosoritide using weight-band dosing

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Wilmington, Delaware

Status

Address

Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)

Wilmington, Delaware, 19803

Children's National Medical Center, Washington, District of Columbia

Status

Address

Children's National Medical Center

Washington, District of Columbia, 20010

Chicago, Illinois

Status

Address

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611

Medical College of Wisconsin, Milwaukee, Wisconsin

Status

Address

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226

International Sites

Murdoch Children's Research Institute, Parkville, Victoria, Australia

Status

Address

Murdoch Children's Research Institute

Parkville, Victoria, 3052

Edmonton, Alberta, Canada

Status

Address

University of Alberta Stollery Children's Hospital

Edmonton, Alberta,

Toronto, Ontario, Canada

Status

Address

SickKids - The Hospital for Sick Children

Toronto, Ontario,

Montréal, Quebec, Canada

Status

Address

Centre Hospitalier Universitaire Sainte-Justine

Montréal, Quebec,

Lyon, France

Status

Address

Hospices Civils de Lyon - Hopital Femme Mere Enfant

Lyon, ,

Hopital de la Timone, Marseille, France

Status

Address

Hopital de la Timone

Marseille, ,

Hopital Necker-Enfants Malade, Paris, France

Status

Address

Hopital Necker-Enfants Malade

Paris, ,

CHU de Toulouse, Toulouse, France

Status

Address

CHU de Toulouse

Toulouse, ,

Uniklinik Koln, Köln, Germany

Status

Address

Uniklinik Koln

Köln, ,

Univeristatskinderklinik Magdeburg, Magdeburg, Germany

Status

Address

Univeristatskinderklinik Magdeburg

Magdeburg, ,

Instituto Giannina Gaslini, Genoa, Italy

Status

Address

Instituto Giannina Gaslini

Genoa, ,

Rome, Italy

Status

Address

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, ,

Ospedale Pediatrico Bambino Gesu, Rome, Italy

Status

Address

Ospedale Pediatrico Bambino Gesu

Rome, ,

Osaka University Hosptial, Osaka, Japan

Status

Address

Osaka University Hosptial

Osaka, ,

Tokushima University Hospital, Tokushima, Japan

Status

Address

Tokushima University Hospital

Tokushima, ,

Institute of Science Tokyo Hospital, Tokyo, Japan

Status

Address

Institute of Science Tokyo Hospital

Tokyo, ,

Tottori University Hospital, Tottori, Japan

Status

Address

Tottori University Hospital

Tottori, ,

Vithas Hospital San Jose, Vitoria-Gasteiz, Spain

Status

Address

Vithas Hospital San Jose

Vitoria-Gasteiz, ,

London, United Kingdom

Status

Address

Great Ormond Street Hospital for Children NHS Foundation Trust

London, ,

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