A Phase 3 interventional study of Vosoritide in Hypochondroplasia, sponsored by BioMarin Pharmaceutical. Enrolling by invitation at 30 sites in 9 countries. Per ClinicalTrials.gov, last updated 2026-09-09.
Sponsored by BioMarin Pharmaceutical · Phase 3, Interventional, and Treatment
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
BioMarin Pharmaceutical is the lead sponsor of 112 studies on the registry; 13 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Open-label active drug
Drug: Vosoritide
Open-label administration of vosoritide using weight-band dosing
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)
As measured by change from baseline in height Z-score
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate the maintenance effect of vosoritide treatment on standing height
change from baseline in height at Week 52
Time frame: Baseline to week 52
Evaluate the maintenance effect of vosoritide treatment on standing height Z-score
Change from baseline in height Z-score at Week 52
Time frame: Baseline to week 52
Evaluate long-term effect of vosoritide treatment on growth velocity
Absolute 6- or 12-month interval AGV
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on body proportionality
Change from baseline in upper to lower body segment ratio
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone age versus chronological age
As measured by left-hand and wrist x-ray
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone metabolism biomarkers
Change from baseline in serum collagen X marker
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term changes in Health-Related Quality of Life (HRQoL)
Change from baseline as measured by Quality of Life In Short-Statured Youth (QoLISSY) total score
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on bone quality
Change from baseline in bone mineral density (BMD) as measured by Dual X-Ray Absorptiometry
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Evaluate long-term effect of vosoritide treatment on pubertal growth as measured by Tanner Staging
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the incidence of otitis media
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
To monitor the frequency of seizures
Time frame: Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
Plan to share: Undecided
No publications or documents are linked to this record.
Started Jun 2025 (actual). Primary completion is estimated for Oct 2044, 232 months after the start.
How these figures are worked out, and which studies are left out of them.
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Studies of the drug tested here: Vosoritide
Hypochondroplasia