Table of Contents
- Clinical trials overview
- Conditions being studied
- Trial designs and phases
- What the trials measure
- Who may take part
- Long-term follow-up studies
- Summary of the main trials
Clinical trials overview
The trial data show that Vosoritide is being studied in children with several growth conditions, which are health conditions linked to short height or slower growth.[1] The studies are looking at both safety and possible benefit for growth, which means researchers want to know whether the treatment can be used over time and whether it may help children grow better.[1][2]
These trials include children with achondroplasia, hypochondroplasia, Turner syndrome, short stature homeobox-containing gene deficiency, Noonan syndrome, and idiopathic short stature.[1][2][3][4]
Conditions being studied
Achondroplasia is studied in two trials, including a long-term study in children and a later comparison study against BMN 333.[1][5]
Hypochondroplasia is studied in three trials: one Phase 3 study in children, one Phase 2 study in infants and young children aged 0 to less than 36 months, and one long-term extension study.[2][3][4]
Two other trials study children with Turner syndrome, short stature homeobox-containing gene deficiency, and Noonan syndrome, or children with idiopathic short stature.[6][7]
Trial designs and phases
The studies use different trial designs, including randomized, double-blind, and placebo-controlled designs.[2][3][6] Randomized means children are put into groups by chance. Double-blind means neither the family nor the study team knows who gets which treatment during the trial. Placebo-controlled means some participants receive a look-alike treatment with no active study drug, so results can be compared fairly.
The trial phases range from Phase 2 to Phase 4.[1][2][4][5][6][7][8] Phase 2 trials in this set look at early safety and growth effects, while Phase 3 trials test the treatment in larger groups and Phase 4 studies look at later comparisons or follow-up.[2][4][5][8]
What the trials measure
The main growth outcome in several studies is annualized growth velocity (AGV), which means how much a child grows in one year.[2][3][6][7][8] Some trials measure change from baseline, which means they compare results with the starting point before treatment began.[2][3][6][7][8]
Other important outcomes include height Z-score, height, and the upper-to-lower body segment ratio.[3][4][5] The height Z-score shows how a child’s height compares with other children of the same age and sex. The upper-to-lower body segment ratio helps researchers understand body proportions, which is important in some growth conditions.
Safety outcomes are also important. Researchers track adverse events, serious adverse events, laboratory tests, vital signs, physical examination findings, electrocardiograms, echocardiograms, imaging, and clinical hip assessment in some studies.[1][3][5] Adverse events are unwanted medical problems that happen during a study, and serious adverse events are more serious problems that may need special care.
Who may take part
The studies are designed for children, not adults, and the age groups vary by trial.[1][3][6][7] One hypochondroplasia study includes infants and young children from birth to under 36 months.[3] Other studies include children with specific growth conditions such as achondroplasia, Turner syndrome, SHOX deficiency, Noonan syndrome, or idiopathic short stature.[1][2][5][6][7]
Some trials compare Vosoritide with placebo, while others compare it with another treatment approach or continue treatment over a longer time.[2][3][4][6][7][8]
Long-term follow-up studies
Several trials are long-term extension studies, which means they follow children after the first part of the study ends.[1][4][5] These studies are important because they look at treatment effects over time, including until final adult height, which is the height a person reaches when growth is finished.[4]
The long-term studies in achondroplasia and hypochondroplasia track both growth and safety, including treatment-emergent adverse events and yearly changes in height measures.[1][4][5]
Summary of the main trials
The completed trials include a Phase 2 study in achondroplasia and a Phase 3 study in hypochondroplasia.[1][2] The authorised trials include Phase 2 and Phase 3 studies in hypochondroplasia, a Phase 3 extension study in achondroplasia, a Phase 2 basket study in Turner syndrome, SHOX deficiency, and Noonan syndrome, a Phase 2 study in idiopathic short stature, and a Phase 4 comparison study in achondroplasia.[3][4][5][6][7][8]
Across the studies, the main research questions are whether Vosoritide can improve growth, how its effects compare with placebo or other treatments, and how safe and tolerable it is during short-term and long-term use.[1][2][3][4][5][6][7][8]
