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Study of Vosoritide Safety and Effectiveness in Infants and Young Children (0 to 36 months) with Hypochondroplasia

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on children with hypochondroplasia, a genetic condition that affects bone growth and results in short stature. The research evaluates a medication called vosoritide (also known as BMN 111), which is given as an injection under the skin. The medication comes as a powder that needs to be mixed with a liquid before injection.

The purpose of this research is to determine if vosoritide is safe and effective in improving growth in infants and young children with hypochondroplasia who are less than 3 years old. The study will compare the effects of vosoritide against a placebo. During the study, participants will receive daily injections of either vosoritide or placebo for 52 weeks (one year).

Throughout the study, doctors will monitor the children's growth, measure changes in their height, and check their overall health and safety. They will also look at how the medication affects bone development using special DXA scans (a type of low-dose X-ray that measures bone density). The medication will be given through subcutaneous injections, which means the medicine is injected just under the skin.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial assessment

    Your height and weight measurements will be taken. To participate, you must weigh at least 3 kilograms.

    If you are under 12 months old, your height must be below certain level (height Z-score ≤ -1.0). If you are between 12 and 36 months old, your height must be below a different level (height Z-score ≤ -2.0).

  2. Step 2

    Genetic testing confirmation

    A genetic test will confirm if you have hypochondroplasia, a condition affecting bone growth.

    The test looks for specific changes in a gene called FGFR3.

  3. Step 3

    Treatment phase

    You will receive daily injections under the skin (subcutaneous) of either Voxzogo 0.56 mg or a placebo.

    Neither you nor your doctor will know which treatment you are receiving.

    The treatment will continue for 52 weeks (one year).

  4. Step 4

    Regular monitoring

    Your height will be measured regularly to track growth.

    Regular tests will include blood and urine samples.

    Your vital signs will be checked during visits.

    Body measurements will include arm span and body segment ratios.

    Bone density scans will be performed to check bone health.

  5. Step 5

    Safety monitoring

    Any health changes or side effects will be recorded throughout the study.

    Special attention will be paid to ear infections and seizures if they occur.

    The study team will monitor your overall health and safety throughout the trial.

Who can join the trial?

7 criteria

  • Child must be between 0 and 36 months old (up to 3 years) at the time of starting the study
  • Child must have a confirmed genetic test showing HCH (a type of genetic growth disorder) through detailed DNA testing that shows changes in the FGFR3 gene
  • For babies under 12 months: Height must be below a certain level (Z-score of -1.0 or lower) compared to other children of the same age and sex
  • For children between 12-36 months: Height must be even more below average (Z-score of -2.0 or lower) compared to other children of the same age and sex
  • Child must weigh at least 3 kilograms (about 6.6 pounds) when starting the study
  • Parents or legal guardians must be willing to sign permission forms after being fully informed about the study
  • Parents or caregivers must be willing to give daily injections to their child and complete required training on how to do this properly

Who cannot join the trial?

14 criteria

  • Prior participation in any clinical trial involving vosoritide or similar growth-related medications
  • Having any genetic conditions other than hypochondroplasia (a genetic condition affecting bone growth)
  • History of severe allergic reactions to medications
  • Major surgery in the past 3 months
  • Active or chronic infections
  • Significant heart, liver, or kidney problems
  • Taking growth hormone or other medications that affect growth
  • Inability to attend regular study visits
  • Being outside the age range of 0 to 36 months
  • Having conditions that could interfere with growth measurements
  • Current participation in other clinical trials
  • Medical conditions that could affect the safety of the participant
  • Inability of parent/guardian to provide informed consent
  • Known allergies to the study medication or its components
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Investigated drugs

Vosoritide is a medication being studied to help improve growth in young children with hypochondroplasia, a condition that affects bone growth and results in short stature. The medication works by targeting a specific growth pathway in the body to promote better bone growth and development. It is administered to infants and young children under the age of 3 years to evaluate if it can help improve their linear growth (height).

What is already known about the treatment

Vosoritide - A novel peptide medication administered via subcutaneous injection, specifically developed for treating growth disorders, particularly hypochondroplasia in young children. This drug works by targeting and activating the natriuretic peptide pathway, which plays a crucial role in regulating bone growth and development. Vosoritide belongs to the class of C-type natriuretic peptide (CNP) analogs and functions by stimulating endochondral bone formation through the activation of natriuretic peptide receptor-B (NPR-B) in growth plate chondrocytes. Currently undergoing Phase 2 clinical trials to evaluate its safety and efficacy in infants and young children under 36 months of age with hypochondroplasia, showing promise as a potential treatment option for improving linear growth in affected children.

Investigated diseases

Hypochondroplasia - A genetic disorder that affects bone growth and development, resulting in shorter-than-average arms and legs while the torso remains average size. This condition is characterized by disproportionate short stature that becomes more apparent as children grow. It is caused by mutations in the FGFR3 gene, which provides instructions for making a protein involved in bone development. Individuals with hypochondroplasia typically have shortened limbs, increased head size, and limited range of motion in some joints. The condition shows varying degrees of severity among affected individuals, with some having mild features while others experience more pronounced physical characteristics.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2024-512261-14-00Protocol code111-212Estimated enrolment69 patientsSponsorBiomarin Pharmaceutical Inc.

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