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Study on the Effectiveness and Safety of Vosoritide for Children with Hypochondroplasia

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What is this trial about?

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

This clinical trial is focused on studying a condition called Hypochondroplasia, which is a genetic disorder affecting bone growth, leading to shorter stature. The study will evaluate a treatment called Vosoritide, also known by its code name BMN-111. Vosoritide is a medication that is given as an injection under the skin and is designed to help improve growth in children with this condition.

The purpose of the study is to assess how effective and safe Vosoritide is in increasing the growth rate in children with Hypochondroplasia. Participants in the study will be randomly assigned to receive either Vosoritide or a placebo, which is a substance with no active medication. The study will last for about one year, during which the growth of the children will be monitored and compared to their growth before starting the treatment.

Throughout the study, the main focus will be on changes in the children's growth rate and height. The study aims to provide valuable information on whether Vosoritide can help children with Hypochondroplasia grow taller over time. This research is important for understanding potential new treatments for this condition and improving the quality of life for those affected.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, participants are required to have a confirmed genetic diagnosis of hypochondroplasia, which is a condition affecting bone growth.

    Participants must be between 3 and 18 years old and have undergone genetic testing to confirm the presence of a specific genetic variant associated with hypochondroplasia.

  2. Step 2

    Initial assessments

    Participants undergo initial assessments, including standing height measurements over a period of at least six months before randomization.

    A height Z score is calculated to ensure it is less than or equal to -2.0, based on growth charts from the Center for Disease Control and Prevention.

  3. Step 3

    Randomization

    Participants are randomly assigned to receive either the study medication, vosoritide, or a placebo. This process is double-blind, meaning neither the participants nor the researchers know who receives the medication or placebo.

  4. Step 4

    Medication administration

    The study medication, vosoritide, is administered as a subcutaneous injection. This means it is injected under the skin.

    The medication is available in two dosages: 1.2 mg and 0.56 mg, provided as a powder and solvent for solution for injection.

  5. Step 5

    Treatment duration

    The treatment is administered over a period of 52 weeks. During this time, participants continue to receive either the vosoritide or placebo injections.

  6. Step 6

    Monitoring and assessments

    Throughout the study, participants undergo regular monitoring to assess changes in growth velocity and standing height.

    The primary goal is to evaluate the change in annualized growth velocity at the end of 52 weeks.

  7. Step 7

    Study completion

    At the end of the study period, participants' growth measurements are compared to baseline values to determine the efficacy of vosoritide compared to placebo.

    The study is expected to conclude by March 28, 2026.

Who can join the trial?

9 criteria

  • Participants must be at least 3 years old but younger than 18 years old at the time they join the study.
  • Participants need to have a confirmed genetic diagnosis of **Hypochondroplasia (HCH)**, which means they have a specific change in a gene called **FGFR3** that is linked to HCH. This should be confirmed by previous genetic testing.
  • Participants should have had their height measured while standing for at least 6 months before they are randomly assigned to a treatment group in the study.
  • Participants must have a height **Z score** of -2.0 or lower. A Z score is a way to compare a person's height to the average height of people of the same age and sex, using growth charts from the **Center for Disease Control and Prevention (CDC)**.
  • Both males and females can take part in this study.
  • Females who are 10 years old or older, or who have started their menstrual periods, must have a negative pregnancy test before starting the study and agree to have more pregnancy tests during the study.
  • If participants are sexually active, they must agree to use a very effective method of birth control while they are in the study.
  • Participants must be able to understand and sign a form that shows they agree to take part in the study. This form explains what the study involves and any rules they need to follow.
  • Parents or guardians must be willing and able to provide written consent after the study has been explained to them, before any study-related procedures are done. Participants under 18 must also be willing and able to provide written agreement to participate, if required by local rules.

Who cannot join the trial?

10 criteria

  • Patients with a known allergy to the study medication or any of its ingredients cannot participate.
  • Individuals who have participated in another clinical trial within the last 30 days are not eligible.
  • Patients with a history of certain medical conditions that could interfere with the study results are excluded.
  • Pregnant or breastfeeding women are not allowed to join the study.
  • Individuals who are unable to comply with the study procedures or follow-up visits are not eligible.
  • Patients with severe liver or kidney disease cannot participate.
  • Individuals with a history of drug or alcohol abuse within the past year are excluded.
  • Patients who have received certain medications that could affect the study results are not eligible.
  • Individuals with a history of cancer within the past five years, except for certain types of skin cancer, are excluded.
  • Patients with uncontrolled high blood pressure or diabetes are not eligible.
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Investigated drugs

Vosoritide is a medication being studied for its potential to help children with hypochondroplasia grow taller. Hypochondroplasia is a condition that affects bone growth, leading to shorter stature. The trial aims to see if vosoritide can increase the speed at which these children grow each year.

What is already known about the treatment

Vosoritide – Vosoritide is administered through subcutaneous injection and is currently being studied in clinical trials for its effectiveness in treating hypochondroplasia, a condition characterized by short stature. It is in the advanced stages of research, specifically in a Phase 3 trial, which indicates it is being evaluated for safety and efficacy before potential approval for medical use. The main therapeutic indication for vosoritide is to increase growth velocity in children with hypochondroplasia. At the molecular level, vosoritide works by binding to a specific receptor that regulates bone growth, thereby promoting normal bone development. It is classified pharmacologically as a C-type natriuretic peptide analog.

Investigated diseases

Hypochondroplasia – Hypochondroplasia is a genetic disorder characterized by short stature due to abnormal bone growth. It is similar to achondroplasia but generally presents with milder symptoms. Individuals with hypochondroplasia typically have disproportionate short stature, with shorter limbs compared to the trunk. The condition is caused by mutations in the FGFR3 gene, which affects bone development. As children grow, they may experience delayed motor skills and mild to moderate intellectual disability. The progression of the disease involves continued growth challenges, but the severity can vary among individuals.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IIITrial ID2024-513129-22-00Protocol code111-303Estimated enrolment80 patientsSponsorBiomarin Pharmaceutical Inc.

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