100 Participants Needed

Vosoritide for Idiopathic Short Stature

Recruiting at 7 trial locations
TS
Overseen ByTrial Specialist
Age: < 18
Sex: Any
Trial Phase: Phase 2
Sponsor: BioMarin Pharmaceutical
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you have previously used a growth-promoting agent, you may not be eligible to participate.

What data supports the effectiveness of the drug Vosoritide (Voxzogo) for idiopathic short stature?

Vosoritide has been shown to significantly increase growth velocity in children with achondroplasia, a condition that also causes short stature. This suggests it may be effective for other types of short stature, like idiopathic short stature, although direct evidence for this specific use is not provided.12345

Is vosoritide safe for humans?

Vosoritide, also known as Voxzogo, has been approved for use in children with achondroplasia and is generally considered safe, but it can cause a decrease in blood pressure, which is a serious side effect. This risk is outweighed by the benefits for those with achondroplasia, according to regulatory agencies.12467

How is the drug vosoritide unique for treating idiopathic short stature?

Vosoritide is unique because it is a daily injectable drug originally developed to treat achondroplasia, a specific form of dwarfism, by targeting a genetic mutation that affects bone growth. It works by restoring normal bone growth through a mechanism that inhibits the overactive signaling pathways caused by this mutation.12689

What is the purpose of this trial?

The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH), in children with idiopathic short stature (ISS).

Research Team

MD

Medical Director MD

Principal Investigator

BioMarin Pharmaceutical

Eligibility Criteria

This trial is for children with idiopathic short stature, which means they are significantly shorter than their peers without a known cause. They should have a height Z-score ≤ -2.25 and may have had previous growth treatments. Children with certain genetic conditions causing short stature or those in later stages of puberty are not eligible.

Inclusion Criteria

My physical development is at the earliest puberty stage.
My height is significantly below average for my age and sex.
I have a genetic condition causing short stature, such as Turner syndrome or Noonan syndrome.
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Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observational

Baseline growth is assessed during a minimum 6-month observational period

6 months

Randomized Treatment

Participants receive randomized treatment with vosoritide or placebo for a minimum of 6 months

6 months

Open-label Treatment

Participants receive open-label vosoritide until near-final adult height or age threshold is reached

Variable (until near-final adult height or age threshold)

Human Growth Hormone Treatment

Participants in the hGH group receive open-label hGH for a minimum of 4 years

4 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

  • Vosoritide
Trial Overview The study tests the effects of vosoritide injections compared to human growth hormone in children with ISS. Vosoritide's potential to promote growth will be evaluated over multiple doses and against the established treatment of human growth hormone.
Participant Groups
5Treatment groups
Experimental Treatment
Active Control
Placebo Group
Group I: Vosoritide Dose 3 - High DoseExperimental Treatment1 Intervention
Vosoritide Dose 3 daily injection
Group II: Vosoritide Dose 2 - Medium DoseExperimental Treatment1 Intervention
Vosoritide Dose 2 daily injection
Group III: Vosoritide Dose 1 - Low DoseExperimental Treatment1 Intervention
Vosoritide Dose 1 daily injection
Group IV: Human Growth HormoneActive Control1 Intervention
hGH daily injection
Group V: PlaceboPlacebo Group1 Intervention
Placebo daily injection

Vosoritide is already approved in United States for the following indications:

🇺🇸
Approved in United States as Voxzogo for:
  • To increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses
  • to increase linear growth in pediatric patients less than 5 years of age with achondroplasia with open epiphyses

Find a Clinic Near You

Who Is Running the Clinical Trial?

BioMarin Pharmaceutical

Lead Sponsor

Trials
162
Recruited
115,000+
Alexander Hardy profile image

Alexander Hardy

BioMarin Pharmaceutical

Chief Executive Officer since 2023

MBA from INSEAD

Greg Friberg profile image

Greg Friberg

BioMarin Pharmaceutical

Chief Medical Officer

MD from New York Medical College

Findings from Research

Vosoritide is the first approved therapy specifically designed to increase growth velocity in children with achondroplasia, and early experiences from experts indicate a generally positive reception in clinical practice.
Effective management of vosoritide treatment requires multidisciplinary support, patient education on administration, and monitoring for adherence, as these factors significantly influence treatment outcomes and patient satisfaction.
Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice.Semler, O., Cormier-Daire, V., Lausch, E., et al.[2023]
Voxzogo (vosoritide) is the first drug approved by the FDA and EMA specifically for treating achondroplasia, a leading cause of dwarfism, marking a significant advancement in addressing this rare condition.
Despite the risk of decreased blood pressure as a severe adverse event, the benefits of Voxzogo in fulfilling an unmet medical need for patients with achondroplasia led to its accelerated approval in the U.S. as a treatment for a rare pediatric disease.
Vosoritide, a miracle drug, covering unmet need in achondroplasia: A regulatory update.Simran, ., S, KDS., Dushantrao, SC., et al.[2023]
In a study of 39 short pubertal boys treated for one year, letrozole was found to be more effective in increasing levels of luteinizing hormone (LH) and testosterone while lowering estradiol and IGF-1 compared to anastrozole.
Although both treatments resulted in similar growth velocities, anastrozole showed a greater increase in predicted adult height (PAH) after one year, suggesting it may have a more favorable impact on height potential in the long term.
Letrozole vs anastrozole for height augmentation in short pubertal males: first year data.Neely, EK., Kumar, RB., Payne, SL., et al.[2018]

References

Vosoritide Therapy in Children with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice. [2023]
Vosoritide, a miracle drug, covering unmet need in achondroplasia: A regulatory update. [2023]
Letrozole vs anastrozole for height augmentation in short pubertal males: first year data. [2018]
Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia. [2022]
Efficacy of vosoritide in the treatment of achondroplasia. [2022]
Vosoritide: First Approval. [2022]
Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial. [2023]
New Drug Treats Pediatric Achondroplasia. [2023]
Parents' Experience of Administering Vosoritide: A Daily Injectable for Children with Achondroplasia. [2023]
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