What side effects are associated with this type of intervention?

Common treatments for Turner Syndrome, such as growth hormone (GH) therapy, can cause side effects including joint pain, insulin resistance, and increased intracranial pressure. Vosoritide, a C-type natriuretic peptide (CNP) analog, is being studied for its ability to increase growth velocity by targeting chondrocytes. Potential side effects of Vosoritide may include injection site reactions, hypotension, and possible effects on bone metabolism.

What prior FDA approvals exist?

The primary FDA-approved treatment for Turner Syndrome has been growth hormone (GH) therapy, which aims to increase growth velocity in affected individuals. Vosoritide, a C-type natriuretic peptide analog currently under study, represents a novel approach by targeting chondrocytes to enhance cell proliferation and hypertrophy. There are no other C-type natriuretic peptide analogs approved for Turner Syndrome at this time.

What other types of research exist?

Currently, there are no specific novel alternatives to Vosoritide mentioned in the provided context for Turner Syndrome treatment. However, ongoing research in growth hormone therapy optimization, gene therapy targeting the SHOX gene, and other growth plate-stimulating agents may offer future alternatives. Patients should consult with their healthcare provider to explore the latest treatment options available in 2024.

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C-type natriuretic peptide analog

Vosoritide for Turner Syndrome

Phase 2

Recruiting

Research Sponsored by Roopa Kanakatti Shankar, MBBS, MS

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 12 months

Awards & highlights

No Placebo-Only Group

Summary

This trial tests vosoritide, a medication that helps bone growth, in young girls with Turner syndrome who are short. It aims to see if vosoritide can help them grow taller, especially if they haven't responded well to other treatments.

Who is the study for?

This trial is for pre-pubertal girls under 11 years old with Turner Syndrome, either not treated with growth hormone or who had a poor response to it. They must be willing to follow study procedures and have a height significantly below average for their age. Girls with fused growth plates, Y-chromosome material without gonadectomy, significant unrelated health issues, allergies to the medication, or recent investigational drug use are excluded.

What is being tested?

The trial tests Vosoritide's effectiveness in increasing growth velocity over 12 months in girls with Turner Syndrome who have short stature. It compares their growth rate on Vosoritide against historical data from before treatment. Responders can continue receiving the drug after the initial study period.

What are the potential side effects?

While specific side effects of Vosoritide aren't listed here, similar treatments may cause reactions at injection sites, headaches, nausea, and potential long-term effects on bone development. Safety monitoring during the trial will track any adverse events.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 12 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~12 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 12 months for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from baseline in age-sex standardized height standard deviation score

Change from baseline in annualized growth velocity

Incidence of treatment-emergent adverse events

Secondary study objectives

Change in Bone Age

Changes in arm span minus standing height

Changes in seated height ratio

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Vosoritide treatment armExperimental Treatment1 Intervention

Vosoritide will be administered daily via subcutaneous injection for 12 months using the FDA approved weight-based dosing band strategy for achondroplasia.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Turner Syndrome (TS) is characterized by short stature due to haploinsufficiency of the SHOX gene. The most common treatment for TS is growth hormone (GH) therapy, which is approved despite TS not being associated with GH deficiency. GH therapy works by stimulating growth and increasing height, although the benefits are modest. Vosoritide, a C-type natriuretic peptide (CNP) analog, is being studied as it targets chondrocytes within the growth plate, leading to increased cell proliferation and hypertrophy. This mechanism is significant for TS patients as it directly addresses the underlying growth plate dysfunction, potentially offering a more effective treatment for increasing growth velocity compared to GH therapy alone.

Three new allelic mouse mutations that cause skeletal overgrowth involve the natriuretic peptide receptor C gene (Npr3).