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Vosoritide for Short Stature in Turner Syndrome

NCT05849389 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2024-06-24

No results posted yet for this study

Summary

Turner syndrome (TS) is characterized by a missing whole or part of the second sex chromosome in a phenotypic female, resulting in short stature due to haploinsufficiency of the short-stature homeobox-containing (SHOX) gene. Growth hormone (GH) is an approved therapy for this condition, although not associated with GH deficiency, and benefits are modest. Vosoritide, a C-type natriuretic peptide (CNP) analog, targets chondrocytes within the growth plate leading to increased cell proliferation and hypertrophy. We hypothesize that patients with TS and short stature will respond to vosoritide treatment leading to increased growth velocity. This study will enroll pre-pubertal girls with TS who are either naïve to GH or have had a poor response to GH therapy. All subjects will be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes. Annualized growth velocity (AGV) on vosoritide will be compared to AGV in the 6-18 months prior to initiation of vosoritide based on historical data available in the medical record. Subjects with a positive response to therapy will be given the option to continue in the extension phase of the study during which they will continue to receive vosoritide until growth cessation.

Conditions

  • Turner Syndrome
  • Short Stature

Interventions

DRUG

Vosoritide

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

Sponsors & Collaborators

  • Roopa Kanakatti Shankar, MBBS, MS

    lead OTHER

Principal Investigators

  • Roopa Kanakatti Shankar, MBBS, MS · Children's National Research Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
11 Years
Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-12
Primary Completion
2026-09-30
Completion
2026-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05849389 on ClinicalTrials.gov