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NCT05849389 · ClinicalTrials.gov registry record · Phase 2

Vosoritide for Short Stature in Turner Syndrome

A Phase 2 study of Turner Syndrome and Short Stature, sponsored by Roopa Kanakatti Shankar, MBBS, MS.

Recruiting
Registry status
Phase 2
Development phase
20
Enrollment target
1
Study location

NCT05849389 is a Phase 2 study of Turner Syndrome and Short Stature that is actively recruiting participants, run by Roopa Kanakatti Shankar, MBBS, MS. The registered enrollment target is 20 participants, below the 518-participant average among 14 other Turner Syndrome trials with a reported enrollment target (96% lower). The trial reports 1 study location across 1 state.

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The verdict

NCT05849389, a Phase 2 study of Turner Syndrome and Short Stature, is actively recruiting participants, sponsored by Roopa Kanakatti Shankar, MBBS, MS.

RECRUITING
Registry status
Phase 2
Development phase
20 participants
Enrollment target
1
Study location

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.

Interventions

  • DRUG Vosoritide

Study Locations (1)

District of Columbia

  • Children's National Hospital - Washington D.C.

Trial Details

FieldValue
Enrollment Target 20 participants
Start Date 2024-04-12
Est. Completion 2026-09
Phase Phase 2

What the Registry Record Tells You About NCT05849389

The ClinicalTrials.gov registry entry for NCT05849389 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 518-participant average among 14 other Turner Syndrome trials with a reported enrollment target (96% lower). The listed sponsor is Roopa Kanakatti Shankar, MBBS, MS, which has 1 total studies on file at ClinicalTrials.gov.

The record links to 2 conditions, with Turner Syndrome appearing as the primary indexed condition, and to 1 intervention - of which Vosoritide is the first listed.

NCT05849389 reports 1 study location spanning 1 distinct geographic area - top geographies include District of Columbia.

Frequently Asked Questions

What is clinical trial NCT05849389 about?

NCT05849389 is a clinical study titled "Vosoritide for Short Stature in Turner Syndrome". 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...

What is the current status of trial NCT05849389?

This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 20 participants. The study started on 2024-04-12. Estimated completion is 2026-09.

What conditions does trial NCT05849389 study?

This clinical trial studies the following conditions: Turner Syndrome, Short Stature.

What interventions are being tested in trial NCT05849389?

The interventions under investigation include: Vosoritide (DRUG).

Who is sponsoring clinical trial NCT05849389?

This trial is sponsored by Roopa Kanakatti Shankar, MBBS, MS, which has 1 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT05849389 being conducted?

This trial has 1 study location across District of Columbia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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