Vosoritide for Short Stature in Turner Syndrome

Part of paid clinical trials in Washington D.C., District of Columbia.

Sponsor
Roopa Kanakatti Shankar, MBBS, MS
Study ID
NCT05849389
Phase
PHASE2
Status
Recruiting
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Conditions

  • Short Stature
  • Turner Syndrome

Eligibility Criteria

Sex
FEMALE
Age
3 Years - 11 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Apr 12, 2024
Status verified
Jun 2024
Primary completion
Sep 30, 2026
Completion
Sep 30, 2026

Study Design

Enrollment
20 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

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

Primary Outcome Measure

Incidence of treatment-emergent adverse events [ Time Frame: 12 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's National HospitalWashington D.C.District of Columbia20010
Kimberly Boucher, RN
[email protected]
202-476-1403

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By research site
Children's National Hospital· Washington D.C., DC

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