Actively Recruiting
Vosoritide for Short Stature in Turner Syndrome
Led by Roopa Kanakatti Shankar, MBBS, MS · Updated on 2024-06-24
20
Participants Needed
1
Research Sites
124 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Vosoritide for Short Stature in Turner Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Parent(s) or guardian(s) willing and able to provide informed consent; subjects under 18 able to provide assent if required
- Willingness to comply with all study procedures and availability for the study duration
- Age greater than 3 years and less than 11 years
- Pre-pubertal female defined as Tanner Stage 1 breasts
- Height below -2 standard deviation score based on CDC growth charts
- Confirmed diagnosis of Turner Syndrome by karyotype or chromosomal microarray with mosaicism of at least 10% if applicable
- Subjects either naive to growth hormone or with poor response to growth hormone therapy defined by specified growth velocity criteria
You will not qualify if you...
- Growth plate fusion indicated by bone age of 13 years or more
- Current treatment with growth hormone or recombinant IGF-1 without at least 1 week discontinuation before screening
- Prior or current treatment with estrogen, GnRH analogs, aromatase inhibitors, or oxandrolone
- History of any malignancy
- Presence of Y-chromosome material unless gonadectomy performed with fully external female genitalia
- Chronic medical conditions affecting growth including cystic fibrosis, diabetes, inflammatory bowel disease, untreated celiac disease (unless on gluten-free diet >12 months and normal antibody levels), severe asthma requiring high-dose inhaled steroids, daily oral glucocorticoid use
- Malnutrition defined as BMI below 5th percentile
- Clinically significant abnormalities in screening tests
- Known or suspected allergy to study medication or related components
- Receipt of any investigational drug within 90 days prior to the trial
AI-Screening
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Trial Site Locations
Total: 1 location
1
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
Research Team
R
Roopa Kanakatti Shankar, MBBS, MS
CONTACT
K
Kimberly Boucher, RN
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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