What is vosoritide studying for short stature in Turner syndrome?

Vosoritide is being studied to see if it can increase growth rate in girls with Turner syndrome who have short stature. The research focuses on pre-pubertal girls who have not responded well to growth hormone or have never used it. The study is led by Roopa Kanakatti Shankar, MBBS, MS.

Who may be eligible for this Turner syndrome clinical trial?

Girls aged 3 to under 11 years with a confirmed diagnosis of Turner syndrome and short stature may be eligible. They must be pre-pubertal and either have never used growth hormone or had a poor response to it. Participation requires willingness to comply with study procedures. Please review eligibility criteria for more details.

Is there any cost to participate in this Turner syndrome clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly.

Actively Recruiting

Phase 2

Age: 3Years - 11Years

FEMALE

ID05849389

Vosoritide for Treatment of Short Stature in Girls With Turner Syndrome

Led by Roopa Kanakatti Shankar, MBBS, MS · Updated on 2024-06-24

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are investigating the use of vosoritide, a C-type natriuretic peptide analog, to treat short stature in pre-pubertal girls with Turner syndrome. Turner syndrome is a condition where part or all of the second sex chromosome is missing in females, causing short height due to a specific gene deficiency. This study focuses on girls who have never used growth hormone or have not responded well to it, aiming to see if vosoritide can increase their growth rate over 12 months. Participants will receive daily subcutaneous injections of vosoritide for one year, using dosing based on body weight following FDA-approved guidelines. The study includes an initial 12-month treatment phase where growth velocity and safety are monitored. If participants respond positively, they may continue with vosoritide in an extension phase until their growth stops. During the study, girls will be regularly assessed for safety and changes in height, including growth velocity and standardized height scores. Researchers will also measure other growth-related factors such as seated height ratio, arm span compared to standing height, and bone age. The study will compare growth over the treatment period to historical data from before starting vosoritide, with ongoing monitoring throughout participation.

CONDITIONS

Brief Title

Vosoritide for Short Stature in Turner Syndrome

Who Can Participate

Age: 3Years - 11Years

FEMALE

Eligibility Criteria

You may qualify if you...

Parent or guardian provides signed informed consent and child provides assent if required
Willingness to follow all study procedures and be available for study duration
Female children older than 3 years and younger than 11 years
Pre-pubertal with Tanner Stage 1 breast development
Height less than -2 standard deviation score based on CDC growth charts
Confirmed Turner syndrome diagnosis by chromosome testing with specified mosaicism levels
Either never treated with growth hormone or had a poor response to growth hormone therapy

You will not qualify if you...

Growth plate fusion indicated by bone age of 13 years
Current treatment with growth hormone or IGF-1 (must stop at least one week before screening)
Previous or current use of estrogen, GnRH analogs, aromatase inhibitors, or oxandrolone
History of any malignancy
Presence of Y-chromosome material unless gonadectomy performed and external female genitalia present
Chronic medical conditions affecting growth such as cystic fibrosis, diabetes, inflammatory bowel disease, untreated celiac disease unless well-controlled, severe asthma, or daily oral glucocorticoids use
Malnutrition defined as BMI below 5th percentile
Clinically significant abnormalities on screening tests unless resolved
Known or suspected allergy to the study drug or related products
Receipt of any investigational drug within 90 days prior to the trial start

Research Team

Roopa Kanakatti Shankar, MBBS, MS

Kimberly Boucher, RN

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