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New treatment option for Turner Syndrome
Official title Vosoritide for Short Stature in Turner Syndrome
ClinicalTrials.gov ID: NCT05849389
What this study is testing
What is Vosoritide?
Vosoritide is an investigational medicine, given as an once-daily injection under the skin, being studied as a potential treatment for turner syndrome.
Also referred to as Voxzogo.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 11, women only
You may be able to join if
- Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior...
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Age \>3 years 0 days AND \<10 years 364 days
- Pre-pubertal defined as Tanner Stage 1 breasts in females.
- Patient height \<-2 SDS. All height SDS values are calculated using the CDC growth charts/data tables.
You likely can't join if
- Growth plate fusion - Defined as a bone age via the Greulich and Pyle method of 13 years. These patients have limited remaining growth potential.
- Concomitant treatment with growth hormone or recombinant insulin-like growth factor-1 (IGF-1). Patients may have been previously treated with growth...
- Prior or concomitant treatment with any form of estrogen, gonadotropin-releasing hormone (GnRH) analog, aromatase inhibitor or oxandrolone
- History of any type of malignancy
- people known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia
- Chronic medical condition known to affect growth including but not limited to: A. Cystic fibrosis B. Diabetes C. Inflammatory Bowel Disease D...
See the full eligibility criteria
- Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any research-related procedure. Also, people under...
- Stated willingness to comply with all study procedures and availability for the duration of the study
- Age \>3 years 0 days AND \<10 years 364 days
- Pre-pubertal defined as Tanner Stage 1 breasts in females.
- Patient height \<-2 SDS. All height SDS values are calculated using the CDC growth charts/data tables.
- Patients must have a confirmed diagnosis of Turner Syndrome based on a karyotype with a minimum of 30 cells or on a chromosomal microarray. people with Turner Syndrome mosaicism (such as a 46,XX/45,X karyotype) must...
- people must either be naïve to growth hormone or have a poor response to growth hormone therapy defined as either:
- people completed at least one year of treatment with GH and first year height velocity (HV) below -1 SD according to the National Cooperative Growth Study TS 1st year response to growth hormone height velocity curve.
- people receiving GH for more than a year with AGV in the last 6 months \< 50%ile for US girls for age/sex). people meeting this criterion are no longer showing catch up growth and may benefit from an alternative form of...
- Growth plate fusion - Defined as a bone age via the Greulich and Pyle method of 13 years. These patients have limited remaining growth potential.
- Concomitant treatment with growth hormone or recombinant insulin-like growth factor-1 (IGF-1). Patients may have been previously treated with growth hormone or IGF-1 therapy. If the patient is currently on one of these...
- Prior or concomitant treatment with any form of estrogen, gonadotropin-releasing hormone (GnRH) analog, aromatase inhibitor or oxandrolone
- History of any type of malignancy
- people known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia
- Chronic medical condition known to affect growth including but not limited to: A. Cystic fibrosis B. Diabetes C. Inflammatory Bowel Disease D. Untreated Celiac Disease - If a subject has been diagnosed with celiac...
- Malnutrition - Defined as a BMI \<5th percentile (CDC growth charts)
- Any clinically significant abnormality on screening tests as determined by the principal investigator. Abnormal screening labs may be repeated up to 3 months after the screening visit. If those labs are normal on...
- Known or suspected allergy to trial medication, excipients, or related products
- The receipt of any investigational drug within 90 days prior to this trial
The study team makes the final eligibility decision.
Where it's taking place
- Washington D.C., District of Columbia, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling female, 3 years to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Washington D.C., District of Columbia, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.