Vosoritide for Short Stature in Turner Syndrome

Recruiting Phase 2 Interventional Study
Turner Syndrome Short Stature
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
3 – 11
Sex
Female
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
20 (estimated)
Sponsor
Roopa Kanakatti Shankar, MBBS, MS · Other
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About This Trial
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 (C…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: 1. 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, subjects under the age of 18 are willing and able to provide assent (if…
Contacts

Roopa Kanakatti Shankar, MBBS, MS

202-476-2121

roopa.shankar@childrensnational.org

CONTACT

Kimberly Boucher, RN

202-476-1403

kboucher@childrensnational.org

CONTACT