RECRUITING

Vosoritide for Short Stature in Turner Syndrome

Conditions

Turner Syndrome Short Stature Vosoritide

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Study Overview

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Description

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.

Official Title

Vosoritide for Treatment of Short Stature in Girls With Turner Syndrome

Quick Facts

Study Start:2024-04-12

Study Completion:2026-09

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05849389

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:3 Years to 11 Years

Sexes Eligible for Study:FEMALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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 required) after the nature of the study has been explained and prior to performance of any research-related procedure. 2. Stated willingness to comply with all study procedures and availability for the duration of the study 3. Age \>3 years 0 days AND \<10 years 364 days 4. Pre-pubertal defined as Tanner Stage 1 breasts in females. 5. Patient height \<-2 SDS. All height SDS values are calculated using the CDC growth charts/data tables. 6. Patients must have a confirmed diagnosis of Turner Syndrome based on a karyotype with a minimum of 30 cells or on a chromosomal microarray. Subjects with Turner Syndrome mosaicism (such as a 46,XX/45,X karyotype) must have a minimum of 10% mosaicism of 45,X cell line in order to participate in the study. 7. Subjects must either be naïve to growth hormone or have a poor response to growth hormone therapy defined as either: 1. Subjects 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. 2. Subjects receiving GH for more than a year with AGV in the last 6 months \< 50%ile for US girls for age/sex). Subjects meeting this criterion are no longer showing catch up growth and may benefit from an alternative form of therapy.	1. Growth plate fusion - Defined as a bone age via the Greulich and Pyle method of 13 years. These patients have limited remaining growth potential. 2. 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 therapies, they will be required to discontinue at least 1 week prior to the screening visit. That decision will be deferred to their treating clinical endocrinologists in conjunction with the patient's guardians. We anticipate that only patients who are having a poor response to their therapy will be interested in enrolling in the current study as there is no rationale for a patient who is receiving growth hormone therapy and having a positive response to enroll in the current study. 3. Prior or concomitant treatment with any form of estrogen, gonadotropin-releasing hormone (GnRH) analog, aromatase inhibitor or oxandrolone 4. History of any type of malignancy 5. Subjects known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia 6. Chronic medical condition known to affect growth including but not limited to: 7. Malnutrition - Defined as a BMI \<5th percentile (CDC growth charts) 8. 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 repeat, the subject may proceed into the trial. 9. Known or suspected allergy to trial medication, excipients, or related products 10. The receipt of any investigational drug within 90 days prior to this trial

Inclusion Criteria

Exclusion 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 required) after the nature of the study has been explained and prior to performance of any research-related procedure.
2. Stated willingness to comply with all study procedures and availability for the duration of the study
3. Age \>3 years 0 days AND \<10 years 364 days
4. Pre-pubertal defined as Tanner Stage 1 breasts in females.
5. Patient height \<-2 SDS. All height SDS values are calculated using the CDC growth charts/data tables.
6. Patients must have a confirmed diagnosis of Turner Syndrome based on a karyotype with a minimum of 30 cells or on a chromosomal microarray. Subjects with Turner Syndrome mosaicism (such as a 46,XX/45,X karyotype) must have a minimum of 10% mosaicism of 45,X cell line in order to participate in the study.
7. Subjects must either be naïve to growth hormone or have a poor response to growth hormone therapy defined as either:
1. Subjects 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.
2. Subjects receiving GH for more than a year with AGV in the last 6 months \< 50%ile for US girls for age/sex). Subjects meeting this criterion are no longer showing catch up growth and may benefit from an alternative form of therapy.

1. Growth plate fusion - Defined as a bone age via the Greulich and Pyle method of 13 years. These patients have limited remaining growth potential.
2. 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 therapies, they will be required to discontinue at least 1 week prior to the screening visit. That decision will be deferred to their treating clinical endocrinologists in conjunction with the patient's guardians. We anticipate that only patients who are having a poor response to their therapy will be interested in enrolling in the current study as there is no rationale for a patient who is receiving growth hormone therapy and having a positive response to enroll in the current study.
3. Prior or concomitant treatment with any form of estrogen, gonadotropin-releasing hormone (GnRH) analog, aromatase inhibitor or oxandrolone
4. History of any type of malignancy
5. Subjects known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia
6. Chronic medical condition known to affect growth including but not limited to:
7. Malnutrition - Defined as a BMI \<5th percentile (CDC growth charts)
8. 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 repeat, the subject may proceed into the trial.
9. Known or suspected allergy to trial medication, excipients, or related products
10. The receipt of any investigational drug within 90 days prior to this trial

Contacts and Locations

Study Contact

Roopa Kanakatti Shankar, MBBS, MS

CONTACT

202-476-2121

roopa.shankar@childrensnational.org

Kimberly Boucher, RN

CONTACT

202-476-1403

kboucher@childrensnational.org

Principal Investigator

Roopa Kanakatti Shankar, MBBS, MS

PRINCIPAL_INVESTIGATOR

Children's National Research Institute

Study Locations (Sites)

Children's National Hospital

Washington, District of Columbia, 20010

United States

Collaborators and Investigators

Sponsor: Roopa Kanakatti Shankar, MBBS, MS

Roopa Kanakatti Shankar, MBBS, MS, PRINCIPAL_INVESTIGATOR, Children's National Research Institute

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-04-12

Study Completion Date2026-09

Study Record Updates

Study Start Date2024-04-12

Study Completion Date2026-09

Terms related to this study

Keywords Provided by Researchers

Turner syndrome
Short Stature
Vosoritide

Additional Relevant MeSH Terms

Turner Syndrome
Short Stature