Turner SyndromePhase 2NCT05849389
Vosoritide for Short Stature in Turner Syndrome
A Phase 2 study testing Vosoritide in people with Turner Syndrome and Short Stature, run by Roopa Kanakatti Shankar, MBBS, MS at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 3 to 11 years, women only
- Conditions the study is for
- Turner Syndrome, Short Stature
- What is being tested?
- Vosoritide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Apr 2024to Sep 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 11 years
- Women only
- No healthy volunteers
- Child
Who may be able to join
- 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.
- 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. 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.
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- Subjects must either be naïve to growth hormone or have a poor response to growth hormone therapy defined as either:
- 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.
- 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.
Who cannot join
- 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 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.
- Prior or concomitant treatment with any form of estrogen, gonadotropin-releasing hormone (GnRH) analog, aromatase inhibitor or oxandrolone
- History of any type of malignancy
- Subjects 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:
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- Cystic fibrosis B. Diabetes C. Inflammatory Bowel Disease D. Untreated Celiac Disease - If a subject has been diagnosed with celiac disease and has been on a gluten free diet for >12 months and has a tissue transglutaminase antibody within the normal range at screening, then they are eligible for the trial.
- Asthma requiring a daily inhaled steroid dose > 400 micrograms of inhaled budesonide per day or equivalent F. Taking daily oral glucocorticoids for any reason G. Note - Attention Deficit hyperactivity Disorder (ADHD) treated with a stimulant and treated hypothyroidism with a normal thyroid stimulating hormone (TSH) will NOT exclude the subject from participating in the trial. Subjects on either stimulant medication or thyroid hormone replacement must be on a stable dose for 3 months prior to the screening visit.
- Congenital heart disease which places the subject at increased risk of an adverse cardiac outcome in the setting of hypotension including but not limited to: hypertrophic cardiomyopathy, aortic stenosis with peak gradient >50mmHg, severe aortic regurgitation (defined as pressure half time >500ms by echocardiogram), coronary insufficiency, or any anatomy with a need for an afterload reducing agent. Any patient with baseline abnormalities on echocardiogram will be reviewed with a pediatric cardiologist for appropriateness for inclusion in the study.
- 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 repeat, the subject may proceed into the trial.
- Known or suspected allergy to trial medication, excipients, or related products
- The receipt of any investigational drug within 90 days prior to this trial
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
Kimberly Boucher, RN, 202-476-1403, kboucher@childrensnational.org
About this study
Official title: Vosoritide for Treatment of Short Stature in Girls With Turner Syndrome
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
- Turner Syndrome, Short Stature
- Being tested
- Vosoritide
- Keywords
- Turner syndrome, Short Stature, Vosoritide
- Medical subject headings
- Turner Syndrome, Dwarfism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VosoritideDrug
Also called Voxzogo
Vosoritide administered daily via subcutaneous injection for 12 months using the FDA approved weight-based dosing band strategy for achondroplasia.
| Study group | Role | What they receive |
|---|---|---|
| Vosoritide treatment arm | Experimental | Vosoritide will be administered daily via subcutaneous injection for 12 months using the FDA approved weight-based dosing band strategy for achondroplasia. Receives: Drug: Vosoritide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events
Number of treatment-emergent adverse events or serious adverse events per study participant
Measured: 12 months
Change from baseline in annualized growth velocity
To evaluate the change from baseline in annualized growth velocity after 12 months of daily subcutaneous injections of vosoritide
Measured: 12 months
Change from baseline in age-sex standardized height standard deviation score
To evaluate the change from baseline in age-sex standardized height standard deviation score (SDS) after 12 months of daily subcutaneous injections of vosoritide
Measured: 12 months
Secondary outcomes (3)
Changes in seated height ratio
To evaluate the seated height ratio as a measure of body proportions compared to baseline after daily subcutaneous injections of vosoritide
Measured: 12 months
Changes in arm span minus standing height
To evaluate the arm span minus standing height as a measure of body proportion compared to baseline after daily subcutaneous injections of vosoritide
Measured: 12 months
Change in Bone Age
To evaluate changes from baseline in bone age/chronological age after 12 months of daily subcutaneous injections of vosoritide
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 May 2023
- Study start12 Apr 2024
- Primary completionSep 2026 (estimated)
- Study completionSep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Turner SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Roopa Kanakatti Shankar, MBBS, MS
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Roopa Kanakatti Shankar, MBBS, MS, Children's National Research Institute
- Investigators
- Roopa Kanakatti Shankar, MBBS, MS, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05849389
- Sponsor's protocol ID
- STUDY0000567
- Status last verified
- Jun 2024
- First submitted
- 28 Apr 2023
- Last updated
- 24 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Vosoritide for Treatment of Short Stature in Girls With Turner Syndrome
- ClinicalTrials.gov ID
- NCT05849389
- Acronym / sponsor ID
- STUDY0000567
- Conditions
- Turner Syndrome, Short Stature
- Keywords
- Turner syndrome, Short Stature, Vosoritide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Roopa Kanakatti Shankar, MBBS, MSUniversity, hospital or other
- Study start
- 12 Apr 2024 (actual)
- Primary completion
- Sep 2026 (estimated)
- Study completion
- Sep 2026 (estimated)
- First posted
- 8 May 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vosoritide treatment arm | Experimental | Drug: VosoritideVosoritide will be administered daily via subcutaneous injection for 12 months using the FDA approved weight-based dosing band strategy for achondroplasia. |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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.
- 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.
Key exclusion criteria (13 listed)
- 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 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.
- Prior or concomitant treatment with any form of estrogen, gonadotropin-releasing hormone (GnRH) analog, aromatase inhibitor or oxandrolone
- History of any type of malignancy
- Subjects known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| VosoritideAlso: VoxzogoVosoritide administered daily via subcutaneous injection for 12 months using the FDA approved weight-based dosing band strategy for achondroplasia. | Drug | Vosoritide treatment arm |
Primary outcome
Incidence of treatment-emergent adverse events
Time frame: 12 months
Number of treatment-emergent adverse events or serious adverse events per study participant
Change from baseline in annualized growth velocity
Time frame: 12 months
To evaluate the change from baseline in annualized growth velocity after 12 months of daily subcutaneous injections of vosoritide
Change from baseline in age-sex standardized height standard deviation score
Time frame: 12 months
To evaluate the change from baseline in age-sex standardized height standard deviation score (SDS) after 12 months of daily subcutaneous injections of vosoritide
3 secondary outcomes
Changes in seated height ratio
Time frame: 12 months
Changes in arm span minus standing height
Time frame: 12 months
Change in Bone Age
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Jun 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Roopa Kanakatti Shankar, MBBS, MS principal investigator
Children's National Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vosoritide in people with Turner Syndrome and Short Stature, run by Roopa Kanakatti Shankar, MBBS, MS at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 24 Jun 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Roopa Kanakatti Shankar, MBBS, MS (202-476-2121, roopa.shankar@childrensnational.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (time frame: 12 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Jun 2024.