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New treatment option for Noonan Syndrome
Official title A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
ClinicalTrials.gov ID: NCT06668805
What this study is testing
What is Vosoritide Injection?
Vosoritide Injection is an investigational medicine, being studied as a potential treatment for noonan syndrome.
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
- The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
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
You may be able to join if
- Participants must be ≥ 3 years old, and \< 11 years old (females) or \< 12 years old (males), at the time of signing the informed consent form
- A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.
- A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of...
- Tanner Stage 1, at time of signing the ICF.
- Previous or current hGH treatment for short stature associated with their condition.
You likely can't join if
- Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.
- Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal...
- Bone age advanced beyond chronological age by more than 2 years.
- Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
- Have an unstable condition likely to require surgical intervention during the study.
- Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using...
See the full eligibility criteria
- Participants must be ≥ 3 years old, and \< 11 years old (females) or \< 12 years old (males), at the time of signing the informed consent form
- A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.
- A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile for height) in reference to the general population of the same age and sex.
- Tanner Stage 1, at time of signing the ICF.
- Previous or current hGH treatment for short stature associated with their condition.
- Inadequate growth confirmed with an AGV that is less than age- and sex-matched average stature AGV determined using median heights from CDC growth charts
- Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.
- Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and...
- Bone age advanced beyond chronological age by more than 2 years.
- Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
- Have an unstable condition likely to require surgical intervention during the study.
- Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays.
- Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period.
- Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Orange, California, United States
- Aurora, Colorado, United States
- Wilmington, Delaware, United States
- Washington D.C., District of Columbia, United States
- Miami, Florida, United States
- Atlanta, Georgia, United States
- Boise, Idaho, United States
- Chicago, Illinois, United States
- Indianapolis, Indiana, United States
- Iowa City, Iowa, United States
- Lexington, Kentucky, United States
- Boston, Massachusetts, United States
- Minneapolis, Minnesota, United States
- New York, New York, United States
- The Bronx, New York, United States
- Charlotte, North Carolina, United States
- Cincinnati, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Nashville, Tennessee, United States
+ 16 more site(s).
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 all sexes, 3 years to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Orange, California, United States; Aurora, Colorado, United States; Wilmington, Delaware, United States; Washington D.C., District of Columbia, United States; Miami, Florida, United States and 30 more location(s). 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.