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A Study of Vosoritide in Children with Noonan Syndrome with Inadequate Growth During or After Human Growth Hormone Treatment
EU CTIS ID: 2024-515861-33-00
What this study is testing
To evaluate the effect of 3 vosoritide doses on AGV after 6 months of treatment
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. 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.
- 2. A clinical diagnosis of Noonan syndrome
- 3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile in height) in reference to the general population of the same age and sex.
- 4. Tanner Stage 1, at time of signing the ICF.The criteria for Tanner Staging includes only genitalia stage for males and breast stage for females.
- 5. Previous or current hGH for the treatment of short stature associated with their condition. Note: this can include patients who have stopped hGH treatment due to intolerance provided they also demonstrate inadequate growth for a minimum of 6 months.
- 6. 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
You likely can't join if
- 1. Diagnosis of systemic disease or condition that may cause short stature other than or Noonan syndrome, eg, renal, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.
- 2. Bone age advanced beyond chronological age by more than 2 years
- 3. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension.
- 4. Have an unstable condition likely to require surgical intervention during the study.
- 5. 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.
- 6. Previous limb-lengthening surgery, or planned or expected to have limb-lengthening surgery during the study period.
See the full eligibility criteria
- 1. 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.
- 2. A clinical diagnosis of Noonan syndrome
- 3. A height assessment corresponding to a height Z-score of ≤ -1.28 SDs (below the 10th percentile in height) in reference to the general population of the same age and sex.
- 4. Tanner Stage 1, at time of signing the ICF.The criteria for Tanner Staging includes only genitalia stage for males and breast stage for females.
- 5. Previous or current hGH for the treatment of short stature associated with their condition. Note: this can include patients who have stopped hGH treatment due to intolerance provided they also demonstrate inadequate growth for a minimum of 6 months.
- 6. 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
- 1. Diagnosis of systemic disease or condition that may cause short stature other than or Noonan syndrome, eg, renal, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.
- 2. Bone age advanced beyond chronological age by more than 2 years
- 3. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension.
- 4. Have an unstable condition likely to require surgical intervention during the study.
- 5. 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.
- 6. Previous limb-lengthening surgery, or planned or expected to have limb-lengthening surgery during the study period.
- 7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period. Prior/Concurrent Clinical Study Experience
The study team makes the final eligibility decision.
Where it's taking place
- Australia
- United States
- Canada
- Turkey
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, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Australia; United States; Canada; Turkey. 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 EU CTIS; the study team decides eligibility.