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Trial · Interventional

Active, not recruitingPhase 2Registry data

A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

NCTNCT06668805 (opens ClinicalTrials.gov in a new tab)Other id111-211
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Active, not recruiting
Phase
Phase 2
Enrollment
30 participants (estimated)
Sponsor
BioMarin Pharmaceutical · Industry
Design
Randomized · Triple masking
Countries
7 countries

United States, Australia, Canada, France, Germany, Italy, Spain

Start
22 Nov 2024
Primary completion
Mar 2027 (estimated)
Completion
Sept 2041 (estimated)
First posted
31 Oct 2024
Last update posted
23 Sept 2026
Conditions
Noonan Syndrome

Timeline

1 trial · 1 ongoing

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2025203020352040
  1. Ph 2

    A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

    NCT06668805

    22 Nov 2024 to est. Sept 2041

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

  • Vosoritide InjectionDrug

Arms3

  • Experimental

    Vosoritide Dose 1 - Low Dose

    Drug: Vosoritide - Dose 1 Injection • Experimental Drug Lyophilized powder for reconstitution

  • Experimental

    Vosoritide Dose 2 - Medium Dose

    Drug: Vosoritide - Dose 2 Injection • Experimental Drug Lyophilized powder for reconstitution

  • Experimental

    Vosoritide Dose 3- High Dose

    Drug: Vosoritide Dose 3 Injection • Experimental Drug Lyophilized powder for reconstitution

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Change from baseline in Annualized Growth Velocity (AGV)

    Time frame · At 6 months

Secondary outcomes(20)

  1. Incidence of treatment-emergent adverse events

    Time frame · Until the end of the study, up to 15 years

  2. Incidence of new diagnosis of hypertrophic cardiomyopathy in children with Noonan syndrome

    Time frame · Every 12 months through the end of the study, up to 15 years

  3. Incidence of cardiac conditions requiring discontinuation of study treatment

    Time frame · Every 12 months through the end of the study, up to 15 years

  4. Change from baseline in height

    Time frame · Every 6 months through the end of the study, up to 15 years

  5. Change from baseline in height Z-score

    Time frame · Every 6 months through the end of the study, up to 15 years

  6. Change from baseline in 12-month interval AGV

    Time frame · Until the end of the study, up to 15 years

  7. Change from baseline in upper to lower body segment ratio

    Time frame · Until the end of the study, up to 15 years

  8. Change from baseline in arm span to height ratio

    Time frame · Until the end of the study, up to 15 years

  9. Change from baseline in height up to Final Adult Height (FAH)

    Time frame · Every 6 months through the end of the study, up to 15 years

  10. Change from baseline in height Z-score up to FAH

    Time frame · Every 6 months through the end of the study, up to 15 years

  11. 12-month interval AGV summarized by age and sex up to FAH

    Time frame · Every 12 months through the end of the study, up to 15 years

  12. Tanner stage over the course of the study

    Time frame · Every 6 months through the end of the study, up to 15 years

  13. Time vosoritide is present at maximum concentration (Tmax)

    Time frame · Every 6 months through the end of the study, up to 15 years

  14. Maximum vosoritide observed plasma concentration (Cmax)

    Time frame · Every 6 months through he end of the study, up to15 years

  15. Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)

    Time frame · Every 6 months through the end of the study, up to 15 years

  16. Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)

    Time frame · Every 6 months through the end of the study, up to 15 years

  17. Elimination half-life of vosoritide (t½)

    Time frame · Every 6 months through the end of the study, up to 15 years

  18. Apparent clearance of vosoritide (CL/F)

    Time frame · Every 6 months through the end of the study, up to 15 years

  19. Apparent volume of distribution of vosoritide (Vz/F)

    Time frame · Every 6 months through the end of the study, up to 15 years

  20. Change from pre-dose in urine cyclic guanine monophosphate (cGMP)

    Time frame · Every 6 months through the end of the study, up to 15 years

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex
All
Age
from 3 Years up to 11 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion 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 for height) in reference to the general population of the same age and sex. 4. Tanner Stage 1, at time of signing the ICF. 5. Previous or current hGH treatment for short stature associated with their condition. 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 Exclusion Criteria: 1. Diagnosis of systemic disease or condition that may cause short stature other than Noonan syndrome, eg, renal, neoplastic, 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.

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