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Active, not recruitingNCT06668805Updated Sep 23, 2026

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

A Phase 2 interventional study of Vosoritide Injection in Noonan Syndrome, sponsored by BioMarin Pharmaceutical. Active, not recruiting at 38 sites in 7 countries. Open to participants aged 3 Years to 11 Years. Per ClinicalTrials.gov, last updated 2026-09-23.

Sponsored by BioMarin Pharmaceutical · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
30
Allocation
Randomized
Ages
3 Years to 11 Years
Sex
All
01

Study summary

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.

Read the detailed description

This is a Phase 2, randomized, multicenter, study of vosoritide in children with Noonan syndrome who have inadequate growth during or after human growth hormone (hGH) treatment. The study is intended to characterize the short-term efficacy and safety of 3 dosing regimens of vosoritide. The efficacy and safety of the vosoritide therapeutic dose will be further evaluated, and an analysis of the impact of vosoritide on final adult height (FAH).

02

Conditions studied

  • Noonan Syndrome

Keywords

  • Short Stature
  • Musculoskeletal Diseases
  • Bone Diseases
  • Developmental Endocrine System Diseases Natriuretic Peptide, C-Type
03

In context

Noonan Syndrome

49 studies on the registry are indexed under Noonan Syndrome; 21 are open to participants now.

This study's planned enrollment of 30 is below the median of 52 across 32 interventional studies indexed under Noonan Syndrome.

Browse Noonan Syndrome studies →

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 112 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Browse BioMarin Pharmaceutical studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
3 Years to 11 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 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

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.
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Care provider, Investigator)
Enrollment
30 participants (estimated)

Study arms

  • Experimental
    Vosoritide Dose 1 - Low Dose

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

    Drug: Vosoritide Injection

  • Experimental
    Vosoritide Dose 2 - Medium Dose

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

    Drug: Vosoritide Injection

  • Experimental
    Vosoritide Dose 3- High Dose

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

    Drug: Vosoritide Injection

Interventions

06

What researchers measure

Primary outcomes

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

    Time frame: At 6 months

Secondary outcomes

  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

  21. Change from baseline in serum collagen X marker (CXM)

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

  22. Change from baseline in bone age/chronological age

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

  23. Change from baseline in total body (less head) BMD Z-score

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

  24. Change from baseline in lumbar spine bone mineral density (BMD) Z-score

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

  25. Change from baseline in total body (less head) bone mineral content (BMC)

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

  26. Change from baseline in lumbar spine BMC. Change from baseline in lower extremity BMD/BMC [Time Frame: Every 12 months through the end of the study, up to 15 years].

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

  27. Change in the growth plates, long bone growth and bone morphology based on whole length lower extremity X-rays [Time Frame: Every 12 months through the end of the study, up to 15 years]

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

  28. Incidence of bone-related events of special interest (fracture, slipped capital femoral epiphysis and avascular necrosis or osteonecrosis)

    Time frame: Throughout study

  29. Change from baseline in the physical domain score and total score of the QoLISSY

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

  30. Change from baseline in the physical and social domain scores and total score of the PedsQL

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

  31. Change from baseline in PGI-S and CaGI-S item scores

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

  32. PGI-C and CaGI-C item scores

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

  33. Change from baseline in PROMIS-SF Physical Activity score

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

  34. Change from baseline in KABC-II NVI scores

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

07

Study locations

38 sites
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 23, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06668805
Lead sponsor
BioMarin Pharmaceutical
Responsible party
Sponsor
First posted
Oct 31, 2024
Start date
Nov 22, 2024
Primary completion
Mar 2027 (estimated)
Completion
Sep 2041 (estimated)
Last update
Sep 23, 2026

Study contacts

Medical Director, MD, PhD
study director · BioMarin Pharmaceutical

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.

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