Current partner codePEPTIDESDE
NCT06668805·Phase 2·INTERVENTIONAL

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

Status

Recruiting

Phase

Phase 2

Enrollment

30

Locations

36

Results

Not posted

Publications

0

Study summary

What the protocol is 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.

Full 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).

Interventions

Treatment arms and agents.

DRUG

Vosoritide Injection

Modified recombinant human C-type natriuretic peptide Vosoritide

Timeline

From registration to results.

  1. First posted

    Oct 31, 2024

  2. Study start

    Nov 22, 2024

  3. Primary completion

    Mar 2027

  4. Study completion

    Sep 2041

  5. Results posted

    Not reported

  6. Registry updated

    May 11, 2026

Outcomes

What the study measures.

Primary outcomes

Change from baseline in Annualized Growth Velocity (AGV)

Time frame · At 6 months

Secondary outcomes

Incidence of treatment-emergent adverse events

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

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

Incidence of cardiac conditions requiring discontinuation of study treatment

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

Change from baseline in height

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

Change from baseline in height Z-score

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

Change from baseline in 12-month interval AGV

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

Change from baseline in upper to lower body segment ratio

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

Change from baseline in arm span to height ratio

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

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

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

Eligibility

Who can take part.

Minimum age
3 Years
Maximum age
11 Years
Sex
ALL
Healthy volunteers
No

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 genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or 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. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia. 2. 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 metabolic disease. 3. Bone age advanced beyond chronological age by more than 2 years. 4. Uncorrected congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension, 5. Have an unstable condition likely to require surgical intervention during the study. 6. 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. 7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period. 8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.

Study locations

36 registered sites.

Australia · Canada · France · Germany · Italy · Spain · United States. Showing up to 24 locations stored in the fast local snapshot.

Childrens Hospital Los Angeles

Los Angeles, California, United States

Children's Hospital of Orange County Main Campus - Orange

Orange, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)

Wilmington, Delaware, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Nicklaus Children's Hospital

Miami, Florida, United States

Emory University School of Medicine

Atlanta, Georgia, United States

St. Luke's Children's Endocrinology and Diabetes

Boise, Idaho, United States

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States

University of Iowa

Iowa City, Iowa, United States

Kentucky Children's Hospital

Lexington, Kentucky, United States

New York Medical College

Boston, Massachusetts, United States

M Health Fairview Pediatric Specialty Clinic - Explorer

Minneapolis, Minnesota, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Children's Hospital at Montefiore

The Bronx, New York, United States

Atrium Health Carolinas Medical Center

Charlotte, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Childrens Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Children's Medical Center Dallas

Dallas, Texas, United States

University of Texas Health Science Center at Houston (UT Health)

Houston, Texas, United States

Royal North Shore Hospital

Saint Leonards, New South Wales, Australia

Childrens Health Queensland Hospital and Health Service

South Brisbane, Queensland, Australia

Publications

Results and literature.

No PMID-linked publications were present in this registry snapshot.

Primary links

Continue at the source.

Related trials

More studies on Vosoritide.

Related PeptideStat pages

Put the record in context.

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