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

Part of paid clinical trials in Los Angeles, California.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT06668805
Phase
PHASE2
Status
Recruiting
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Conditions

  • Noonan Syndrome

Eligibility Criteria

Sex
ALL
Age
3 Years - 11 Years
Healthy Volunteers
Not accepted

Interventions

Study Details

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.

Key Dates

Start date
Nov 22, 2024
Status verified
May 2026
Primary completion
Mar 31, 2027
Completion
Sep 30, 2041

Study Design

Enrollment
30 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • 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 Outcome Measure

Change from baseline in Annualized Growth Velocity (AGV) [ Time Frame: At 6 months ]

Central Contacts

Locations (22)

FacilityCityStateZIPSite coordinators
Childrens Hospital Los AngelesLos AngelesCalifornia90027
Anna Ryabets-Lienhard
[email protected]
323-361-3861
Children's Hospital of Orange County Main Campus - OrangeOrangeCalifornia92868
Amrit Bhangoo
[email protected]
7145098634
Children's Hospital ColoradoAuroraColorado80045
Shanlee Davis
[email protected]
720-777-5251
Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)WilmingtonDelaware19803
Chijioke Ikomi
[email protected]
302-651-5965
Children's National Medical CenterWashington D.C.District of Columbia20010
Andrew Dauber
[email protected]
Nicklaus Children's HospitalMiamiFlorida33155
Ana Maria Rodriguez Barreto
[email protected]
Emory University School of MedicineAtlantaGeorgia30322
William Wilcox
[email protected]
(404) 778-8518
St. Luke's Children's Endocrinology and DiabetesBoiseIdaho83712
Daniel Flynn
[email protected]
2083817340
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611
Carlos Prada
312-227-6120
Riley Hospital for Children at Indiana University HealthIndianapolisIndiana46202
John Fuqua
[email protected]
3172741201
University of IowaIowa CityIowa52242
Michael Tansey
(319) 356-4443
Kentucky Children's HospitalLexingtonKentucky40506
Yuri Zarate
[email protected]
859-257-1000
New York Medical CollegeBostonMassachusetts02115
Shilpa Mehta
[email protected]
M Health Fairview Pediatric Specialty Clinic - ExplorerMinneapolisMinnesota55454
Kyriakie Sarafoglou
[email protected]
Icahn School of Medicine at Mount SinaiNew YorkNew York10029
Cassie Mintz
[email protected]
Children's Hospital at MontefioreThe BronxNew York10467
Laurie Cohen
[email protected]
718-920-4664
Atrium Health Carolinas Medical CenterCharlotteNorth Carolina28203
Edwin Ferren
[email protected]
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-3026
Gajanthan Muthuvel
(513) 636-8444
Childrens Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Vaneeta Bamba
[email protected]
215-590-4470
Vanderbilt University Medical CenterNashvilleTennessee37232-2578
Scott Ward
[email protected]
615-936-5475
Children's Medical Center DallasDallasTexas75235
Nadia Merchant
[email protected]
214-456-5959
University of Texas Health Science Center at Houston (UT Health)HoustonTexas77030
David Rodriguez Buritica
[email protected]

Find similar trials in Los Angeles, CA

By research site
Childrens Hospital Los Angeles· Los Angeles, CAChildren's Hospital of Orange County Main Campus - Orange· Orange, CAChildren's Hospital Colorado· Aurora, CONemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)· Wilmington, DEChildren's National Medical Center· Washington D.C., DCNicklaus Children's Hospital· Miami, FL

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