A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Part of paid clinical trials in California City, California.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT06382155
Phase
PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Idiopathic Short Stature

Eligibility Criteria

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

Interventions

  • Vosoritide Injection — DRUG
    Experimental Drug Lyophilized powder for reconstitution
  • Human Growth Hormone — DRUG
    Commercial product containing somatotropin
  • Placebo — DRUG
    Lyophilized powder for reconstitution

Study Details

The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).

Key Dates

Start date
Oct 21, 2024
Status verified
May 2026
Primary completion
Jun 30, 2026
Completion
Dec 31, 2036

Study Design

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

Arms

  • Experimental: Vosoritide Dose 1 - Low Dose
    Vosoritide Dose 1 daily injection
  • Experimental: Vosoritide Dose 2 - Medium Dose
    Vosoritide Dose 2 daily injection
  • Experimental: Vosoritide Dose 3 - High Dose
    Vosoritide Dose 3 daily injection
  • Placebo Comparator: Placebo
    Placebo daily injection
  • Active Comparator: Human Growth Hormone
    hGH daily injection (US only)

Primary Outcome Measure

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

Central Contacts

Locations (35)

FacilityCityStateZIPSite coordinators
Children's Hospital of Orange County Main Campus - OrangeCalifornia CityCalifornia92868
Amrit Bhangoo
[email protected]
+17145098634
Consano Clinical Research, LLCCalifornia CityCalifornia78231
Daniel Katselnik
[email protected]
210-545-4900
Rady Children's Hospital - San DiegoCalifornia CityCalifornia92123
Mary (Marybeth) Patterson
[email protected]
858-966-4032
Children's Hospital Los AngelesLos AngelesCalifornia90027
Anna Ryabets-Lienhard
[email protected]
323-361-6478
Children's Hospital Oakland Research InstituteOaklandCalifornia94609
Dr Hannah Chesser
[email protected]
Center Of Excellence in Diabetes and EndocrinologySacramentoCalifornia95821
Dr Gnanagurudasan Prakasam
[email protected]
916-570-2756
Lundquist Institute for Biomedical Innovation (LA BioMed)TorranceCalifornia90502
Dr Lynda Polgreen
[email protected]
3102221961
The Children's National Research InstituteWashington D.C.District of Columbia20012
Dr Andrew Dauber
[email protected]
Nemours Children's Specialty Care JacksonvilleJacksonvilleFlorida32207
Dr Larry Fox
[email protected]
Nicklaus Children's HospitalMiamiFlorida33155
Dr Adriana Carrillo Iregui
[email protected]
Optimal Research SitesOrange CityFlorida32763
Dr Jaqueline Freyre-Cubano
[email protected]
386-218-5911
Nemours Children's Health System - Corporate HeadquartersPensacolaFlorida32514
Dr Jean-Claude Desmangles
[email protected]
850-505-4793
Centricity ResearchColumbusGeorgia31904
Steven Leichter Principal Investigator
[email protected]
833-3235646
St. Luke's Children's EndocrinologyBoiseIdaho83712-
Rocky Mountain Clinical Research - Idaho FallsIdaho FallsIdaho83404
Dr Joshua Smith
[email protected]
208-522-6005
University of Iowa Stead Family Children's HospitalIowa CityIowa52242
Dr Michael Tansey
[email protected]
(319) 356-4443
Kentucky Children's HospitalLexingtonKentucky40536
Dr Yuri Zarate
[email protected]
859-257-1000
University of Louisville School of MedicineLouisvilleKentucky40202
Dr Catalina Cabrera Salcedo
[email protected]
Velocity Clinical Research - MedPharmics - LafayetteLafayetteLouisiana70508
Dr Jibran Atwi
[email protected]
(337) 519-4742
Ochsner Health Center for Children - New OrleansNew OrleansLouisiana70121-
University of MichiganAnn ArborMichigan48109
Dr Inas H. Thomas
[email protected]
734-674-5175
University of Minnesota Masonic Children's HospitalMinneapolisMinnesota55454
Dr Bradley Miller
[email protected]
612-626-2916
UBMD PediatricsBuffaloNew York14203
Dr Teresa Quattrin
[email protected]
716-323-0170
NYU Langone Ambulatory Care Garden CityGarden CityNew York11530-
Atlantic Health Children's Specialty Center at MorristownNew YorkNew York07960
Dr Lawrence Silverman
[email protected]
973-971-4340
Icahn School of Medicine at Mount SinaiNew YorkNew York10029
Robert Rapaport
[email protected]
(212) 241-8523
The Clinical and Translational Science InstituteNew YorkNew York10016
Dr Emily Breidbart
[email protected]
(212) 263-5290
Children's Hospital at MontefioreThe BronxNew York10467
Dr Laurie Cohen
[email protected]
(718)920-4664
University of North Carolina Children's Specialty ClinicChapel HillNorth Carolina27514
Nina Jain
[email protected]
919-718-9512
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229
Dr Gajanthan Muthuvel
[email protected]
(513) 636-8444
Prisma Health Children's Hospital - MidlandsColumbiaSouth Carolina29203
Dr Alison Lunsford
[email protected]
803-434-7990
University of Texas Southwestern Medical CenterDallasTexas75390
Dr Nadia Merchant
[email protected]
214-456-5959
Cook Children's EndocrinologyFort WorthTexas76104-
Baylor College of MedicineHoustonTexas77030
Dr Lin Yuezhen
[email protected]
Seattle Children's HospitalSeattleWashington98105
Dr Patricia Fechner
[email protected]
206-884-8540

Find similar trials in California City, CA

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Children's Hospital of Orange County Main Campus - Orange· California City, CAConsano Clinical Research, LLC· California City, CARady Children's Hospital - San Diego· California City, CAChildren's Hospital Los Angeles· Los Angeles, CAChildren's Hospital Oakland Research Institute· Oakland, CACenter Of Excellence in Diabetes and Endocrinology· Sacramento, CA

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