A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
BioMarin Pharmaceutical · NCT06382155
Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.
Key facts
- Compensation
- Not posted. Ask the study team before screening.
- Who can join
- Ages 3–11 · All sexes
- Healthy volunteers
- Not accepted (a condition is required)
- Study type
- Interventional · Phase 2
- Target enrollment
- 100
- US sites recruiting
- 31
- Study start
- October 21, 2024
- Record last updated
- May 22, 2026
Status re-checked against ClinicalTrials.gov within the last 12 hours.
What the study is about
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).
Being studied: Vosoritide Injection; Human Growth Hormone; Placebo
Conditions: Idiopathic Short Stature
Eligibility criteria
Copied from the official record. The study team makes the final decision at screening.
Show full criteria
Key Inclusion Criteria
- Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts
- If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs).
Key Exclusions
- Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency.
- Previous treatment with a growth promoting agent
Recruiting US locations
Consano Clinical Research, LLC
California City, CA 78231
Site contact: Daniel Katselnik · 210-545-4900 · katselnik.MD@ConsanoSA.com
Rady Children's Hospital - San Diego
California City, CA 92123
Site contact: Mary (Marybeth) Patterson · 858-966-4032 · mpatterson2@rchsd.org
Children's Hospital of Orange County Main Campus - Orange
California City, CA 92868
Site contact: Amrit Bhangoo · +17145098634 · abhangoo@choc.org
Children's Hospital Los Angeles
Los Angeles, CA 90027
Site contact: Anna Ryabets-Lienhard · 323-361-6478 · aryabets@chla.usc.edu
Children's Hospital Oakland Research Institute
Oakland, CA 94609
Site contact: Dr Hannah Chesser · hannah.chesser@ucsf.edu
Center Of Excellence in Diabetes and Endocrinology
Sacramento, CA 95821
Site contact: Dr Gnanagurudasan Prakasam · 916-570-2756 · Gnanagurudasan.Prakasam@sutterhealth.org
Lundquist Institute for Biomedical Innovation (LA BioMed)
Torrance, CA 90502
Site contact: Dr Lynda Polgreen · 3102221961 · lpolgreen@lundquist.org
The Children's National Research Institute
Washington D.C., DC 20012
Site contact: Dr Andrew Dauber · adauber@childrensnational.org
Nemours Children's Specialty Care Jacksonville
Jacksonville, FL 32207
Site contact: Dr Larry Fox · larry.fox@nemours.org
Nicklaus Children's Hospital
Miami, FL 33155
Site contact: Dr Adriana Carrillo Iregui · adriana.carrillo@nicklaushealth.org
Optimal Research Sites
Orange City, FL 32763
Site contact: Dr Jaqueline Freyre-Cubano · 386-218-5911 · jfreyre@optimalresearchsites.net
Nemours Children's Health System - Corporate Headquarters
Pensacola, FL 32514
Site contact: Dr Jean-Claude Desmangles · 850-505-4793 · jean-claude.desmangles@nemours.ort
Centricity Research
Columbus, GA 31904
Site contact: Steven Leichter Principal Investigator · 833-3235646 · sleichter@centricityresearch.com
Rocky Mountain Clinical Research - Idaho Falls
Idaho Falls, ID 83404
Site contact: Dr Joshua Smith · 208-522-6005 · jms-research@idahomed.com
University of Iowa Stead Family Children's Hospital
Iowa City, IA 52242
Site contact: Dr Michael Tansey · (319) 356-4443 · michael-tansey@uiowa.edu
Kentucky Children's Hospital
Lexington, KY 40536
Site contact: Dr Yuri Zarate · 859-257-1000 · yuri.zarate@uky.edu
University of Louisville School of Medicine
Louisville, KY 40202
Site contact: Dr Catalina Cabrera Salcedo · catalina.salcedo@louisville.edu
Velocity Clinical Research - MedPharmics - Lafayette
Lafayette, LA 70508
Site contact: Dr Jibran Atwi · (337) 519-4742 · jiatwi@velocityclinical.com
University of Michigan
Ann Arbor, MI 48109
Site contact: Dr Inas H. Thomas · 734-674-5175 · inash@med.umich.edu
University of Minnesota Masonic Children's Hospital
Minneapolis, MN 55454
Site contact: Dr Bradley Miller · 612-626-2916 · mille685@umn.edu
UBMD Pediatrics
Buffalo, NY 14203
Site contact: Dr Teresa Quattrin · 716-323-0170 · tquattrin@upa.chob.edu
Atlantic Health Children's Specialty Center at Morristown
New York, NY 07960
Site contact: Dr Lawrence Silverman · 973-971-4340 · lawrence.silverman@atlantichealth.org
The Clinical and Translational Science Institute
New York, NY 10016
Site contact: Dr Emily Breidbart · (212) 263-5290 · emily.breidbart@nyulangone.org
Icahn School of Medicine at Mount Sinai
New York, NY 10029
Site contact: Robert Rapaport · (212) 241-8523 · robert.rapaport@mountsinai.org
Children's Hospital at Montefiore
The Bronx, NY 10467
Site contact: Dr Laurie Cohen · (718)920-4664 · lacohen@montefiore.org
University of North Carolina Children's Specialty Clinic
Chapel Hill, NC 27514
Site contact: Nina Jain · 919-718-9512 · nina_jain@med.unc.edu
Cincinnati Children's Hospital Medical Center
Cincinnati, OH 45229
Site contact: Dr Gajanthan Muthuvel · (513) 636-8444 · gajanthan.muthuvel@cchmc.org
Prisma Health Children's Hospital - Midlands
Columbia, SC 29203
Site contact: Dr Alison Lunsford · 803-434-7990 · alison.lunsford@prismahealth.org
University of Texas Southwestern Medical Center
Dallas, TX 75390
Site contact: Dr Nadia Merchant · 214-456-5959 · nadia.merchant@utsouthwestern.edu
Baylor College of Medicine
Houston, TX 77030
Site contact: Dr Lin Yuezhen · yuezhenl@bcm.edu
Seattle Children's Hospital
Seattle, WA 98105
Site contact: Dr Patricia Fechner · 206-884-8540 · patricia.fechner@seattlechildrens.org
Study contacts (from the official record)
- Trial Specialist · +1 415.475.5852 · medinfo@bmrn.com