A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
Purpose
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).
Condition
- Idiopathic Short Stature
Eligibility
- Eligible Ages
- Between 3 Years and 11 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Criteria
Key Inclusion Criteria:
1. 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
2. Tanner Stage 1, at time of signing the ICF (unless too young to stage).
Key Exclusions:
1. 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)
2. Previous treatment with a growth promoting agent
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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) |
|
Recruiting Locations
California City, California 78231
California City, California 92123
California City, California 92868
Los Angeles, California 90027
Oakland, California 94609
Sacramento, California 95821
Torrance, California 90502
Washington D.C., District of Columbia 20012
Jacksonville, Florida 32207
Miami, Florida 33155
Orange City, Florida 32763
Pensacola, Florida 32514
Columbus, Georgia 31904
Boise, Idaho 83712
Idaho Falls, Idaho 83404
Iowa City, Iowa 52242
Lexington, Kentucky 40536
Louisville, Kentucky 40202
Lafayette, Louisiana 70508
Ann Arbor, Michigan 48109
Minneapolis, Minnesota 55454
Buffalo, New York 14203
New York, New York 07960
New York, New York 10016
New York, New York 10029
The Bronx, New York 10467
Chapel Hill, North Carolina 27514
Cincinnati, Ohio 45229
Columbia, South Carolina 29203
Dallas, Texas 75390
Fort Worth, Texas 76104
Seattle, Washington 98105
More Details
- NCT ID
- NCT06382155
- Status
- Recruiting
- Sponsor
- BioMarin Pharmaceutical
Detailed Description
Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years.