Vosoritide Clinical Trials (2026): 10 Studies for Hypochondroplasia & Short Stature
Hipa.ai Research · Source: ClinicalTrials.gov / AACT
Synced daily from ClinicalTrials.gov via AACT. Last sync: .
10 Vosoritide clinical trials are catalogued on Hipa.ai from the AACT mirror of ClinicalTrials.gov, spanning 7 indications (including Hypochondroplasia & Short Stature) and 672 total planned enrollment. 2 Phase 3 Vosoritide trials and 6 Phase 2 are listed. 6 Vosoritide clinical trials are actively recruiting at 63 U.S. sites across 26 states. Most recent activity: NCT06668805 (Sep 2041).
Where to Participate: All Vosoritide Trial Sites in the U.S. (63 sites across 26 states)
Every actively recruiting Vosoritidetrial site, sorted by state then city. Each row links to the trial detail page (eligibility, contacts, full study record). Sites no longer enrolling at the location level are excluded. ClinicalTrials.gov / AACT does not provide street-level addresses; the map link uses the facility's geocoded coordinates where available.
| State | Facility | City | Trial | Map |
|---|---|---|---|---|
| AZ | Phoenix Children's Hospital - Thomas Campus (Main) | Phoenix85016 | NCT07126262 | Map |
| CA | Children's Hospital of Orange County Main Campus - Orange | California City92868 | NCT06382155 | Map |
| CA | Consano Clinical Research, LLC | California City78231 | NCT06382155 | Map |
| CA | Rady Children's Hospital - San Diego | California City92123 | NCT06382155 | Map |
| CA | Children's Hospital Los Angeles | Los Angeles90027 | NCT06382155 | Map |
| CA | Childrens Hospital Los Angeles | Los Angeles90027 | NCT06668805 | Map |
| CA | Benioff Children's Hospital - Oakland | Oakland94609 | NCT07126262 | Map |
| CA | Children's Hospital Oakland Research Institute | Oakland94609 | NCT06382155 | Map |
| CA | UCSF Benioff Children's Hospital Oakland | Oakland94609 | NCT07441876 | Map |
| CA | Children's Hospital of Orange County Main Campus - Orange | Orange92868 | NCT06668805 | Map |
| CA | Center Of Excellence in Diabetes and Endocrinology | Sacramento95821 | NCT06382155 | Map |
| CA | Lundquist Institute for Biomedical Innovation (LA BioMed) | Torrance90502 | NCT06382155 | Map |
| CO | Children's Hospital Colorado | Aurora80045 | NCT06668805 | Map |
| DE | Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children) | Wilmington19803 | NCT06668805 | Map |
| DC | Children's National Hospital | Washington D.C.20010 | NCT05849389 | Map |
| DC | Children's National Medical Center | Washington D.C.20010 | NCT06668805 | Map |
| DC | Children's National Medical Center | Washington D.C.20010 | NCT07126262 | Map |
| DC | The Children's National Research Institute | Washington D.C.20012 | NCT06382155 | Map |
| FL | Nemours Children's Specialty Care Jacksonville | Jacksonville32207 | NCT06382155 | Map |
| FL | Nicklaus Children's Hospital | Miami33155 | NCT06382155 | Map |
| FL | Optimal Research Sites | Orange City32763 | NCT06382155 | Map |
| FL | Nemours Children's Health System - Corporate Headquarters | Pensacola32514 | NCT06382155 | Map |
| GA | Emory University School of Medicine | Atlanta30322 | NCT06668805 | Map |
| GA | Centricity Research | Columbus31904 | NCT06382155 | Map |
| ID | St. Luke's Children's Endocrinology and Diabetes | Boise83712 | NCT06668805 | Map |
| ID | Rocky Mountain Clinical Research - Idaho Falls | Idaho Falls83404 | NCT06382155 | Map |
| IL | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago60611 | NCT06668805 | Map |
| IL | Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago60611 | NCT07126262 | Map |
| IN | Riley Hospital for Children at Indiana University Health | Indianapolis46202 | NCT06668805 | Map |
| IA | University of Iowa | Iowa City52242 | NCT06668805 | Map |
| IA | University of Iowa Stead Family Children's Hospital | Iowa City52242 | NCT06382155 | Map |
| KY | Kentucky Children's Hospital | Lexington40536 | NCT06382155 | Map |
| KY | Kentucky Children's Hospital | Lexington40506 | NCT06668805 | Map |
| KY | University of Louisville School of Medicine | Louisville40202 | NCT06382155 | Map |
| LA | Velocity Clinical Research - MedPharmics - Lafayette | Lafayette70508 | NCT06382155 | Map |
| MD | The Johns Hopkins University School of Medicine | Baltimore21205 | NCT07126262 | Map |
| MA | New York Medical College | Boston02115 | NCT06668805 | Map |
| MI | University of Michigan | Ann Arbor48109 | NCT06382155 | Map |
| MN | M Health Fairview Pediatric Specialty Clinic - Explorer | Minneapolis55454 | NCT06668805 | Map |
| MN | University of Minneasota Masonic Children's Hospital | Minneapolis55454 | NCT07126262 | Map |
| MN | University of Minnesota Masonic Children's Hospital | Minneapolis55454 | NCT06382155 | Map |
| MO | University of Missouri | Columbia65211 | NCT07126262 | Map |
| NY | UBMD Pediatrics | Buffalo14203 | NCT06382155 | Map |
| NY | Atlantic Health Children's Specialty Center at Morristown | New York07960 | NCT06382155 | Map |
| NY | Icahn School of Medicine at Mount Sinai | New York10029 | NCT06382155 | Map |
| NY | Icahn School of Medicine at Mount Sinai | New York10029 | NCT06668805 | Map |
| NY | The Clinical and Translational Science Institute | New York10016 | NCT06382155 | Map |
| NY | Children's Hospital at Montefiore | The Bronx10467 | NCT06382155 | Map |
| NY | Children's Hospital at Montefiore | The Bronx10467 | NCT06668805 | Map |
| NC | University of North Carolina Children's Specialty Clinic | Chapel Hill27514 | NCT06382155 | Map |
| NC | Atrium Health Carolinas Medical Center | Charlotte28203 | NCT06668805 | Map |
| OH | Cincinnati Children's Hospital Medical Center | Cincinnati45229 | NCT06382155 | Map |
| OH | Cincinnati Children's Hospital Medical Center | Cincinnati45229-3026 | NCT06668805 | Map |
| PA | Childrens Hospital of Philadelphia | Philadelphia19104 | NCT06668805 | Map |
| SC | Prisma Health Children's Hospital - Midlands | Columbia29203 | NCT06382155 | Map |
| TN | Vanderbilt University Medical Center | Nashville37232-2578 | NCT06668805 | Map |
| TX | Children's Medical Center Dallas | Dallas75235 | NCT06668805 | Map |
| TX | University of Texas Southwestern Medical Center | Dallas75390 | NCT06382155 | Map |
| TX | Baylor College of Medicine | Houston77030 | NCT06382155 | Map |
| TX | Texas Children Hospital, Baylor College of Medicine, Houston TX | Houston77030 | NCT07441876 | Map |
| TX | University of Texas Health Science Center at Houston (UT Health) | Houston77030 | NCT06668805 | Map |
| TX | Consano Clinical Research, LLC | San Antonio78231 | NCT07441876 | Map |
| WA | Seattle Children's Hospital | Seattle98105 | NCT06382155 | Map |
Vosoritide clinical trials at a glance: phase × recruitment status
| Phase | recruiting | active not recruiting | enrolling by invitation | Total |
|---|---|---|---|---|
| P1/P2 | 1 | 1 | ||
| P2 | 4 | 2 | 6 | |
| P2/P3 | 1 | 1 | ||
| P3 | 1 | 1 | 2 |
Recruiting Vosoritide clinical trials (6)
Studies currently enrolling participants. Click any NCT ID to view the trial detail page (eligibility, sites, contacts).
| NCT ID | Title | Phase | Indication | Enrollment | Started |
|---|---|---|---|---|---|
| NCT06668805 | A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment | P2 | Noonan Syndrome | 30 | Nov 2024 |
| NCT07073014 | CANOPY-HCH-EXTLong-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | P3 | Hypochondroplasia | 140 | Jun 2025 |
| NCT06382155 | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | P2 | Idiopathic Short Stature | 100 | Oct 2024 |
| NCT07441876 | ASPENStudy to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | P2/P3 | Achondroplasia | 160 | Apr 2026 |
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | P2 | Hypochondroplasia | 60 | Jul 2025 |
| NCT05849389 | Vosoritide for Short Stature in Turner Syndrome | P2 | Turner Syndrome | 20 | Apr 2024 |
Vosoritide clinical trials by indication
Hypochondroplasia (3)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT07073014 | CANOPY-HCH-EXT | P3 | enrolling by invitation | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia |
| NCT07126262 | — | P2 | recruiting | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months |
| NCT06455059 | CANOPY-HCH-3 | P3 | active not recruiting | Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia |
Achondroplasia (2)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT07441876 | ASPEN | P2/P3 | recruiting | Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia |
| NCT04554940 | — | P2 | active not recruiting | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia |
Noonan Syndrome (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT06668805 | — | P2 | recruiting | A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment |
Idiopathic Short Stature (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT06382155 | — | P2 | recruiting | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature |
Turner Syndrome (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT05849389 | — | P2 | recruiting | Vosoritide for Short Stature in Turner Syndrome |
MPS IVA (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT05845749 | — | P1/P2 | active not recruiting | Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI |
Short Stature (1)
| NCT | Acronym | Phase | Status | Title |
|---|---|---|---|---|
| NCT04219007 | — | P2 | active not recruiting | Vosoritide for Selected Genetic Causes of Short Stature |
Vosoritide pivotal trial highlights (Phase 2/3/4)
Phase-2/3/4 studies with named acronyms — the registration-grade trials most cited in label discussions and Wikipedia.
| Acronym | NCT | Phase | Status | Indication | Enrollment | PubMed |
|---|---|---|---|---|---|---|
| CANOPY-HCH-EXT | NCT07073014 | P3 | enrolling by invitation | Hypochondroplasia | 140 | — |
| CANOPY-HCH-3 | NCT06455059 | P3 | active not recruiting | Hypochondroplasia | 80 | — |
All Vosoritide trials (10)
Sorted by most recent activity (completion → start → posted date). Each row links to the trial detail page on Hipa.ai.
| NCT ID | Acronym | Title | Phase | Status | Enrollment | Sponsor | Started |
|---|---|---|---|---|---|---|---|
| NCT06668805 | A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment | P2 | recruiting | 30 | BioMarin Pharmaceutical | Nov 2024 | |
| NCT07073014 | CANOPY-HCH-EXT | Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia | P3 | enrolling by invitation | 140 | BioMarin Pharmaceutical | Jun 2025 |
| NCT06382155 | A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | P2 | recruiting | 100 | BioMarin Pharmaceutical | Oct 2024 | |
| NCT04219007 | Vosoritide for Selected Genetic Causes of Short Stature | P2 | active not recruiting | 56 | Andrew Dauber | Aug 2020 | |
| NCT05845749 | Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI | P1/P2 | active not recruiting | 6 | University of California, San Francisco | Sep 2023 | |
| NCT07441876 | ASPEN | Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | P2/P3 | recruiting | 160 | BioMarin Pharmaceutical | Apr 2026 |
| NCT07126262 | A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months | P2 | recruiting | 60 | BioMarin Pharmaceutical | Jul 2025 | |
| NCT04554940 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | P2 | active not recruiting | 20 | BioMarin Pharmaceutical | Oct 2020 | |
| NCT05849389 | Vosoritide for Short Stature in Turner Syndrome | P2 | recruiting | 20 | Roopa Kanakatti Shankar, MBBS, MS | Apr 2024 | |
| NCT06455059 | CANOPY-HCH-3 | Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia | P3 | active not recruiting | 80 | BioMarin Pharmaceutical | Jun 2024 |
Vosoritide clinical trials: frequently asked questions
- How many Vosoritide clinical trials are there?
- 10 Vosoritide clinical trials are catalogued on Hipa.ai, sourced from the AACT mirror of ClinicalTrials.gov. 6 are actively recruiting participants.
- Are Vosoritide clinical trials recruiting in 2026?
- Yes — 6 Vosoritide trials are actively recruiting in 2026, with 63 U.S. sites across 26 states. See the recruiting trials and the per-state site list further down this page.
- What conditions is Vosoritide being studied for?
- Vosoritide is being studied for Hypochondroplasia, Short Stature, Achondroplasia, MPS VI, Noonan Syndrome and 3 more. The trials are grouped by indication on this page.
- What phase is Vosoritide in?
- Vosoritide clinical trials span 2 Phase 3, 6 Phase 2. The phase × status matrix at the top of this page breaks down counts by phase and recruitment status.
- Who sponsors Vosoritide clinical trials?
- The lead sponsor across the most Vosoritide trials is BioMarin Pharmaceutical, with 3 additional lead sponsors listed in the data. Each trial row links to the full study detail page including sponsor information.
Sources and methodology
Trial inventory derived from the AACT mirror of ClinicalTrials.gov. Trials are filtered to those naming Vosoritideas an intervention. Phase, status, enrollment, sponsor, and dates are taken from the source `studies` table; primary indication is the first listed condition. Acronyms are extracted from each trial's brief title where present. PubMed links are populated for trials whose published primary outcomes have been indexed in our evidence cache.
Last refreshed .
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