Idiopathic Short StaturePhase 2NCT06382155
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
A Phase 2 study testing Vosoritide Injection and Human Growth Hormone in people with Idiopathic Short Stature, run by BioMarin Pharmaceutical at 47 sites in 6 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 3 to 11 years
- Conditions the study is for
- Idiopathic Short Stature
- What is being tested?
- Vosoritide Injection and Human Growth Hormone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 100 participants
- Where
- 47 sitesin 6 countries
- Study dates
- 21 Oct 2024to Dec 2036
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
None listed.
Trial locations and contacts
47 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
47 sites in 6 countries
United States35 sites, 31 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
Dr Inas H. Thomas, 734-674-5175, inash@med.umich.edu
- Withdrawn
St. Luke's Children's Endocrinology
Boise, Idaho, 83712
- Recruiting
UBMD Pediatrics
Buffalo, New York, 14203
Dr Teresa Quattrin, 716-323-0170, tquattrin@upa.chob.edu
- Recruiting
Consano Clinical Research, LLC
California City, California, 78231
Daniel Katselnik, 210-545-4900, katselnik.MD@ConsanoSA.com
- Recruiting
Rady Children's Hospital - San Diego
California City, California, 92123
Mary (Marybeth) Patterson, 858-966-4032, mpatterson2@rchsd.org
- Recruiting
Children's Hospital of Orange County Main Campus - Orange
California City, California, 92868
Amrit Bhangoo, +17145098634, abhangoo@choc.org
- Recruiting
University of North Carolina Children's Specialty Clinic
Chapel Hill, North Carolina, 27514
Nina Jain, 919-718-9512, nina_jain@med.unc.edu
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Dr Gajanthan Muthuvel, (513) 636-8444, gajanthan.muthuvel@cchmc.org
- Recruiting
Prisma Health Children's Hospital - Midlands
Columbia, South Carolina, 29203
Dr Alison Lunsford, 803-434-7990, alison.lunsford@prismahealth.org
- Recruiting
Centricity Research
Columbus, Georgia, 31904
Steven Leichter Principal Investigator, 833-3235646, sleichter@centricityresearch.com
- Recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Dr Nadia Merchant, 214-456-5959, nadia.merchant@utsouthwestern.edu
- Withdrawn
Cook Children's Endocrinology
Fort Worth, Texas, 76104
- Withdrawn
NYU Langone Ambulatory Care Garden City
Garden City, New York, 11530
- Recruiting
Baylor College of Medicine
Houston, Texas, 77030
Dr Lin Yuezhen, yuezhenl@bcm.edu
- Recruiting
Rocky Mountain Clinical Research - Idaho Falls
Idaho Falls, Idaho, 83404
Dr Joshua Smith, 208-522-6005, jms-research@idahomed.com
- Recruiting
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242
Dr Michael Tansey, (319) 356-4443, michael-tansey@uiowa.edu
- Recruiting
Nemours Children's Specialty Care Jacksonville
Jacksonville, Florida, 32207
Dr Larry Fox, larry.fox@nemours.org
- Recruiting
Velocity Clinical Research - MedPharmics - Lafayette
Lafayette, Louisiana, 70508
Dr Jibran Atwi, (337) 519-4742, jiatwi@velocityclinical.com
- Recruiting
Kentucky Children's Hospital
Lexington, Kentucky, 40536
Dr Yuri Zarate, 859-257-1000, yuri.zarate@uky.edu
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Anna Ryabets-Lienhard, 323-361-6478, aryabets@chla.usc.edu
- Recruiting
University of Louisville School of Medicine
Louisville, Kentucky, 40202
Dr Catalina Cabrera Salcedo, catalina.salcedo@louisville.edu
- Recruiting
Nicklaus Children's Hospital
Miami, Florida, 33155
Dr Adriana Carrillo Iregui, adriana.carrillo@nicklaushealth.org
- Recruiting
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55454
Dr Bradley Miller, 612-626-2916, mille685@umn.edu
- Withdrawn
Ochsner Health Center for Children - New Orleans
New Orleans, Louisiana, 70121
- Recruiting
Atlantic Health Children's Specialty Center at Morristown
New York, New York, 07960
Dr Lawrence Silverman, 973-971-4340, lawrence.silverman@atlantichealth.org
- Recruiting
The Clinical and Translational Science Institute
New York, New York, 10016
Dr Emily Breidbart, (212) 263-5290, emily.breidbart@nyulangone.org
- Recruiting
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
Robert Rapaport, (212) 241-8523, robert.rapaport@mountsinai.org
- Recruiting
Children's Hospital Oakland Research Institute
Oakland, California, 94609
Dr Hannah Chesser, hannah.chesser@ucsf.edu
- Recruiting
Optimal Research Sites
Orange City, Florida, 32763
Dr Jaqueline Freyre-Cubano, 386-218-5911, jfreyre@optimalresearchsites.net
- Recruiting
Nemours Children's Health System - Corporate Headquarters
Pensacola, Florida, 32514
Dr Jean-Claude Desmangles, 850-505-4793, jean-claude.desmangles@nemours.ort
- Recruiting
Center Of Excellence in Diabetes and Endocrinology
Sacramento, California, 95821
Dr Gnanagurudasan Prakasam, 916-570-2756, Gnanagurudasan.Prakasam@sutterhealth.org
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Dr Patricia Fechner, 206-884-8540, patricia.fechner@seattlechildrens.org
- Recruiting
Children's Hospital at Montefiore
The Bronx, New York, 10467
Dr Laurie Cohen, (718)920-4664, lacohen@montefiore.org
- Recruiting
Lundquist Institute for Biomedical Innovation (LA BioMed)
Torrance, California, 90502
Dr Lynda Polgreen, 3102221961, lpolgreen@lundquist.org
- Recruiting
The Children's National Research Institute
Washington D.C., District of Columbia, 20012
Dr Andrew Dauber, adauber@childrensnational.org
France4 sites, 4 recruiting
- Recruiting
Centre Hospitalier Universitaire d'Angers
Angers, 49 933
Regis Coutant, +33 2 41 355 655, recoutant@chu-angers.fr
- Recruiting
Hôpital Bicêtre
Le Kremlin-Bicêtre, Île-de-France Region, 94270
Agnes Linglart, +33 01 45 21 78 33, agnes.linglart@aphp.fr
- Recruiting
Hôpital de la Timone - Enfants
Marseille, 13005
Rachel Reynaud, +33 4 91 38 83 83, rachel.reynaud@ap-hm.fr
- Recruiting
CHU de Toulouse - Hôpital des Enfants
Toulouse, 31059
Thomas Edouard, +33 5 61 99 28 24, edouard.t@chu-toulouse.fr
Italy3 sites, 3 recruiting
- Recruiting
Azienda Ospedaliero Universitaria - Ospedale Pediatrico Meyer
Florence, Florence, 50139
Stefano Stagi, 055-566-2585, stefano.stagi@unifi.it
- Recruiting
IRCCS Istituto Giannina Gaslini
Genova, 16147
Mohamad Maghnie, +39 01056362574, mohamadmaghnie@gaslini.org
- Recruiting
Ospedale Pediatrico Bambino Gesù
Roma, Rome, 00165
Annalisa Deodati, +39 06-685-92508, annalisa.deodati@opbg.net
South Korea3 sites, 3 recruiting
- Recruiting
Inha University Hospital
Incheon, 22332
Ji-Eun Lee, +82-32-890-3617, anicca@inha.ac.kr
- Recruiting
Chungnam National University Sejong Hospital (CNUSH)
Sejong, 30099
Yoo-Mi Kim, +82-10-2329-1514, ym.kim@cnu.ac.kr
- Recruiting
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, 13620
Jaehyun Kim, +82-31-787-7287, joyminer@gmail.com
Australia1 site, 1 recruiting
- Recruiting
Murdoch Children's Research Institute
Parkville, Victoria, 3052
Ravi Savarirayan, +61399366541, ravi.savarirayan@mcri.edu.au
Germany1 site, 1 recruiting
- Recruiting
Universitätsklinikum des Saarlandes
Homburg, Saarland, 66421
Tilman Rohrer, +49 (0) 68-41-16-2-83-01, tilman.rohrer@uks.eu
About this study
Official title: A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
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).
Read the 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.
A Treatment Completion visit will occur approximately 4 weeks after the last administration of investigational product. Participants who discontinue study treatment after ≥13 weeks of exposure may remain in the study and complete follow-up assessments per the protocol schedule (including annual and periodic assessments through End of Study). If a participant discontinues treatment and declines further participation, they will be asked to return for a final Treatment Completion and End of Study visit approximately 4 weeks after the last injection.
Safety monitoring includes regular clinical and imaging assessments, including hip and lower-extremity evaluations, monitoring for hypotension, fractures, and slipped capital femoral epiphysis, with oversight by an independent Data Monitoring Committee
An independent Data Monitoring Committee will periodically review safety data and may recommend treatment discontinuation based on predefined safety signals
- Conditions
- Idiopathic Short Stature
- Being tested
- Vosoritide Injection, Human Growth Hormone, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vosoritide InjectionDrug
Experimental Drug Lyophilized powder for reconstitution
Human Growth HormoneDrug
Commercial product containing somatotropin
PlaceboDrug
Lyophilized powder for reconstitution
| Study group | Role | What they receive |
|---|---|---|
| Vosoritide Dose 1 - Low Dose | Experimental | Vosoritide Dose 1 daily injection Receives: Drug: Vosoritide Injection |
| Vosoritide Dose 2 - Medium Dose | Experimental | Vosoritide Dose 2 daily injection Receives: Drug: Vosoritide Injection |
| Vosoritide Dose 3 - High Dose | Experimental | Vosoritide Dose 3 daily injection Receives: Drug: Vosoritide Injection |
| Placebo | Placebo comparator | Placebo daily injection Receives: Drug: Placebo |
| Human Growth Hormone | Active comparator | hGH daily injection (US only) Receives: Drug: Human Growth Hormone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in Annualized Growth Velocity (AGV)
Measured: At 6 months
Change from baseline in height
Measured: At 4 years
Change from baseline in height Z-score
Measured: At 4 years
Secondary outcomes (18)
Incidence of treatment-emergent adverse events
Measured: Until the end of the study, up to 15 years
Change from baseline in Height Z-score (average stature reference)
Measured: At 6 months
Change from baseline in height
Measured: Every 6 months through the end of study, up to 15 years
Change from baseline in height Z score
Measured: Every 6 months through the end of study, up to 15 years
Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP)
Measured: Every 6 months through the end of study, up to 15 years
Change from baseline at pre-specified timepoints in serum collagen X marker (CXM)
Measured: Every 6 months through the end of study, up to 15 years
Change from baseline in bone age minus chronological age at pre-specified timepoints
Measured: Every 6 months through the end of study, up to 15 years
Change from baseline in whole body (less head) bone mineral density (BMD) Z-score
Measured: Every 6 months through the end of study, up to 15 years
Change from baseline in lumbar spine BMD Z-score
Measured: Every 6 months through the end of study, up to 15 years
Change from baseline in total body (less head) bone mineral content (BMC)
Measured: Every 6 months through the end of study, up to 15 years
Change from baseline in lumbar spine BMC
Measured: Every 6 months through the end of study, up to 15 years
Maximum concentration (Cmax) of vosoritide in plasma
Measured: Every 6 months through the end of study, up to 15 years
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)
Measured: Every 6 months through the end of study, up to 15 years
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Measured: Every 6 months through the end of study, up to 15 years
Elimination half-life of vosoritide (t½)
Measured: Every 6 months through the end of study, up to 15 years
Apparent clearance of vosoritide
Measured: Every 6 months through the end of study, up to 15 years
Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)
Measured: Every 6 months through the end of study, up to 15 years
Time vosoritide is present at maximum concentration (Tmax)
Measured: Every 6 months through the end of study, up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2024
- Study start21 Oct 2024
- Primary completionJun 2026 (estimated)
- Study completionDec 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Short StatureStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director MD, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06382155
- Sponsor's protocol ID
- 111-210
- Status last verified
- May 2026
- First submitted
- 19 Apr 2024
- Last updated
- 22 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
- ClinicalTrials.gov ID
- NCT06382155
- Acronym / sponsor ID
- 111-210
- Conditions
- Idiopathic Short Stature
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 21 Oct 2024 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Dec 2036 (estimated)
- First posted
- 24 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
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.
A Treatment Completion visit will occur approximately 4 weeks after the last administration of investigational product. Participants who discontinue study treatment after ≥13 weeks of exposure may remain in the study and complete follow-up assessments per the protocol schedule (including annual and periodic assessments through End of Study). If a participant discontinues treatment and declines further participation, they will be asked to return for a final Treatment Completion and End of Study visit approximately 4 weeks after the last injection.
Safety monitoring includes regular clinical and imaging assessments, including hip and lower-extremity evaluations, monitoring for hypotension, fractures, and slipped capital femoral epiphysis, with oversight by an independent Data Monitoring Committee
An independent Data Monitoring Committee will periodically review safety data and may recommend treatment discontinuation based on predefined safety signals
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Vosoritide Dose 1 - Low Dose | Experimental | Drug: Vosoritide InjectionVosoritide Dose 1 daily injection |
| Vosoritide Dose 2 - Medium Dose | Experimental | Drug: Vosoritide InjectionVosoritide Dose 2 daily injection |
| Vosoritide Dose 3 - High Dose | Experimental | Drug: Vosoritide InjectionVosoritide Dose 3 daily injection |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo daily injection |
| Human Growth Hormone | Active comparator | Drug: Human Growth HormonehGH daily injection (US only) |
Who may qualify
- Ages
- 3 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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).
- 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
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vosoritide InjectionExperimental Drug Lyophilized powder for reconstitution | Drug | Vosoritide Dose 1 - Low Dose, Vosoritide Dose 2 - Medium Dose, Vosoritide Dose 3 - High Dose |
| Human Growth HormoneCommercial product containing somatotropin | Drug | Human Growth Hormone |
| PlaceboLyophilized powder for reconstitution | Drug | Placebo |
Primary outcome
Change from baseline in Annualized Growth Velocity (AGV)
Time frame: At 6 months
Change from baseline in height
Time frame: At 4 years
Change from baseline in height Z-score
Time frame: At 4 years
18 secondary outcomes
Incidence of treatment-emergent adverse events
Time frame: Until the end of the study, up to 15 years
Change from baseline in Height Z-score (average stature reference)
Time frame: At 6 months
Change from baseline in height
Time frame: Every 6 months through the end of study, up to 15 years
Change from baseline in height Z score
Time frame: Every 6 months through the end of study, up to 15 years
Change from baseline at prespecified timepoints in urine cyclic guanine monophosphate (cGMP)
Time frame: Every 6 months through the end of study, up to 15 years
Change from baseline at pre-specified timepoints in serum collagen X marker (CXM)
Time frame: Every 6 months through the end of study, up to 15 years
Change from baseline in bone age minus chronological age at pre-specified timepoints
Time frame: Every 6 months through the end of study, up to 15 years
Change from baseline in whole body (less head) bone mineral density (BMD) Z-score
Time frame: Every 6 months through the end of study, up to 15 years
Change from baseline in lumbar spine BMD Z-score
Time frame: Every 6 months through the end of study, up to 15 years
Change from baseline in total body (less head) bone mineral content (BMC)
Time frame: Every 6 months through the end of study, up to 15 years
Change from baseline in lumbar spine BMC
Time frame: Every 6 months through the end of study, up to 15 years
Maximum concentration (Cmax) of vosoritide in plasma
Time frame: Every 6 months through the end of study, up to 15 years
Area under the plasma vosoritide concentration time-curve from time 0 to infinity (AUC0-∞)
Time frame: Every 6 months through the end of study, up to 15 years
Area under the plasma vosoritide concentration time-curve from time 0 to the last measurable concentration (AUC0-t)
Time frame: Every 6 months through the end of study, up to 15 years
Elimination half-life of vosoritide (t½)
Time frame: Every 6 months through the end of study, up to 15 years
Apparent clearance of vosoritide
Time frame: Every 6 months through the end of study, up to 15 years
Apparent volume of distribution of vosoritide based upon the terminal phase (Vz/F)
Time frame: Every 6 months through the end of study, up to 15 years
Time vosoritide is present at maximum concentration (Tmax)
Time frame: Every 6 months through the end of study, up to 15 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director MD study director
BioMarin Pharmaceutical
47 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vosoritide Injection and Human Growth Hormone in people with Idiopathic Short Stature, run by BioMarin Pharmaceutical at 47 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 22 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 47 sites in United States, France, Italy, South Korea, Australia and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Specialist (+1 415.475.5852, medinfo@bmrn.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in Annualized Growth Velocity (AGV) (time frame: At 6 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2026.