Vosoritide for Idiopathic short stature

Inclusion criteria

• {"criterion_text":"- 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\n- 2. Tanner Stage 1, at time of signing the ICF (unless too young to stage).\n- 3. Participants must be ≥ 3 years old, and < 10 years old (female) or < 11 years old (males) at the time of signing the informed consent form (ICF)."}

Exclusion criteria

• {"criterion_text":"- 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)\n- 2. Previous treatment with a growth promoting agent"}

Primary endpoints

• {"endpoint_text":"- Dose-Finding Phase: Change from baseline in AGV at 6 months Long-Term Phase: Change from baseline in height and height Z-score at 4 years","definition_or_measurement_approach":"Change from baseline in annualized growth velocity (AGV) at 6 months (Dose-Finding Phase); change from baseline in height and height Z-score at 4 years (Long-Term Phase). Baseline AGV determined from at least 6 months of pretreatment growth assessments."}

Secondary endpoints

• {"endpoint_text":"- 1. Incidence of treatment-emergent adverse events over the course of the study","definition_or_measurement_approach":"Safety: incidence and reporting of treatment-emergent adverse events during study follow-up per protocol safety assessments."}
• {"endpoint_text":"- 2. Change from baseline in height Z-score at 6 months","definition_or_measurement_approach":"Change from baseline in height Z-score at 6 months compared to baseline measurements."}
• {"endpoint_text":"- 3. Change from baseline in height at each visit and at FAH","definition_or_measurement_approach":"Change from baseline in absolute height measured at each visit and at final adult height (FAH)."}
• {"endpoint_text":"- 4. Change from baseline in height Z-score at each visit and at FAH","definition_or_measurement_approach":"Change from baseline in height Z-score measured at each visit and at FAH."}
• {"endpoint_text":"- 5. PK parameters (eg, Tmax, Cmax, AUC0-t, AUC0-inf, t1/2, CL/F, Vz/F)","definition_or_measurement_approach":"Pharmacokinetic parameters including Tmax, Cmax, AUC0-t, AUC0-inf, t1/2, CL/F, Vz/F derived from plasma concentration-time data per PK sampling schedule."}
• {"endpoint_text":"- 6. Change from baseline at prespecified timepoints in: • urine cGMP • serum CXM","definition_or_measurement_approach":"Biomarker changes from baseline at prespecified timepoints: urinary cGMP and serum CXM measured using specified laboratory assays."}
• {"endpoint_text":"- 7. Change from baseline in bone age/chronological age at prespecified timepoints","definition_or_measurement_approach":"Change from baseline in bone age relative to chronological age assessed at prespecified timepoints (radiographic assessment)."}
• {"endpoint_text":"- 8. Change from baseline at each visit in the following, as measured by DXA: • total body (less head) BMD Z-score • lumbar spine BMD Z-score • total body (less head) BMC • lumbar spine BMC","definition_or_measurement_approach":"DXA-assessed changes from baseline at each visit for total body (less head) and lumbar spine BMD Z-scores and BMC measurements."}

Methods

• Germany: Recruitment materials include website text, digital ad campaign/social media ads, flyers, study brochure, patient letter, ICF flipbook, advocacy flyer and growth clinical trial material packet (documents listed under German associated Part II). Channels: website, digital ads/social media, printed flyers/brochures, site-distributed patient letters.
• Italy: Recruitment materials include ICF flipbook, patient brochure, social media and website ads, patient letter, advocacy flyer, digital ad campaigns, flyers and growth clinical trial material packet (documents listed under Italian associated Part II). Channels: website, social media, printed materials, site outreach.
• France: Recruitment materials include digital ad campaign, website text, advocacy flyer, flyers, patient letter, study brochure, ICF flipbook and growth clinical trial material packet (documents listed under French associated Part II). Channels: website, social media/digital ads, printed materials and advocacy engagement.

Germany

Earliest CTIS Part Ii Submission Date

12-06-2025

Latest Decision Or Authorization Date

08-07-2025

Processing Time Days

26

Number Of Sites

1

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

20-06-2025

Latest Decision Or Authorization Date

16-07-2025

Processing Time Days

26

Number Of Sites

3

Number Of Participants

9

France

Earliest CTIS Part Ii Submission Date

16-05-2025

Latest Decision Or Authorization Date

24-06-2025

Processing Time Days

39

Number Of Sites

4

Number Of Participants

20

Primary sponsor

Full Name

Biomarin Pharmaceutical Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Icon Clinical Research Limited

Responsibilities

Medical imaging, In-home health, Site and contract negotiation, Patient recruitment materials, Language service (and responsibilities coded as 1,12,15,2,4,5,8)

Name

Medpace Belgium

Responsibilities

Operational duties (code 4)

Name

Almac Clinical Services (Ireland) Limited

Responsibilities

Logistics/operational duties (code 14)

Name

Almac Clinical Services Limited

Responsibilities

Logistics/operational duties (code 14)

Third parties

• {"country":"United States","full_name":"Precision For Medicine Inc.","duties_or_roles":"Sample Storage (code 15)","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"Medpace Belgium","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient reimbursment (code 15)","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code 7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Germany","full_name":"Centogene GmbH","duties_or_roles":"code 4","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Multiple duties including codes 1,12,15 (Medical imaging, In-home health, Site and contract negotiation, Patient recruitment materials, Language service), 2,4,5,8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"code 3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Ireland","full_name":"Almac Clinical Services (Ireland) Limited","duties_or_roles":"code 14","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"code 14","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Yprime LLC","duties_or_roles":"code 7","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"code 4","organisation_type":"Pharmaceutical company"}