VOSORITIDE for Hypochondroplasia

Inclusion criteria

• {"criterion_text":"- 1. Participants must have completed the Week 52 visit for 111 303 or 111-212 and have open epiphyses as assessed by left hand antero posterior (AP) X-rays, by the Greulich and Pyle method (Greulich 1971) as per standard of care\n- 2. Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any study-related procedure.\n- 3. Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Baseline visit and be willing to have additional pregnancy tests during the study.\n- 4. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.\n- 5. Participants are willing and able to perform all study procedures as physically possible.\n- 6. Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training."}

Exclusion criteria

• {"criterion_text":"- 1. Permanently discontinued study treatment in the studies 111-303 or 111-212.\n- 10. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.\n- 2. Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed as per standard of care.\n- 3. Taking or planning to take any of the prohibited medications listed in Section 6.4.\n- 4. Planned or expected to have limb-lengthening surgery during the study period.\n- 5. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.\n- 6. Require any investigational agent prior to completion of study period.\n- 7. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy\n- 8. Have known hypersensitivity to vosoritide or its excipients.\n- 9. Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study."}

Primary endpoints

• {"endpoint_text":"- 1. Incidence of treatment emergent adverse events over time until FAH\n- 2. Change from baseline in height Z-score yearly until FAH\n- 3. Change from baseline in height yearly until FAH","definition_or_measurement_approach":"1. Measured as incidence of treatment-emergent adverse events (TEAEs) recorded over time until final adult height (FAH).\n2. Annual change from baseline in height Z-score measured yearly until FAH (baseline defined per parent study dosing initiation).\n3. Annual change from baseline in standing height measured yearly until FAH."}

Secondary endpoints

• {"endpoint_text":"- 1. Change from baseline in height at Week 52 of Study 111-308\n- 2. Change from baseline in height Z-score at Week 52 of Study 111-308 Baseline is based on first dose of study drug (vosoritide or placebo) in the parent study (111-303 or 111 212).","definition_or_measurement_approach":"1. Change from baseline in height assessed at Week 52 of this study (111-308).\n2. Change from baseline in height Z-score at Week 52; baseline is defined as the first dose of study drug (vosoritide or placebo) in the parent study (111-303 or 111-212)."}

Methods

• Country-specific recruitment procedures documented (K1 documents) for Germany, Italy, Spain, France (site-based recruitment at participating paediatric hospitals/clinics).
• Telephone pre-screening and telephone consent/data-consent handled by Scout Clinical (documents: Scout Pre-ICF Telephone, Scout Clinical Telephone Data Consent).
• Use of patient recruitment materials and participant reimbursement (sponsor duties list includes patient recruitment materials and patient reimbursement).
• Site and contract negotiation, in-home health services and language services provided as part of site support (listed in ICON responsibilities).
• Electronic platforms and eClinical support (vendors listed include Medidata, Suvoda, Yprime) used to support study procedures and remote processes.

Germany

Earliest CTIS Part Ii Submission Date

12-08-2025

Latest Decision Or Authorization Date

03-11-2025

Processing Time Days

83

Number Of Sites

2

Number Of Participants

14

Italy

Earliest CTIS Part Ii Submission Date

22-10-2025

Latest Decision Or Authorization Date

11-11-2025

Processing Time Days

20

Number Of Sites

3

Number Of Participants

14

Spain

Earliest CTIS Part Ii Submission Date

31-10-2025

Latest Decision Or Authorization Date

12-11-2025

Processing Time Days

12

Number Of Sites

1

Number Of Participants

6

France

Earliest CTIS Part Ii Submission Date

20-10-2025

Latest Decision Or Authorization Date

13-11-2025

Processing Time Days

24

Number Of Sites

4

Number Of Participants

10

Primary sponsor

Full Name

Biomarin Pharmaceutical Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Medpace Belgium

Name

Icon Clinical Research Limited

Responsibilities

Site and contract negotiation, Medical Imaging, In-home health, language services, Patient recruitement Materials.

Name

Icon Clinical Research Services (Imperial Clinical Research Services International Ltd.)

Responsibilities

printing

Name

Almac Clinical Services Limited

Name

Longboat Clinical Limited

Responsibilities

Site Training portal

Third parties

• {"country":"Belgium","full_name":"Medpace Belgium","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Ireland","full_name":"Longboat Clinical Limited","duties_or_roles":"Site Training portal","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Quipment","duties_or_roles":"Equipmental Rental","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Yprime LLC","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Precision For Medicine Inc.","duties_or_roles":"sample storage","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Clinical Outcomes Solutions Limited","duties_or_roles":"Data Collection & Analysis: Gathering and analyzing patient-reported data","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Site and contract negotiation, Medical Imaging, In-home health, language services, Patient recruitement Materials.","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Clinical Outcomes Solutions LLC","duties_or_roles":"Data Collection & Analysis: Gathering and analyzing patient-reported data","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Imperial Clinical Research Services International Ltd.","duties_or_roles":"printing","organisation_type":"Pharmaceutical company"}