A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biomarin Pharmaceutical Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hormonal diseases [C19]

Trial duration

10 Oct 2025 → ongoing

Decision date (initial)

2025-06-24

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

BioMarin Pharmaceutical Inc.

External identifiers

EU CT number

2024-520137-74-00

ClinicalTrials.gov

NCT06382155

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Efficacy, Dose response, Safety, Pharmacodynamic, Pharmacokinetic

Dose-Finding Phase: To evaluate the effect of 3 vosoritide doses versus placebo on AGV after 6 months of treatment
Long-Term Phase: To evaluate the effect of the therapeutic dose of vosoritide versus hGH on height and height Z-score after long-term treatment

Secondary objectives 7

1. 1. To evaluate the safety and tolerability of vosoritide
2. 2. To evaluate the effect of 3 vosoritide doses on linear growth after 6 months of treatment
3. 3. To evaluate the effect of the therapeutic dose of vosoritide on growth up to FAH
4. 4. To evaluate the PK profile of vosoritide
5. 5. To evaluate the effect of vosoritide on PD and bone growth biomarkers
6. 6. To evaluate the effect of vosoritide on bone age/chronological age
7. 7. To evaluate the effect of vosoritide on bone quality

Conditions and MedDRA coding

Idiopathic Short Stature (ISS)

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

Swedish Medical Products Agency, Food And Drug Administration, Federal Institute For Drugs And Medical Devices

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 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. 2. Tanner Stage 1, at time of signing the ICF (unless too young to stage).
3. 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 2

1. 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. 2. Previous treatment with a growth promoting agent

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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

Secondary endpoints 8

1. 1. Incidence of treatment-emergent adverse events over the course of the study
2. 2. Change from baseline in height Z-score at 6 months
3. 3. Change from baseline in height at each visit and at FAH
4. 4. Change from baseline in height Z-score at each visit and at FAH
5. 5. PK parameters (eg, Tmax, Cmax, AUC0-t, AUC0-inf, t1/2, CL/F, Vz/F)
6. 6. Change from baseline at prespecified timepoints in: • urine cGMP • serum CXM
7. 7. Change from baseline in bone age/chronological age at prespecified timepoints
8. 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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 3

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Biomarin Pharmaceutical Inc.

Sponsor organisation

Biomarin Pharmaceutical Inc.

Address

105 Digital Drive

City

Novato

Postcode

94949-8703

Country

United States

Scientific contact point

Organisation

Biomarin Pharmaceutical Inc.

Contact name

BioMarin Pharmaceutical Inc.

Public contact point

Organisation

Biomarin Pharmaceutical Inc.

Contact name

BioMarin Pharmaceutical Inc.

Third parties 11

Locations

3 EU/EEA countries · 8 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 75 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications