Long-Term Extension Study of Vosoritide to Treat Children with Hypochondroplasia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biomarin Pharmaceutical Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

28 Jan 2026 → ongoing

Decision date (initial)

2025-11-13

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

BioMarin Pharmaceutical Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy

To evaluate the long-term safety, tolerability, and efficacy of vosoritide treatment until final adult height (FAHa) in patients from studies 111-303 and 111-212, respectively.

Secondary objectives 5

1. 1. To evaluate the maintenance effect of vosoritide treatment on standing height and height Z-score
2. 2. To evaluate the long-term effect of vosoritide treatment on growth velocity and body proportions until FAH
3. 3. To evaluate the long-term effect of vosoritide on bone age/chronological age
4. 4. To evaluate the long-term effect of vosoritide on bone metabolism biomarkers
5. 5. To evaluate long-term changes in HRQoL, as measured by QoLISSY

Conditions and MedDRA coding

Hypochondroplasia

Regulatory references

Scientific advice from competent authorities

Pharmaceuticals And Medical Devices Agency, Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-002033-PIP02-23

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. 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
2. 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.
3. 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.
4. 4. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.
5. 5. Participants are willing and able to perform all study procedures as physically possible.
6. 6. Parent(s) or caregiver(s) are willing to administer daily injections to the participants and willing to complete the required training.

Exclusion criteria 10

1. 1. Permanently discontinued study treatment in the studies 111-303 or 111-212.
2. 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.
3. 3. Taking or planning to take any of the prohibited medications listed in Section 6.4.
4. 4. Planned or expected to have limb-lengthening surgery during the study period.
5. 5. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.
6. 6. Require any investigational agent prior to completion of study period.
7. 7. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy
8. 8. Have known hypersensitivity to vosoritide or its excipients.
9. 9. Is pregnant or breastfeeding at Baseline or planning to become pregnant (self or partner) at any time during the study.
10. 10. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. 1. Incidence of treatment emergent adverse events over time until FAH
2. 2. Change from baseline in height Z-score yearly until FAH
3. 3. Change from baseline in height yearly until FAH

Secondary endpoints 2

1. 1. Change from baseline in height at Week 52 of Study 111-308
2. 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).

Investigational products

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

Test 3

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 14

Locations

4 EU/EEA countries · 10 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 67 files

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

Application history

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