A study of BMN 333 versus Vosoritide in Children with Achondroplasia

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]

Decision date (initial)

2026-05-05

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

Phase 2: To evaluate the effect of 3 doses of BMN 333 versus vosoritide XXX on AGV (interim analysis)
Phase 3: To evaluate the effect of BMN 333 at a selected dose versus vosoritide on AGV after 52 weeks of treatment.

Secondary objectives 2

1. Phase 2: 1.To evaluate the effect of 3 doses of BMN 333 versus vosoritide on AGV 2.To evaluate the effect of 3 doses of BMN 333 versus vosoritide on growth parameters 3.To evaluate the safety and tolerability of 3 doses of BMN 333
2. Phase 3: 1. To evaluate the effect of BMN 333 versus vosoritide on additional growth parameters after 52 weeks of treatment

Conditions and MedDRA coding

Achondroplasia

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration, Pharmaceuticals And Medical Devices Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. 1.Participants must be aged ≥ 2 to < 11 years (Phase 2) or ≥ 2 to < 18 years (Phase 3), at the time of signing the informed consent.
2. 2.Participants must have ACH (confirmed by documented genetic testing) and open epiphyses assessed using left hand postero-anterior (PA) X-rays, by the Greulich and Pyle method (Greulich 1971) as per standard of care.
3. 3.Females ≥ 10 years old or who have begun menses must have a negative pregnancy test at the Screening visit and be willing to have additional pregnancy tests during the study.
4. 4.Are Tanner Stage I (Phase 2) or any Tanner stage (Phase 3)
5. 5.Have a body weight ≥ 8.0 kg at the Screening visit
6. 6.Are ambulatory and able to stand without assistance
7. 7.Caregivers are willing to administer injections to the participants and willing to complete the required training.

Exclusion criteria 3

1. 1.Have any short stature condition other than ACH (eg, hypochondroplasia, trisomy 21, pseudoachondroplasia, GH deficiency).
2. 2.Have any of the following disorders:Hypothyroidism or hyperthyroidism (unless treated) ,Diabetes mellitus (unless considered well-controlled), Autoimmune inflammatory disease, Autoimmune inflammatory disease ,Autonomic neuropathy.
3. 3.Have history of: Renal insufficiency, Cardiac or vascular disease, unstable clinical condition , significant hip pathology.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Phase 2: Predicted AGV at Week 52 (based on AGV at Weeks 26, 39, and 52 [available cumulative data])
2. Phase 3: AGV at Week 52

Secondary endpoints 2

1. Phase 2: 1.AGV at Weeks 26 and 52 2.Change from Baseline in standing height, height Z-score and in upper to lower body segment ratio at Weeks 26 and 52 3.Incidence of AEs, SAEs, and EOIsa; physical examinations; vital signs; ECG findings; imaging results (X-ray and DXA); clinical laboratory tests.
2. Phase 3: • Change from Baseline in standing height, height Z-score, upper to lower body segment ratio at Week 52

Investigational products

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

Test 2

Comparator 2

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 18

Locations

3 EU/EEA countries · 7 investigational sites

By country

Investigational sites

Results and documents

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

Documents 59 files

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

Application history

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