A Long-term Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia

Phase 1

Conditions: Achondroplasia
MedDRA version: 25.0Level: LLTClassification code: 10000452Term: Achondroplasia Class: 10010331
Therapeutic area: Phenomena and Processes [G] - Musculoskeletal and Neural Physiological Phenomena [G11]

Registration Number: CTIS2023-508754-26-00

Lead Sponsor: Biomarin Pharmaceutical Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 29

Inclusion Criteria

Must have completed 24 months (± 14 days) of BMN 111 treatment in Study 111-202., Parent(s) or guardian(s) are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to performance of any researchrelated procedure. Also, subjects under the age of 18 are willing and able to provide written assent (if required by local regulations or the IRB/EC) after the nature of the study has been explained and prior to performance of any researchrelated procedure., If sexually active, is willing to use a highly effective method of contraception while participating in the study, 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, Are willing and able to perform all study procedures as physically possible, Caregivers are willing to administer daily injections to the subjects and complete the required training – not applicable in France

Exclusion Criteria

Requires any investigational agent prior to completion of study period, Have a condition or circumstance that, in the view of the Investigator, places the subject at high risk for poor treatment compliance or for not completing the study, Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason, Permanently discontinued BMN 111 during the 111-202 study, Subject is pregnant at the Baseline visit or planning to become pregnant (self or partner) at any time during the study, Current chronic therapy with any of the following restricted medications: • Antihypertensive medications • Angiotensin-converting enzyme (ACE) inhibitors • Angiotensin II receptor blockers • Diuretics • Beta-blockers • Calcium-channel blockers • Cardiac glycosides • Systemic anticholinergic agents • Any medication that may impair or enhance compensatory tachycardia, diuretics, or other drugs known to alter renal or tubular function