A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)

BioMarin Pharmaceutical

Status and phase: Active, not recruiting; Phase 2

Conditions: Achondroplasia

Treatments: Drug: BMN 111

Study type: Interventional

Funder types: Industry

Identifiers

NCT02724228

2015-004004-30 (EudraCT Number)

111-205

Details and patient eligibility

About

This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia. The primary objective is to evaluate the long-term safety and tolerability of daily SC injections of BMN 111 in children with ACH who have completed two years of treatment in the 111-202 study.

Enrollment: 30 patients

Sex: All

Ages: 7+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Have completed 24 months of BMN 111 treatment in Study 111-202.
• Parent(s) or guardian(s) are willing and able to provide written, signed informed consent. Subjects under the age of majority are willing and able to provide written assent (if required). Subjects who reach the age of majority in their country will be asked to provide their own written consent upon reaching the legal age of majority.
• If sexually active, willing to use a highly effective method of contraception while participating in the study.
• Females >= 10 years old or who have started menses must have a negative pregnancy test at baseline and be willing to have additional pregnancy tests during the study
• Willing and able to perform all study procedures as physically possible
• Parents/caregivers willing to administer daily injections to the subjects and complete the required training.

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 Baseline visit or planning to become pregnant (self or partner) at any time during the study.
• Current chronic therapy with restricted medications.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

BMN 111 - Subcutaneous Injection

Experimental group

Description:

111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen.

Treatment:

Drug: BMN 111