BMN 111 for Achondroplasia
(ACH Trial)

Recruiting in Palo Alto (17 mi)

+8 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: BioMarin Pharmaceutical

No Placebo Group

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

This trial is for children with Achondroplasia who have already completed 24 months of BMN 111 treatment in a previous study. They must be able to perform study procedures, use contraception if sexually active, and not be pregnant. Those reaching legal adult age during the study will need to consent again.

Inclusion Criteria

I am willing and able to follow all study requirements.

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

I am a female over 10 or have started my periods and can take pregnancy tests during the study.

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Exclusion Criteria

Concurrent disease or condition that, in the view of the Investigator, would interfere with study participation or safety evaluations for any reason

Subject is pregnant at Baseline visit or planning to become pregnant (self or partner) at any time during the study

I am on long-term medication that is restricted.

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Treatment Details

Interventions

BMN 111 (Peptide)

Trial OverviewThe trial tests the long-term safety and effectiveness of daily injections of BMN 111 in children with Achondroplasia over an extended period after they've been treated for two years in a prior study (111-202).

Participant Groups

1Treatment groups

Experimental Treatment

Group I: BMN 111 - Subcutaneous InjectionExperimental Treatment1 Intervention

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.