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Clinical Trials/EUCTR2017-002404-28-DE
EUCTR2017-002404-28-DEActive, not recruitingPhase 1

A Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia

BioMarin Pharmaceutical Inc.0 sites119 target enrollmentStarted: December 13, 2018Last updated:
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Conditions
Drugs

Trial Snapshot

Phase
Phase 1
Status
Active, not recruiting
Enrollment
119
Study OverviewEligibility CriteriaInvestigatorsRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

No summary available.

Study Design

Study Type
Interventional clinical trial of medicinal product

Eligibility Criteria

Sex
All

Inclusion Criteria

  • •1. Must have completed Study 111-301
  • •2. 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 research-related procedure. Also, subjects under the age of majority are willing and able to provide written assent (if required by local regulations or the IRB/IEC) after the nature of the study has been explained and prior to performance of any research-related procedure. Subjects who reach the age of majority in their country while the study is ongoing will be asked to provide their own written consent again upon reaching the legal age of majority.
  • •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. If sexually active, are willing to use contraception as specified in section 9.3.3 of the protocol,
  • •5. Are willing and able to perform all study procedures
  • •Are the trial subjects under 18? yes
  • •Number of subjects for this age range: 119
  • •F.1.2 Adults (18-64 years) no
  • •F.1.2.1 Number of subjects for this age range
  • •F.1.3 Elderly (>=65 years) no
  • •F.1.3.1 Number of subjects for this age range

Exclusion Criteria

  • •1. Permanently discontinued BMN 111 or placebo prior to completion of Study 111-301
  • •2. Have a clinically significant finding or arrhythmia on Baseline electrocardiogram (ECG) that indicates abnormal cardiac function or conduction or QTc-F > 450 msec
  • •3. Evidence of decreased growth velocity (AGV < 1.5 cm/year) as assessed over a period of at least 6 months or of growth plate closure (proximal tibia, distal femur) through bilateral lower extremity X-rays including both AP and lateral views
  • •4. Require any investigational agent prior to completion of study period
  • •5. Current therapy with antihypertensive medications, angiotensin-
  • •converting enzyme (ACE) inhibitors, angiotensin II receptor blockers,
  • •diuretics, beta-blockers, calcium-channel blockers, cardiac glycosides,
  • •systemic anticholinergic agents, GnRH agonists, any medication that
  • •may impair or enhance compensatory tachycardia, diuretics, or other
  • •drugs known to alter renal or tubular function (Table 9.3.2.1)
  • •6. Planned or expected to have limb-lengthening surgery during the
  • •study period
  • •7. Pregnant or breastfeeding at the Baseline Visit or planning to become
  • •pregnant (self or partner) at any time during the study
  • •8. Concurrent disease or condition that, in the view of the investigator,
  • •would interfere with study participation or safety evaluations, for any
  • •9. 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

Investigators

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