A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

Part of paid clinical trials in Oakland, California.

Sponsor
BioMarin Pharmaceutical
Study ID
NCT02055157
Phase
PHASE2
Status
Completed

Conditions

  • Achondroplasia

Eligibility Criteria

Sex
ALL
Age
5 Years - 14 Years
Healthy Volunteers
Not accepted

Interventions

  • BMN 111 — DRUG
    BMN 111 will be administered daily for 24 months in an open-label sequential dose adjustment fashion.

Study Details

This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with achondroplasia.

Key Dates

Start date
Jan 13, 2014
Status verified
Dec 2020
Primary completion
Oct 2, 2017
Completion
Oct 2, 2017

Study Design

Enrollment
35 participants (actual)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Cohort 1
    Cohort 1: 2.5 ug/kg
  • Experimental: Cohort 2
    Cohort 2: 7.5 ug/kg,
  • Experimental: Cohort 3
    Cohort 3: 15 ug/Kg
  • Experimental: Cohort 4
    Cohort 4: 30 ug/kg

Primary Outcome Measure

Overall Summary of Adverse Events During Initial 6-Month Period [ Time Frame: Up to Month 6 ± 7 Days ]

Locations (6)

FacilityCityStateZIPSite coordinators
Children's Hospital & Research Center OaklandOaklandCalifornia94609-
Harbor - UCLA Medical CenterTorranceCalifornia90509-
Ann and Robert H. Lurie Childrens Hospital of ChicagoChicagoIllinois60611-
Johns Hopkins McKusick - Institute of Genetic MedicineBaltimoreMaryland21287-
Vanderbilt UniversityNashvilleTennessee37232-2578-
Baylor College of MedicineHoustonTexas77030-

Find similar trials in Oakland, CA

By research site
Children's Hospital & Research Center Oakland· Oakland, CAHarbor - UCLA Medical Center· Torrance, CAAnn and Robert H. Lurie Childrens Hospital of Chicago· Chicago, ILJohns Hopkins McKusick - Institute of Genetic Medicine· Baltimore, MDVanderbilt University· Nashville, TNBaylor College of Medicine· Houston, TX

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