This study is accepting new patients by invitation only

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

a study on Achondroplasia

Summary

for people ages up to 59 months (full criteria)
at Oakland, California and other locations
study started
estimated completion

Description

Summary

Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia.

Official Title

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia, Age 0 to < 60 Months

Keywords

AchondroplasiaDwarfismBone DiseasesBone Diseases, DevelopmentalACHNatriuretic Peptide, C-TypeMusculoskeletal DiseasesNatriuretic AgentsPhysiological Effect of DrugsSkeletal DysplasiasGenetic Diseases, InbornOsteochondrodysplasiasBMN 111

Eligibility

You can join if…

Open to people ages up to 59 months

  • Diagnosis of ACH, confirmed by genetic testing
  • Age 0 to < 60 months at study entry (Day 1)
  • At least 6-month period of pretreatment growth assessment in Study 111-901 immediately before study entry (cohort 1 & 2) or at least 3 months of observation prior to treatment (cohort 3)

You CAN'T join if...

  • Subject weight < 5.0 kg (cohort 1 & 2) or < 4.0 kg (cohort 3)
  • Treatment with growth hormone within 6-months prior to screening or prolonged treatment (> 3 months) at any time
  • Any history of spine or long-bone surgery or any bone-related surgery with chronic complications
  • Any history of limb-lengthening surgery or planned limb-lengthening during the study
  • Fracture of the long bones within 6 months prior to screening

Locations

  • Children's Hospital & Research Center Oakland
    OaklandCalifornia94609United States
  • Harbor - UCLA Medical Center
    TorranceCalifornia90509United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
Links
NIH Genetics Home Reference related topics: Achondroplasia
Description NIH Genetic and Rare Diseases Information Center resources: Achondroplasia
ID
NCT03583697
Phase
Phase 2
Study Type
Interventional
Last Updated