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

Achondroplasia Dwarfism Bone Diseases Bone Diseases, Developmental ACH Natriuretic Peptide, C-Type Musculoskeletal Diseases Natriuretic Agents Physiological Effect of Drugs Skeletal Dysplasias Genetic Diseases, Inborn Osteochondrodysplasias BMN 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...

  1. Have hypochondroplasia or short-stature condition other than achondroplasia (e.g., trisomy 21, pseudoachondroplasia, etc.)
  2. Have any of the following:
  3. Hypothyroidism or hyperthyroidism
  4. Insulin-requiring diabetes mellitus
  5. Autoimmune inflammatory disease (including celiac disease, systemic lupus erythematosus, juvenile dermatomyositis, scleroderma, etc.)
  6. Inflammatory bowel disease
  7. Autonomic neuropathy
  8. Have a clinically significant finding or arrhythmia that indicates abnormal cardiac function or conduction or QTc-F > 450 msec on screening ECG
  9. Have evidence of cervicomedullary compression (CMC) likely to require surgical intervention within 60 days of Screening as determined by the Investigator and informed by the following assessments:
  10. Physical exam (eg, neurologic findings of clonus, opisthotonus, exaggerated reflexes, dilated facial veins)
  11. Polysomnography (eg, severe central sleep apnea)
  12. MRI indicating presence of severe CMC or spinal cord damage
  13. Subject weight < 5.0 kg (cohort 1 & 2) or < 4.0 kg (cohort 3)
  14. Treatment with growth hormone within 6-months prior to screening or prolonged treatment (> 3 months) at any time
  15. Any history of spine or long-bone surgery or any bone-related surgery with chronic complications
  16. Any history of limb-lengthening surgery or planned limb-lengthening during the study
  17. Fracture of the long bones within 6 months prior to screening

Locations

  • Children's Hospital & Research Center Oakland
    Oakland California 94609 United States
  • Harbor - UCLA Medical Center
    Torrance California 90509 United 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