Summary

for people ages 18 years and up (full criteria)
at Orange, California and other locations
study started
estimated completion
Namita Goyal

Description

Summary

A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)

Official Title

A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotropic Lateral Sclerosis

Details

Screening of up to 4 weeks Treatment of up to 76 weeks

Keywords

Amyotrophic Lateral SclerosisarimoclomolALSSclerosisMotor Neuron Disease

Eligibility

You can join if…

Open to people ages 18 years and up

  • Subject meets revised El Escorial criteria for clinically possible, clinically probable / Clinically probable ALS laboratory-supported or clinically definite ALS, or familial ALS
  • 18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath).
  • ALSFRS-R equal to or above 35 and erect (seated) SVC% predicted equal to or above 80% at screening

You CAN'T join if...

  • Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of screening or baseline
  • pregnant or breast-feeding
  • current or anticipated use of diaphragmatic pacing
  • Any other relevant medically significant condition which could present risk to the subject or interfere with the assessment of safety or has an increased risk of causing death during the trial

Locations

  • UC Irvine Health ALS and Neuromuscular Centeraccepting new patients
    OrangeCalifornia92868United States
  • St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Centeraccepting new patients
    PhoenixArizona85013United States

Lead Scientist

  • Namita Goyal
    Authored (or co-authored) 20 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Orphazyme
ID
NCT03491462
Phase
Phase 3
Study Type
Interventional
Last Updated