Research Studies

Study Title

Biogen SOD1 ASO “VALOR” Trial (An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 in Adults with Inherited ALS)

Enrollment Status

Completed

Principal Investigators

Site-PI: Michael Benatar, MD, PhD

Study Type

Drug Trial

Goals

  • Determine the safety and efficacy of the experimental antisense oligonucleotide (ASO) BIIB067 in patients with SOD1-ALS

Enrolling

SOD1 ALS patients

Eligibility

Click to Expand

Individuals affected with ALS, and positive for an SOD1 gene mutation that is associated with rapidly progressive disease

Study Involvement

Click to Expand

Multiple study visits with spinal tap and intrathecal administration of experimental agent. “Loading” comprises 3 administrations, with each dose separated by 2 weeks. “Maintenance” comprises 5 doses, administered every 4 weeks.
Study procedures include:

  • Medical history and medication review
  • Drug dosing
  • Blood, urine and CSF collection
  • Neurological exam
  • Muscle testing
  • Electrocardiogram
  • Physical Exam
  • Surveys
  • Breathing testing
  • Biomarker procedures (EIM, HHD, MUNIX)

Monthly intrathecal administration (following a blinded induction phase) of BIIB067 during the open label extension phase.

Primary Funding Source

Biogen

Related Publications

Click to Expand

No items found

Related Presentations & Lectures

Click to Expand

No items found