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

Active-Not Recruiting

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

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

Study Involvement

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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

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Related Presentations & Lectures

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