Expanded Access Use of microRNA Delivered Intrathecally via Adeno-Associated Virus rh10 as Therapy for Mutant SOD1 Mediated Amyotrophic Lateral Sclerosis (ALS)

Full Study Summary:

This is an open-label, investigational protocol for intrathecal administration of anti-SOD1 miRNA (amiR- SOD1) as an emergency therapeutic in two protocol defined subjects with the SOD1 gene mutations. The intention is to deliver drug into the intrathecal space using adeno- associated virus rhesus strain 10 (AAVrh10). The primary outcome for our study will be clinical status of the patients as assessed by the ALSFRS-R. The safety of anti-SOD1 RNAi will be evaluated using vital signs, weight, clinical laboratory determinations, physical and neurological examinations, ECG’s, AEs, (including death and other SAEs), and use of concomitant medications.

• Eligibility Criteria

  Gender:

  Female, Male

  Minimum Age:

  N/A

  Maximum Age:

  N/A

  Time since Symptom Onset:

  Time since Diagnosis:

  Can participants use Riluzole?

  
  

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