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

Study Purpose:

The purpose of this study is to provide compassionate use of virally delivered miRNA against SOD1 to two subjects with ALS caused by mutations in the SOD1 gene. 

Disease:

Familial ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial, Gene Therapy

Study Status:

Active, currently recruiting

Phase:

Phase I

Study Chair(s)/Principal Investigator(s):

Robert Brown, D. Phil, M.D., UMMC

James Berry, M.D., MGH

Clinicaltrials.gov ID (11 digit #):

Pending

Neals Affiliated?

Yes

Coordinating Center Contact Information


Lindsay Pothier / .(JavaScript must be enabled to view this email address) / 617-643-5582
.(JavaScript must be enabled to view this email address) Boston, Massachusetts United States

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.

Study Sponsor:

Robert H. Brown Jr., D. Phil, M.D.

Participant Duration:

The study volunteers will be followed at weekly (+/- 2 days) intervals for one month post infusion and then monthly (28 days +/- 5 days) until one year post-infusion. Thereafter visits will be every three months (84 days +/- 7 days) up to 24 months post infusion, then every 6 months (168 days +/- 14 days) thereafter indefinitely.

Estimated Enrollment:

2

Estimated Study Start Date:

11/01/2016

Estimated Study Completion Date:

11/01/2020

Posting Last Modified Date:

09/18/2017

Date Study Added to alsconsortium.org:

09/18/2017