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.
Study Status:
Not recruiting
Disease:
Familial ALS
Study Type:
Interventional
Type of Intervention:
Drug Trial, Gene Therapy
Intervention Name:
Placebo:
No
Phase:
Phase 1
Study Chair(s)/Principal Investigator(s):
Robert Brown, D. Phil, M.D., UMass James Berry, M.D., MGH
Clinicaltrials.gov ID:
Neals Affiliated?
Yes
Coordinating Center Contact Information
Massachusetts General Hospital
Lindsay Pothier / email hidden; JavaScript is required / 617-643-5582
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:
04 / 18 / 2018
Date Study Added to neals.org:
09 / 18 / 2017
Massachusetts General Hospital
Boston, Massachusetts
United States
University of Massachusetts
Worcester, Massachusetts
United States