Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)



Status:Active, not recruiting
Healthy:No
Age Range:6 - Any
Updated:3/22/2019
Start Date:May 2015
End Date:October 2023

Use our guide to learn which trials are right for you!

A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Retinoschisin (rAAV2tYF-CB-hRS1) in Patients With X-linked Retinoschisis

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus
vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with X-linked retinoschisis.
Up to 27 participants will be enrolled and 3 dose levels will be evaluated in a dose
escalation format.

This will be a non-randomized, open label, Phase 1/2 dose escalation study.

Up to 27 participants will be enrolled. Each participant will receive the study agent by
intravitreal injection in one eye on a single occasion. Enrollment will begin with the lowest
dose and will proceed to higher doses only after review of safety data by a Data and Safety
Monitoring Committee (DSMC). Participants in the dose escalation phase will be ≥ 18 years of
age. After the maximum tolerated dose is identified individuals ≥ 6 years of age will be
enrolled.

Safety will be measured by the number and proportion of participants experiencing adverse
events and immune response to RS1. Efficacy will be measured by evaluation of changes in
visual function and schisis cavity size.

Inclusion Criteria include:

1. Retinal disease consistent with a diagnosis of XLRS and documented mutations in the
RS1 gene

2. Male individual at least 18 years of age (dose escalation phase) or at least 6 years
of age (maximum tolerated dose phase),

3. Able to perform tests of visual and retinal function,

4. Visual acuity specified for each group

5. Not treated with CAIs currently or within 3 months prior to study enrollment,

6. Have acceptable laboratory parameters.

Exclusion Criteria include:

1. Prior receipt of any AAV gene therapy product,

2. Pre-existing eye conditions that would preclude the planned intravitreal injection or
interfere with interpretation of study endpoints or complications of vector
administration.
We found this trial at
9
sites
Ann Arbor, Michigan 48105
Principal Investigator: John Heckenlively, MD
Phone: 734-763-2280
?
mi
from
Ann Arbor, MI
Click here to add this to my saved trials
Baltimore, Maryland 21287
Principal Investigator: Neil Bressler, MD
Phone: 410-955-5818
?
mi
from
Baltimore, MD
Click here to add this to my saved trials
243 Charles St
Boston, Massachusetts 02114
(617) 523-7900
Principal Investigator: Jason Comander, MD, PhD
Phone: 617-573-3871
Massachusetts Eye & Ear Infirmary Whether you see our physicians at Mass. Eye and Ear's...
?
mi
from
Boston, MA
Click here to add this to my saved trials
Dallas, Texas 75231
Principal Investigator: David Birch, PhD
Phone: 214-363-3911
?
mi
from
Dallas, TX
Click here to add this to my saved trials
2351 Erwin Road
Durham, North Carolina 27710
?
mi
from
Durham, NC
Click here to add this to my saved trials
Houston, Texas 77030
Principal Investigator: Christina Weng, MD
Phone: 713-798-8419
?
mi
from
Houston, TX
Click here to add this to my saved trials
?
mi
from
Miami, FL
Click here to add this to my saved trials
Portland, Oregon 97239
Principal Investigator: Mark Pennesi, MD, PhD
Phone: 503-494-0020
?
mi
from
Portland, OR
Click here to add this to my saved trials
San Francisco, California 94143
Principal Investigator: Anthony Moore, MD
Phone: 415-476-3614
?
mi
from
San Francisco, CA
Click here to add this to my saved trials