The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of:• AAV2-REP1 in subjects with CHM who have been previously treated with AAV2-REP1 and who have exited an antecedent study; these treated…
ID
Source
Brief title
Condition
- Retina, choroid and vitreous haemorrhages and vascular disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary endpoint of this study is safety of AAV2-REP1 and AAV8-RPGR, which
will be evaluated through adverse event (AE) reporting and full ophthalmic
examinations.
Secondary outcome
• Change from Baseline in best-corrected visual acuity (BCVA) as measured by
the Early
Treatment of Diabetic Retinopathy Study (ETDRS) chart
• Proportion of participants with no decrease from Baseline in BCVA or a
decrease from
Baseline in BCVA of < 5 ETDRS letters (in CHM participants only)
• Proportion of participants with an increase from Baseline in BCVA of >= 10
ETDRS
letters (in CHM participants only)
• Proportion of participants with an increase from Baseline in BCVA of >= 15
ETDRS
letters (in CHM participants only)
• Available assessments of fundus autofluorescence at each visit
• Available assessments of fundus photography at each visit
• Available assessments of spectral-domain optical coherence tomography
(SD-OCT) at
each visit
• Available assessments of microperimetry at each visit
• Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25)
• Change from Baseline in visual field (in AAV8-RPGR-treated participants only)
• Proportion of participants with an increase from Baseline in low-luminance
visual acuity
(LLVA) of >= 10 ETDRS letters (in AAV8-RPGR-treated participants only)
• Proportion of participants with an increase from Baseline in LLVA of >= 15
ETDRS letters
(in AAV8-RPGR-treated participants only)
Datasets from the 2 disease populations will be analyzed separately.
Background summary
Choroideremia (CHM) is a rare, untreatable retinal degeneration that begins in
childhood with loss of night vision and gradually progresses to blindness by
middle age. X-linked retinitis pigmentosa (XLRP) is a degenerative X-linked,
rare disease, primarily of males. The disease presents with the onset of night
blindness by the tenth year, with progressive loss of peripheral vision leading
to legal or total blindness from approximately
40 years of age.
Currently there are no approved treatments for CHM or XLRP.
AAV2-REP1 is being developed as a potential gene therapy for the treatment of
CHM. In addition, AAV8-RPGR is being developed as a potential gene therapy for
the treatment of XLRP
The aim of the present study is to evaluate the long-term safety and efficacy
of AAV2-REP1 and AAV8-RPGR up to 5 years post-treatment.
Study objective
The objective of the study is to evaluate the long-term safety and efficacy of
a sub-retinal injection of:
• AAV2-REP1 in subjects with CHM who have been previously treated with
AAV2-REP1 and who have exited an antecedent study; these treated subjects will
be compared with untreated control subjects who have exited the STAR study.
• AAV8-RPGR in subjects with X-linked retinitis pigmentosa (XLRP) who have been
previously treated with AAV8-RPGR and who have exited an antecedent study.
Study design
This is a multi-center, interventional, follow-up study for participants who
have previously received a sub-retinal injection of AAV2-REP1 for the treatment
of CHM or a sub-retinal injection of AAV8-RPGR for the treatment of XLRP. To
allow for more meaningful comparisons of safety and efficacy when investigating
treatment of AAV2-REP1, untreated subjects who served as controls in the
antecedent STAR study (NSR-REP-01), will also be enrolled in SOLSTICE.
At study entry, the number of months from treatment (or Day 0 in the antecedent
STAR study, in the case of control participants) will be defined.
For any timepoints that have been missed following completion of the antecedent
study and prior to entry into SOLSTICE, data will be recorded retrospectively
from the subject*s medical records under the closest visit within 6 months.
For participants who received treatment in an antecedent study, this study will
consist of up to 9 visits over a maximum 48-month study period, to provide 60
months of post-treatment follow up for each treated subject, including time
spent in the prior study.
For untreated control subjects from the antecedent STAR study, this study will
consist of up to 5 visits over a maximum 24-month study period, to provide 36
months of follow-up, including time spent in the prior study.
Study burden and risks
This long-term follow-up study only collects information and has no further
treatment. Only standard eye exams will be done. Therefore, participation in
this study has almost no additional risks associated.
70 Norden Road 70
Maidenhead SL6 4AY
GB
70 Norden Road 70
Maidenhead SL6 4AY
GB
Listed location countries
Age
Inclusion criteria
Inclusion Criteria - Participants are eligible for study participation if they
meet all of the following inclusion criteria:
CHM Participants:
a. Are willing and able to give informed consent for participation in the study
b. Have participated in and exited from an interventional study that
investigated the safety and efficacy of a sub-retinal injection of AAV2-REP1
for CHM
XLRP Participants:
a. Are willing and able to give informed consent for participation in the study
b. Have received a sub-retinal injection of AAV8-RPGR for XLRP and have exited
an antecedent study
Exclusion criteria
Participants are not eligible for study participation if they meet the
following exclusion criterion.
a. In the opinion of the investigator and/or the Sponsor, it is not in the
participant's best interest to participate in the study.
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2017-003104-42-NL |
| ClinicalTrials.gov | NCT03584165 |
| CCMO | NL69445.000.21 |