This study has been transitioned to CTIS with ID 2024-512945-18-00 check the CTIS register for the current data. Objectives:Primary: To evaluate the long-term safety of IVT injected pegcetacoplanSecondary:1. To assess changes in the total area of…
ID
Source
Brief title
Condition
- Ocular structural change, deposit and degeneration NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Endpoints:
Primary:
• Incidence and severity of ocular and systemic adverse events (time frame: up
to 36 months)
Secondary outcome
Secondary:
Change from baseline in the following parameters:
• The total area of GA lesion(s) in the study eye (in mm2) as assessed by FAF
at month 12, month 24, and month 36
• The rate of change in GA lesion growth in the study eye as assessed by FAF at
month 12, month 24, and month 36
• NL-BCVA score (study eye) as assessed by Early Treatment Diabetic Retinopathy
Study (ETDRS) chart at month 12, month 24, and month
• LL-BCVA score (study eye) as assessed by ETDRS chart at month 12, month 24,
and month 36
• Monocular maximum reading speed (study eye), corrected for the number of
words read incorrectly, as assessed by Minnesota Reading (MNRead) charts or
Radner charts at month 12, month 24, and month 36
• Binocular maximum reading speed, corrected for the number of words read
incorrectly, as assessed by MNRead charts or Radner charts at month 12, month
24, and month 36
• The number of scotomatous points (study eye) assessed by mesopic
microperimetry (selected participants [those who had the assessment performed
in the antecedent study] only) at month 12, month 24, and month 36
• Macular sensitivity (study eye) as assessed by mesopic microperimetry
(selected participants [those who had the assessment performed in the
antecedent study] only) at month 12, month 24, and month 36
• Change in additional microperimetry parameters in the study eye (eg, 95%
bivariate contour ellipse area [BCEA], number of points with a clinically
significant decrease in mean sensitivity) at month 12, month 24, and month 36
• Mean Functional Reading Independence (FRI) Index score at month 12, month 24,
and month 36
• National Eye Institute Visual Functioning Questionnaire 25-Item Version (NEI
VFQ-25 and NEI VFQ-39 [at selected sites]) composite score, near activity
subscale score, distance activity subscale score, and NEI VFQ-25 driving
subscale score at month 12, month 24, and month 36
Safety:
• Presence of antibodies to the PEG and/or peptide moiety of pegcetacoplan
(time frame: up to 36 months)
• Incidence of new active choroidal neovascularization in the study eye as
assessed by FA at month 12, month 24, and month 36
• Incidence and severity of ocular and systemic adverse events (time frame: up
to 36 months) of participants being treated bilaterally with pegcetacoplan
Background summary
At this time, there is no effective treatment available for geographic atrophy
secondary to age-related macular degeneration. Patients with geographic
atrophy generally lose vision due to death of cells in the retina. The regions
of dead cells are called GA lesions. This study is being done to see if the
drug pegcetacoplan may be able to slow down growth of the GA lesion.
Study objective
This study has been transitioned to CTIS with ID 2024-512945-18-00 check the CTIS register for the current data.
Objectives:
Primary: To evaluate the long-term safety of IVT injected pegcetacoplan
Secondary:
1. To assess changes in the total area of geographic atrophy (GA) lesion in the
study eye measured by fundus autofluorescence (FAF).
2. To assess changes in visual function as measured by:
a. Normal-luminance best-corrected visual acuity score (NL-BCVA) in the study
eye
b. Low-luminance best-corrected visual acuity score (LL-BCVA) in the study eye
c. Reading speed in the study eye
3. To assess the macular functional response as assessed by mesopic
microperimetry in the study eye (selected participants [those who had the
assessment performed in the antecedent study] only).
4. To evaluate changes in participant-reported outcomes as measured by:
a. National Eye Institute Visual Functioning Questionnaire 25 Item Version (NEI
VFQ-25)
b. Functional Reading Independence (FRI) Index
Study design
Overall Study Design:
This is a phase 3, multicenter, 36-month, open-label extension study to assess
the safety and efficacy of long-term IVT injections of pegcetacoplan in
participants with GA secondary to age-related macular degeneration (AMD).
The study will enroll approximately 1200 participants across approximately 250
multinational sites who previously participated in a study evaluating
pegcetacoplan for GA. Screening can happen on the same day as the last visit in
the antecedent study or within 60 days of the last visit. Participants who meet
all inclusion and none of the exclusion criteria will be included in this
study. Participants will receive monthly pegcetacoplan treatment in the study
eye if they were in the monthly treatment group (pegcetacoplan or sham) in the
antecedent study. Participants will receive EOM pegcetacoplan treatment in the
study eye if they were in the EOM treatment group (pegcetacoplan or sham) in
the antecedent study. The study eye will be the same eye as the study eye of
the antecedent study.
Participants who previously developed exudative AMD in the study eye in the
antecedent study and are currently receiving anti-vascular endothelial growth
factor (VEGF) therapy are eligible to participate in this extension study and
will continue to receive pegcetacoplan. Participants who developed exudative
AMD in the parent study or in this extension study will remain in the study and
may also receive anti VEGF in addition to pegcetacoplan based on the
investigator assessment following standard of care.
Participants who permanently discontinue pegcetacoplan in the study eye will
also be terminated from the study. Participants who discontinue the study
should complete the early termination visit.
The fellow eyes with GA that meet certain criteria are allowed to receive
treatment with pegcetacoplan when the treating physician and participant deem
beneficial. The treatment regimen (monthly or EOM) should be decided by the
investigator.
Intervention
Pegcetacoplan, 15 mg/100 µL (monthly or every other month [EOM]), intravitreal
(IVT) injections
Participants will receive monthly pegcetacoplan treatment if they were in the
monthly treatment group (pegcetacoplan or sham) in the antecedent study.
Participants will receive EOM pegcetacoplan treatment if they were in the EOM
treatment group (pegcetacoplan or sham) in the antecedent study. The study eye
will be the same eye as the study eye of the antecedent study.
Participants who previously developed exudative AMD in the study eye in the
antecedent study and are currently receiving anti-vascular endothelial growth
factor (VEGF) therapy are eligible to participate in this extension study and
will continue to receive pegcetacoplan. Participants who developed exudative
AMD in the parent study or in this extension study will remain in the study and
may also receive anti-VEGF in addition to pegcetacoplan based on the
investigator assessment following standard of care.
Participants who permanently discontinue pegcetacoplan will also be terminated
from the study. Participants who discontinue the study should complete the
early termination visit.
Study burden and risks
Participants who previously developed exudative AMD in the study eye in the
antecedent study and are currently receiving anti-vascular endothelial growth
factor (VEGF) therapy are eligible to participate in this extension study and
will continue to receive pegcetacoplan. Participants who developed exudative
AMD in the parent study or in this extension study will remain in the study and
may also receive anti-VEGF in addition to pegcetacoplan based on the
investigator assessment following standard of care.
Since there is a possible potential health benefit for participants receiving
the study drug, this could be an altered course of GA and slower progression of
AMD, the benefits outweigh the risks associated with participating in this
study.
5th Avenue 100
Waltham MA 02451
US
5th Avenue 100
Waltham MA 02451
US
Listed location countries
Age
Inclusion criteria
Ocular-specific inclusion criteria apply to the study eye.
1. Participated in APL2-103 (NCT03777332) or completed the treatment at month
24 of either APL2-303 (Derby, NCT03525613) or APL2-304 (Oaks, NCT03525600).
Specifically, for the APL2-303 and APL2-304 studies, the following criterion
also applies:
a. Participants who did not permanently discontinue treatment but missed the
month 24 visit are also eligible to participate in this extension study;
however, to be eligible, these subjects must be screened within 60 days from
the last day of the expected month 24 visit window for the antecedent study.
2. Clarity of ocular media, adequate pupillary dilation, and fixation to permit
the collection of good quality images as determined by the investigator.
3. Female participants must be:
a. Women of nonchildbearing potential, or
b. Women of childbearing potential with a negative serum pregnancy test at
screening and must agree to use protocol defined methods of contraception for
the duration of the study and 90 days after their last dose of pegcetacoplan,
and refrain from breastfeeding for the duration of the study.
4. Males with female partners of childbearing potential must agree to use
protocol defined methods of contraception and agree to refrain from donating
sperm for the duration of the study and for 90 days after their last dose of
pegcetacoplan.
5. Willing and able to give informed consent and to comply with the study
procedures and assessments.
Fellow Eye Inclusion Criteria
The following inclusion criterion applies to the fellow eye:
2. Clarity of ocular media, adequate pupillary dilation, and fixation to
permit the collection of good quality images as determined by the investigator.
Exclusion criteria
Ocular-specific exclusion criteria apply to the study eye.
1. Participants who permanently discontinued the study drug prior to month 24
in the APL2-303 or APL2-304 studies and remained only for safety assessments.
Temporary pause of the study drug is not exclusionary.
2. Presence of an active ocular disease that, in the opinion of the
investigator, compromises or confounds visual function, including, but not
limited to, macular hole or other macular diseases (eg, clinically significant
epiretinal membrane). Benign conditions in the opinion of the investigator such
as peripheral retinal dystrophy are not exclusionary.
3. Any contraindication to IVT injection including current ocular or periocular
infection.
4. Medical or psychiatric condition that, in the opinion of the investigator,
is clinically significant and not suitable for study participation or make
consistent follow-up over the 36-month treatment period unlikely.
5. Known hypersensitivity to fluorescein sodium for injection or
hypersensitivity to pegcetacoplan or any of the excipients in pegcetacoplan
solution.
6. Pregnancy, breastfeeding, or positive pregnancy test.
Fellow Eye Exclusion Criteria
The following exclusion criteria apply to the fellow eye:
2. Presence of an active ocular disease that, in the opinion of the
investigator, compromises or confounds visual function, including, but not
limited to, macular hole or other macular diseases (eg, clinically significant
epiretinal membrane). Benign conditions in the opinion of the investigator such
as peripheral retinal dystrophy are not exclusionary.
3. Any contraindication to IVT injection including current ocular or periocular
infection.
Design
Recruitment
Medical products/devices used
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
Kamer G4-214
Postbus 22660
1100 DD Amsterdam
020 566 7389
mecamc@amsterdamumc.nl
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 |
|---|---|
| EU-CTR | CTIS2024-512945-18-00 |
| EudraCT | EUCTR2020-002931-32-NL |
| ClinicalTrials.gov | NCT04770545 |
| CCMO | NL77278.018.21 |