Neovascular Age-related Macular DegenerationPhase 1/2NCT03465709
Pegcetacoplan (APL-2) in Neovascular AMD
A Phase 1/2 study testing Pegcetacoplan in people with Neovascular Age-related Macular Degeneration, run by Apellis Pharmaceuticals, Inc. at 4 sites in United States.
Trial summary
Safety Assessment of Pegcetacoplan in Patients with Neovascular AMD Read more about this study
- Who is it for?
- Ages 60 years and older
- Conditions the study is for
- Neovascular Age-related Macular Degeneration
- What is being tested?
- Pegcetacoplan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 4 sitesUnited States
- Study dates
- 14 Feb 2018to 5 Apr 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 60 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age greater than or equal to 60 years.
- Normal Luminance best corrected visual acuity (NL-BCVA) of 24 letters or better using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (20/320 Snellen equivalent).
- Clinical diagnosis of neovascular AMD with the following criteria met:
- Eligible for an injection of an anti-VEGF injection with macular fluid present at Day -28.
- Must have been treated with anti-VEGF in study eye for at least 6 months prior to joining the study.
- At least 6 months of intravitreal anti-VEGF therapy at intervals not greater than 8 weeks (± 7 days) for the past 2 injections in the eye that is selected to be the study eye.
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- A clinically meaningful (50%) reduction in excess macular fluid or macular thickness in the study eye at the discretion of the investigator between Screening Day -28 and Screening Day -14 as assessed by SD-OCT.
- Female subjects must be:
- Women of non-child-bearing potential (WONCBP), or
- Women of child-bearing potential (WOCBP) with a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study and refrain from breastfeeding for the duration of the study.
- Males with female partners of child-bearing potential must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study.
- Willing and able to give informed consent and to comply with the study procedures and assessments
Who cannot join
- Presence of other causes of choroidal neovascularization (CNV) including pathologic myopia (spherical equivalent ≥ -6 diopters), central serous chorioretinopathy, ocular histoplasmosis syndrome, angioid streaks, choroidal rupture, and multifocal choroiditis.
- History of vitrectomy to the study eye
- Presence of any ophthalmologic condition that reduces the clarity of the media and that, in the opinion of the Investigator, interferes with ophthalmologic examination (e.g. advanced cataract or corneal abnormalities).
- Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization.
- Any history of endophthalmitis.
- Trabeculectomy or aqueous shunt or valve in the study eye.
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- Aphakia or absence of the posterior capsule. Note: previous violation of the posterior capsule is also excluded unless it occurred as a result of yttrium aluminum garnet (YAG) laser posterior capsulotomy in association with prior posterior chamber intraocular lens implantation and at least 60 days prior to baseline.
- Any ophthalmic condition that may require surgery or medical intervention during the study period or, in the opinion of the Investigator, could compromise visual function during the study period (e.g. severe uncontrolled glaucoma, clinically significant diabetic macular edema, ischemic optic neuropathy, retinal vasculopathies).
- Any contraindication to IVT injection including current ocular or periocular infection.
- Current treatment for active systemic or localized infection.
- Participation in any systemic experimental treatment or any other systemic investigational new drug within 6 weeks or 5 half-lives of the active (whichever is longer) prior to the start of study treatment. Note: clinical trials solely involving observation, over-the-counter vitamins, supplements, or diets are not exclusionary
- Medical or psychiatric conditions that, in the opinion of the investigator, make consistent follow-up over the 24- month treatment period unlikely, or would make the subject an unsafe study candidate.
- Any baseline laboratory value (hematology, serum chemistry or urinalysis) that in the opinion of the Investigator is clinically significant and not suitable for study participation.
- Known hypersensitivity to fluorescein sodium for injection or hypersensitivity to pegcetacoplan or any of the excipients in pegcetacoplan solution
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Apellis Clinical Site
Beverly Hills, California, 90211
Apellis Clinical Site
Cleveland, Ohio, 44122
Apellis Clinical Site
Hagerstown, Maryland, 21740
Apellis Clinical Site
Houston, Texas, 77030
About this study
Official title: An 18-Month Phase Ib/II Multi-Center, Open Label Study to Evaluate the Safety of Intravitreal APL-2 Therapy in Patients With Neovascular Age-Related Macular Degeneration (AMD)
Safety Assessment of Pegcetacoplan in Patients with Neovascular AMD
- Being tested
- Pegcetacoplan
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PegcetacoplanDrug
Also called APL-2
Study Drug
| Study group | Role | What they receive |
|---|---|---|
| Pegcetacoplan Study Drug | Experimental | Not described Receives: Drug: Pegcetacoplan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects Experiencing Ocular and Systemic Treatment-Emergent Adverse Events (TEAEs) Including by Maximum Severity
TEAEs were defined as those adverse events (AEs) that developed or worsened after the first dose of study drug, up to 30 days after the last dose. The severity of the TEAEs was classified as mild (asymptomatic/mild symptoms); moderate (minimal, local/non-invasive intervention indicated); severe (medically significant but not life-threatening); life-threatening or leading to death. The number of subjects experiencing 1 TEAE in each category is presented. A maximum of 7 injections of pegcetacoplan (per subject) and a maximum of 13 injections of anti-VEGF (per subject) were received during the study.
Measured: Day 1 up to end of study (up to 1 year).
Secondary outcomes (2)
Mean Change From Baseline in SD-OCT Central Subfield Thickness (CST) up to 12 Months
The CST was measured using SD-OCT throughout the study and the mean change from baseline at each timepoint is presented for the study eye.
Measured: Day 1 up to Day 360 (12 months).
Number of Subjects With Clinically Significant Change From Baseline in Physical Examination Findings, Vital Signs and Laboratory Parameters
Physical examinations, vital signs and laboratory parameters were monitored throughout the study and any subjects showing a clinically significant change from baseline over the study period are presented.
Measured: Baseline (Screening or Day 1) up to end of study (up to 1 year).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2018
- Study start14 Feb 2018
- Primary completion5 Apr 2019
- Study completion5 Apr 2019
Why it stopped: Apellis concluded that sufficient data were collected to meet the study objectives.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Neovascular Age-related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Apellis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Federico Grossi, MD, PhD, Study director, Apellis Pharmaceuticals, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03465709
- Sponsor's protocol ID
- APL2-203
- Status last verified
- Sep 2020
- First submitted
- 8 Mar 2018
- Last updated
- 16 Sep 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An 18-Month Phase Ib/II Multi-Center, Open Label Study to Evaluate the Safety of Intravitreal APL-2 Therapy in Patients With Neovascular Age-Related Macular Degeneration (AMD)
- ClinicalTrials.gov ID
- NCT03465709
- Acronym / sponsor ID
- APL2-203
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Apellis concluded that sufficient data were collected to meet the study objectives.
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Apellis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Feb 2018 (actual)
- Primary completion
- 5 Apr 2019 (actual)
- Study completion
- 5 Apr 2019 (actual)
- First posted
- 14 Mar 2018 (actual)
- Results
- Posted 16 Sep 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Safety Assessment of Pegcetacoplan in Patients with Neovascular AMD
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pegcetacoplan Study Drug | Experimental | Drug: Pegcetacoplan |
Who may qualify
- Ages
- 60 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Age greater than or equal to 60 years.
- Normal Luminance best corrected visual acuity (NL-BCVA) of 24 letters or better using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (20/320 Snellen equivalent).
- Clinical diagnosis of neovascular AMD with the following criteria met:
- A clinically meaningful (50%) reduction in excess macular fluid or macular thickness in the study eye at the discretion of the investigator between Screening Day -28 and Screening Day -14 as assessed by SD-OCT.
- Female subjects must be:
Key exclusion criteria (14 listed)
- Presence of other causes of choroidal neovascularization (CNV) including pathologic myopia (spherical equivalent ≥ -6 diopters), central serous chorioretinopathy, ocular histoplasmosis syndrome, angioid streaks, choroidal rupture, and multifocal choroiditis.
- History of vitrectomy to the study eye
- Presence of any ophthalmologic condition that reduces the clarity of the media and that, in the opinion of the Investigator, interferes with ophthalmologic examination (e.g. advanced cataract or corneal abnormalities).
- Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization.
- Any history of endophthalmitis.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PegcetacoplanAlso: APL-2Study Drug | Drug | Pegcetacoplan Study Drug |
Primary outcome
Number of Subjects Experiencing Ocular and Systemic Treatment-Emergent Adverse Events (TEAEs) Including by Maximum Severity
Time frame: Day 1 up to end of study (up to 1 year).
TEAEs were defined as those adverse events (AEs) that developed or worsened after the first dose of study drug, up to 30 days after the last dose. The severity of the TEAEs was classified as mild (asymptomatic/mild symptoms); moderate (minimal, local/non-invasive intervention indicated); severe (medically significant but not life-threatening); life-threatening or leading to death. The number of subjects experiencing 1 TEAE in each category is presented. A maximum of 7 injections of pegcetacoplan (per subject) and a maximum of 13 injections of anti-VEGF (per subject) were received during the study.
2 secondary outcomes
Mean Change From Baseline in SD-OCT Central Subfield Thickness (CST) up to 12 Months
Time frame: Day 1 up to Day 360 (12 months).
Number of Subjects With Clinically Significant Change From Baseline in Physical Examination Findings, Vital Signs and Laboratory Parameters
Time frame: Baseline (Screening or Day 1) up to end of study (up to 1 year).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Sep 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Federico Grossi, MD, PhD study director
Apellis Pharmaceuticals, Inc.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Pegcetacoplan in people with Neovascular Age-related Macular Degeneration, run by Apellis Pharmaceuticals, Inc. at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 16 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 16 Sep 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Subjects Experiencing Ocular and Systemic Treatment-Emergent Adverse Events (TEAEs) Including by Maximum Severity (time frame: Day 1 up to end of study (up to 1 year).). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2020.