Pegcetacoplan (APL-2) in Neovascular AMD

NCT03465709 | Terminated Phase 1 Results DMC FDA Drug

• 1 other identifier: APL2-203
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 4

Brief Summary

Safety Assessment of Pegcetacoplan in Patients with Neovascular AMD

Conditions

Neovascular Age-related Macular Degeneration

Outcome Measures

Primary Outcomes (1)

• 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.

  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

  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

  Baseline (Screening or Day 1) up to end of study (up to 1 year).

Study Arms (1)

Pegcetacoplan Study Drug

EXPERIMENTAL

Drug: Pegcetacoplan

Interventions

Pegcetacoplan DRUG

Study Drug

Also known as: APL-2

Pegcetacoplan Study Drug

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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

You may not qualify if:

• 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.
• 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.
• 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

Sponsors & Collaborators

• Apellis Pharmaceuticals, Inc.: lead

Study Sites (4)

Apellis Clinical Site

Beverly Hills, California, 90211, United States

Location

Apellis Clinical Site

Hagerstown, Maryland, 21740, United States

Location

Apellis Clinical Site

Cleveland, Ohio, 44122, United States

Location

Apellis Clinical Site

Houston, Texas, 77030, United States

Location

MeSH Terms

Interventions

pegcetacoplan

Limitations and Caveats

Exposure was shorter than expected as some subjects experienced intraocular inflammation in the study eye due to an identified impurity. The sponsor terminated the study early as sufficient data were collected to meet study objectives.

Results Point of Contact

• Title: Apellis Clinical Trial Information Line
• Organization: Apellis Pharmaceuticals. Inc.

clinicaltrials@apellis.com

1-833-284-6361

Study Officials

• Federico Grossi, MD, PhD

  Apellis Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 8, 2018
• First Posted: March 14, 2018
• Study Start: February 14, 2018
• Primary Completion: April 5, 2019
• Study Completion: April 5, 2019
• Last Updated: September 16, 2020
• Results First Posted: September 16, 2020

Record last verified: 2020-09

Locations

US (4)