Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD (ASAP II)

Apellis Pharmaceuticals

Status and phase: Completed; Phase 1

Conditions: Neovascular Age-Related Macular Degeneration

Treatments: Drug: Pegcetacoplan

Study type: Interventional

Funder types: Industry

Identifiers

NCT02461771

POT-CP043014

Details and patient eligibility

About

The objective of this study is to provide initial safety, tolerability and pharmacokinetics information of intravitreal administration of pegcetacoplan in order to support further development into larger Phase II studies for treatment of patients with AMD.

Enrollment: 13 patients

Sex: All

Ages: 50+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Male or Female

2. Age ≥ 50 years

3. The presence of an active choroidal neovascular lesion secondary to AMD

4. On treatment with anti-VEGF therapy (Lucentis®, Eylea® or Avastin®)

5. Must have received at least 3 anti-VEGF treatments over the 26-week period prior to screening (Screening Visit)

6. Evidence that the macular fluid has responded to anti-VEGF in the past based on OCT in the opinion of PI

7. At screening, evidence of subretinal fluid and retinal cystic changes

8. Must have received anti-VEGF treatment within 10 days prior to pegcetacoplan treatment (anti-VEGF can be administered on the same day of the screening visit after the screening procedures have been completed)

9. OCTs of sufficient quality to allow for the assessment of the central macular fluid can be obtained

10. 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

11. 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

12. Willing and able to give informed consent

Exclusion criteria

1. Choroidal neovascularization associated with other ocular diseases such as pathologic myopia, ocular histoplasmosis or posterior uveitis, etc

2. Decreased vision due to retinal disease not attributable to choroidal neovascularization, such as nonexudative forms of AMD, geographic atrophy, inherited retinal dystrophy, uveitis or epiretinal membrane, a vitelliform-like lesion of the outer retina (e.g., as in pattern dystrophies or basal laminar drusen), idiopathic parafoveal telangiectasis, or central serous retinopathy

3. Additional ocular diseases that have irreversibly compromised or, during follow-up, could likely compromise the VA of the study eye including amblyopia, anterior ischemic optic neuropathy, clinically significant diabetic macular edema, severe non proliferative diabetic retinopathy, or proliferative diabetic retinopathy

4. Decreased vision due to significant media opacity such as corneal disease or cataract, or opacity precluding photography of the retina

5. Cataract surgery within three months of enrollment

6. Presence of any hemorrhage

7. History of treatment for CNV:

   1. Previous PDT treatment within 30 days prior to enrollment in the study
   2. Previous extrafoveal or juxtafoveal thermal laser photocoagulation within 30 days prior to enrollment in the study

8. Intraocular surgery (including lens replacement surgery) within 3 months prior to randomization

9. Medical problems that make consistent follow-up over the treatment period unlikely (e.g. stroke, severe MI, end stage malignancy), or in general a poor medical risk because of other systemic diseases or active uncontrolled infections

10. Hypersensitivity to fluorescein

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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13 participants in 3 patient groups

Pegcetacoplan Cohort 1

Experimental group

Description:

4 mg of pegcetacoplan 100 μL IVT injection

Treatment:

Drug: Pegcetacoplan

Pegcetacoplan Cohort 2

Experimental group

Description:

10 mg of pegcetacoplan 100 μL IVT injection

Treatment:

Drug: Pegcetacoplan

Pegcetacoplan Cohort 3

Experimental group

Description:

20 mg of pegcetacoplan 100 μL IVT injection

Treatment:

Drug: Pegcetacoplan