---
title: A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH
nct_id: NCT03593200
overall_status: COMPLETED
phase: PHASE2
sponsor: Apellis Pharmaceuticals, Inc.
study_type: INTERVENTIONAL
primary_condition: PNH
countries: Bulgaria, Serbia
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT03593200.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT03593200"
ct_last_update_post_date: 2020-12-22
last_seen_at: "2026-05-12T06:32:20.085Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH

**Official Title:** Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH).

**NCT ID:** [NCT03593200](https://clinicaltrials.gov/study/NCT03593200)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE2
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 4
- **Lead Sponsor:** Apellis Pharmaceuticals, Inc.
- **Conditions:** PNH
- **Start Date:** 2018-08-16
- **Completion Date:** 2019-10-22
- **CT.gov Last Update:** 2020-12-22

## Brief Summary

This is a Phase IIa, open-label, multiple dose, study in patients with PNH who have not received eculizumab (Soliris ®) in the past. A single cohort of subjects is planned for evaluation.

## Eligibility

- **Minimum age:** 18 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
Inclusion Criteria:

* At least 18 years old (inclusive)
* Diagnosed with PNH (white blood cell (WBC) clone \>10%)
* Lactose dehydrogenase (LD) ≥2 times the upper limit of normal
* Screening Ferritin ≥ normal and Total Iron Binding Capacity (TIBC) \< LLN based on central lab reference ranges. If a subject is receiving iron supplements at screening, the investigator must ensure that his/her dose has been stable for 8 weeks prior to enrolment and must be maintained throughout the study
* Last transfusion within 12 months prior to screening
* Platelet count of \>30,000/mm3 at the screening visit
* Absolute neutrophil count \>500/ mm3 at the screening visit
* Women of child-bearing potential (WOCBP) must have a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study
* Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study
* Vaccination against Neisseria meningitides types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing, or within 14 days after starting treatment with pegcetacoplan. Unless documented evidence exists that subjects are non-responders to vaccination as evidenced by titers or display titer levels within acceptable local limits
* Willing and able to give informed consent

Exclusion Criteria:

* Prior eculizumab (Soliris®) treatment
* Active bacterial infection
* Hereditary complement deficiency
* History of bone marrow transplantation
* Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin
* Participation in any other investigational drug trial or exposure to another investigational agent, device or procedure within 30 days
* Evidence of QTcF prolongation defined as \>450 ms for males and \>470 ms for females at screening
* Breast-feeding women
* History of meningococcal disease
```

## Arms

- **Experimental: Cohort 1** (EXPERIMENTAL) — 270 mg/day (up to 360 mg/day from Day 29) from Day 1 to Day 364\*

## Interventions

- **Pegcetacoplan** (DRUG) — Complement (C3) Inhibitor

## Primary Outcomes

- **Number of Subjects With Treatment Emergent Adverse Events (TEAEs) Including by Severity** _(time frame: From Day 1 to 30 days after the last dose (approximately 56 weeks))_ — TEAEs were defined as adverse events (AE) that occurred after dosing on Day 1 and up to 30 days after the last dose of study drug. A treatment-related TEAE was defined as a TEAE with a relationship to study drug of possible, probable, or definite. TEAEs were graded according to the Common Terminology Criteria for Adverse Events (v4.03) based on: Grade 1: Mild, Grade 2: Moderate, Grade 3: Severe, Grade 4: Life-threatening, Grade 5: Death related to AE.
- **Mean Change From Baseline in Lactate Dehydrogenase (LDH) Level** _(time frame: Baseline and Day 365)_ — Serum chemistry assessments of LDH were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the treatment period.
- **Mean Change From Baseline in Haptoglobin Level** _(time frame: Baseline and Day 365)_ — Serum chemistry assessments of haptoglobin were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the treatment period.
- **Mean Change From Baseline in Hemoglobin (Hb) Level** _(time frame: Baseline and Day 365)_ — Hematology assessments of Hb were made at the last measurement prior to the first dose of pegcetacoplan (baseline) and periodically throughout the treatment period.

## Secondary Outcomes

- **Mean Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Score** _(time frame: Baseline and Day 365)_
- **Mean Change From Baseline in Absolute Reticulocyte Count (ARC) Level** _(time frame: Baseline and Day 365)_
- **Mean Change From Baseline in Total Bilirubin Level** _(time frame: Baseline and Day 365)_
- **Mean Number of Red Blood Cell (RBC) Transfusions Per Month** _(time frame: From Day 1 to Day 364)_
- **Mean Change From Baseline in Linear Analog Scale Assessment (LASA) Score for QoL** _(time frame: Baseline and Day 365)_
- **Mean Serum Concentrations of Pegcetacoplan** _(time frame: Day 365)_
- **Mean Area Under the Serum Concentration Versus Time Curve From Time 0 to the Last Measurable Concentration at the End of the Study (AUCtotal)** _(time frame: Blood samples were collected predose and at least 2.5 hours post dose on Day 1 and predose on Days 2 up to Day 365.)_
- **Mean Maximum Observed Predose Serum Concentration During the Study (Ctrough,Max,Total)** _(time frame: Blood samples were collected predose and at least 2.5 hours post dose on Day 1 and predose on Days 2 up to Day 365.)_

## Locations (3)

- Acibadem City Clinic MHAT Tokuda EAD Sofia, Sofia, Bulgaria
- Specialized Hospital for Active Treatment of Hematologic Diseases EAD, Sofia, Sofia, Bulgaria
- Klinički centar Srbije, Belgrade, Serbia

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.acibadem city clinic mhat tokuda ead sofia|sofia||bulgaria` — added _(2026-05-12)_
- `locations.specialized hospital for active treatment of hematologic diseases ead, sofia|sofia||bulgaria` — added _(2026-05-12)_
- `locations.klinički centar srbije|belgrade||serbia` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT03593200.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT03593200*  
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