A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH

• Register: ClinicalTrials.gov
• Last refreshed on: 24 October 2022
• Main ID: NCT04085601
• Date of registration: 09/09/2019
• Prospective Registration: No
• Primary sponsor: Apellis Pharmaceuticals, Inc.
• Public title: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
• Scientific title: A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Date of first enrolment: August 27, 2019
• Target sample size: 53
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04085601
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Colombia, Hong Kong, Malaysia, Mexico, Peru, Philippines, Poland, Serbia, Singapore, Thailand

Key inclusion & exclusion criteria

Inclusion Criteria:

- Be at least 18 years old (inclusive).

- Have LDH =1.5 x ULN at the screening visit.

- Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or
monocyte clone >10%).

- Have Hb less than the lower limit of normal (LLN) at the screening visit.

- Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC)
less than/equal to ULN at the screening visit, based on central laboratory reference
ranges. If a subject is receiving iron supplements at screening, the Investigator must
ensure that the subject's dose has been stable for 4 weeks prior to screening, and it
must be maintained throughout the study. Subjects not receiving iron at screening must
not start iron supplementation during the course of the study.

- Body mass index (BMI) = 35 kg/m2 at the screening visit.

- Have a platelet count of >50,000/mm3 at the screening visit.

- Have an absolute neutrophil count >500/mm3 at the screening visit.

Exclusion Criteria:

- Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to
screening.

- Hereditary complement deficiency.

- History of bone marrow transplantation.

- Concomitant use of any of the following medications is prohibited if not on a stable
regimen for the time period indicated below prior to screening:

- Erythropoietin or immunosuppressants for at least 8 weeks

- Systemic corticosteroids for at least 4 weeks

- Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio
(INR) for at least 4 weeks

- Iron supplements, vitamin B12, or folic acid for at least 4 weeks

- Low-molecular-weight heparin for at least 4 weeks



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Intervention(s)

Drug: APL-2

Primary Outcome(s)

Change From Baseline in Lactate Dehydrogenase (LDH) Concentration At Week 26 [Time Frame: Baseline (Day 1) and Week 26]

Number of Subjects Who Achieved Hemoglobin (Hb) Stabilization [Time Frame: From Baseline (Day 1) up to Week 26]

Secondary Outcome(s)

Change From Baseline in Linear Analog Assessment (LASA) Scales Score at Week 26 [Time Frame: Baseline and Week 26]

Number of Subjects With Failure of Hb Stabilization [Time Frame: Up to Week 26]

Percentage of Subjects With LDH Normalization at Week 26 [Time Frame: At Week 26]

Number of PRBC Units Transfused From Baseline Through Week 26 [Time Frame: Up to Week 26]

Change From Baseline in European Organization for Research and Treatment of Cancer (EORTC) 30-item Core Quality of Life Questionnaire (QLQ-C30) Scores at Week 26 [Time Frame: Baseline and Week 26]

Percentage of Subjects Who Received Transfusion or Decrease of Hb >2 g/dL From Baseline [Time Frame: At Week 26]

Percentage of Subjects With ARC Normalization [Time Frame: At Week 26]

Change From Baseline in Absolute Reticulocyte Count (ARC) at Week 26 [Time Frame: Baseline and Week 26]

Change From Baseline in Functional Assessment of Chronic Illness Therapy- (FACIT-Fatigue) Scale Score at Week 26 [Time Frame: Baseline and Week 26]

Number of Subjects With an Hb Response in the Absence of Transfusions [Time Frame: Baseline and Week 26]

Time to First PRBC Transfusion [Time Frame: Up to Week 26]

Change From Baseline in Hb Concentration at Week 26 [Time Frame: Baseline and Week 26]

Percentage of Subjects With Hb Normalization Levels at Week 26 [Time Frame: Baseline and Week 26]

Percentage of Subjects With Transfusion Avoidance [Time Frame: At Week 26]

Secondary ID(s)

APL2-308

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 21/10/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT04085601