Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Interventions
Product Name: Pegcetacoplan (APL-2)
Product Code: Pegcetacoplan (APL-2)
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: pegcetacoplan
CAS Number: 2019171-69-6
Current Sponsor
Sponsors
Apellis Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subjects at least 18 years of age with PNH who have participated in a Pegcetacoplan (APL-2) clinical trial. Subjects who received treatment with Pegcetacoplan (APL-2) must have experienced clinical benefit and adequate tolerability in the opinion of the Investigator. Note: Subjects with PNH who completed an Pegcetacoplan (APL-2) clinical trial without receiving Pegcetacoplan (APL-2) (or without receiving Pegcetacoplan (APL-2) for long enough to demonstrate clinical benefit) may be enrolled in this study if, in the opinion of the Investigator, the subject is expected to demonstrate clinical benefit upon the initiation or continuation of Pegcetacoplan (APL-2) therapy. 2. Vaccination against Neisseria meningitidis types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing of this study, or within 14 days after starting treatment with pegcetacoplan. Vaccination is mandatory unless documented evidence exists that subjects are nonresponders to vaccination as evidenced by titers or display titer levels within acceptable local limits. Immunization status checks will be performed to determine whether subjects require primary or booster vaccinations. 3. Willing and able to give written informed consent. 4. Willing and able to self-administer pegcetacoplan (administration by a caregiver will be allowed). 5. Women of childbearing potential (WOCBP), defined as any females who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative pregnancy test and must agree to continue to use an approved method of contraception for the duration of the study and 90 days after their last dose of study drug. Note: Postmenopausal is defined as 12 consecutive months with no menses without an alternative medical cause. 6. Males must agree to continue to use an approved method of contraception and must agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 129 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 31
Exclusion criteria
Exclusion criteria: 1. Subjects who withdrew from a Pegcetacoplan (APL-2) clinical study clinical study and/or subjects who met study drug discontinuation criteria during a pegcetacoplan clinical study. 2. Any condition that could increase the subject’s risk by participating in the study 3. Any comorbidity or condition (such as malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound the study data. 4. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration. 5. Known infection with hepatitis B, C, or HIV. 6. Hereditary complement deficiency. 7. History of bone marrow transplant. 8. 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. 9. History of meningococcal disease. 10. Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab). 11. Pregnancy, breastfeeding, or positive pregnancy test.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The objectives of this study are to: 1. Establish the long-term safety of Pegcetacoplan (APL-2) in PNH subjects 2. Establish the long-term efficacy of Pegcetacoplan (APL-2) in PNH subjects;Secondary Objective: Not applicable;Primary end point(s): Safety Endpoints • Incidence and severity of Treatment-emergent Adverse Events (TEAE) • Incidence of thromboembolic events • Laboratory parameters • ECG parameters Efficacy Endpoints • Lactate dehydrogenase (LDH) level • Absolute Reticulocyte count (ARC) • Hemoglobin level • Red blood cell (RBC) transfusions • FACIT-fatigue scale score;Timepoint(s) of evaluation of this end point: Safety Endpoints • Incidence and severity of Treatment-emergent Adverse Events (TEAE): after dosing begins, or worsen of severity, for up to 30 days after the last dose of Pegcetacoplan (APL-2) • Incidence of thromboembolic events: after dosing begins for up to 30 days after the last dose of Pegcetacoplan (APL-2) • Laboratory parameters: Please refer to Schedule Events in Protocol • ECG parameters: Please refer to Schedule Events in Protocol Efficacy Endpoints: Please Refer to Schedule Events in Protocol | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Pharmacokinetic Endpoint • Pegcetacoplan (APL-2) pharmacokinetic concentrations Pharmacodynamic Endpoints • Complement (e.g., CH50, AH50, and C3) levels • C3 deposition on RBC cells • Clonal distribution of PNH RBCs • Incidence of anti-APL-2 and anti-PEG antibodies;Timepoint(s) of evaluation of this end point: Pharmacokinetic Endpoints: Blood samples will be collected at the time points delineated in the Schedule of Events in Section 3 of Protocol Pharmacodynamic Endpoints: Blood samples will be collected at the time points delineated in the Schedule of Events in Section 3 of Protocol | — |
Countries
Australia, Bulgaria, Canada, France, Hong Kong, Japan, Korea, Republic of, Malaysia, Russian Federation, Serbia, Spain, Thailand, United Kingdom, United States
Contacts
Public ContactEuropean Project Operations
Biorasi GmbH
Outcome results
None listed