NIPH Clinical Trials Search

JAPANESE
国立保健医療科学院
JRCT ID: jRCT2031210645

Registered date:04/03/2022

A Phase 3, Randomized, Multicenter, Open-label, Active-comparator Controlled Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Basic Information

Recruitment status Complete
Health condition(s) or Problem(s) studiedParoxysmal Nocturnal Hemoglobinuria (PNH)
Date of first enrollment04/03/2022
Target sample size8
Countries of recruitmentthe U.S.,Korea,Australia,Canada,Belgium,France,Germany,Russia,Spain,the United Kingdom,Japan
Study typeInterventional
Intervention(s)A subcutaneous dose of pegcetacoplan (APL-2) 1080 mg twice weekly (or change to a dose of 1080 mg every 3 days as necessary).

Outcome(s)

Primary OutcomeChange from baseline to Week 16 in hemoglobin level
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum>= 18age old
Age maximumNot applicable
GenderBoth
Include criteria1. At least 18 years of age 2. Primary diagnosis of PNH confirmed by high-sensitivity flow cytometry 3. On treatment with eculizumab. Dose of eculizumab must have been stable for at least 3 months prior to the screening visit 4. Hb <10.5 g/dL at the screening visit 5. Absolute reticulocyte count >1.0 x ULN at the screening visit 6. Platelet count of >50,000/mm3 at the screening visit 7. Absolute neutrophil count >500/mm3 at the screening visit 8. 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, 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 9. Women of child-bearing potential (WOCBP) must have a negative pregnancy test at the screening and Day -28 visit (run-in period) and must agree to use protocol defined methods of contraception for the duration of the study and 90 days after their last dose of study drug 10. Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study and 90 days after their last dose of study drug 11. Willing and able to give informed consent 12. Willing and able to self-administer pegcetacoplan (administration by caregiver will be allowed) 13. Have a body mass index (BMI) <35.0 kg/m2
Exclude criteria1. Active bacterial infection that has not resolved within 1 week of Day -28 (first dose of pegcetacoplan) 2. Receiving iron, folic acid, vitamin B12 and EPO, unless the dose is stable, in the 4 weeks prior to screening 3. Hereditary complement deficiency 4. History of bone marrow transplantation 5. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration 6. Participation in any other investigational drug trial or exposure to other investigational agent within 30 days or 5 half-lives (whichever is longer) 7. Currently breast-feeding women 8. Inability to cooperate or any condition that, in the opinion of the investigator, could increase the subjects risk of participating in the study or confound the outcome of the study This study includes cardiac safety evaluations. The following cardiac eligibility criteria are necessary to avoid confounding the cardiac safety outcomes: 9. History or family history of Long QT Syndrome or torsade de pointes, unexplained syncope, syncope from an uncorrected cardiac etiology, or family history of sudden death 10. Myocardial infarction, CABG, coronary or cerebral artery stenting and /or angioplasty, stroke, cardiac surgery, or hospitalization for congestive heart failure within 3 months or greater than Class 2 Angina Pectoris or NYHA Heart Failure Class >2 11. QTcF >470 ms, PR >280 ms 12. Mobitz II 2nd degree AV Block, 2:1 AV Block, High Grade AV Block, or Complete Heart Block unless the patient has an implanted pacemaker or implantable cardiac defibrillator (ICD) with backup pacing capabilities 13. Receiving Class 1 or Class 3 antiarrhythmic agents, or arsenic, methadone, ondansetron or pentamidine at screening 14. Receiving any other QTc-prolonging drugs (see Appendix 4), at a stable dose for less than 3 weeks prior to dosing 15. Receiving prophylactic ciprofloxacin, erythromycin or azithromycin for less than one week prior to the first dose of study medication (must have a repeat screening ECG after one week of prophylactic antibiotics with QTcF <470 ms)

Related Information

Contact

Public contact
Name Maho Moribe
Address Harumi Triton Square Office Tower Y 8F 1-8-11, Harumi, Chuo-ku, Tokyo Tokyo Japan 104-6108
Telephone +81-50-3850-5519
E-mail Maho.Moribe@labcorp.com
Affiliation Labcorp Development Japan K.K.
Scientific contact
Name Zurab Machaidze
Address 100 5th Ave, Waltham, MA 02451 USA Japan 02451
Telephone 1-617-834-5239
E-mail Zurab.Machaidze@apellis.com
Affiliation Apellis Pharmaceuticals, Inc.