臨床研究等提出・公開システム

AN OPEN-LABEL, NONRANDOMIZED, MULTICENTER EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF PEGCETACOPLAN IN THE TREATMENT OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) (APL2-307)

Pegcetacoplan Long-term Safety and Efficacy Extension Study (APL2-307)

Uchendu Uchendu

Apellis Pharmaceuticals, Inc.

100 5th Ave, Waltham, MA 02451 USA

uchendu.uchendu@apellis.com

Moribe Maho

Fortrea Japan K.K.

Harumi Triton Square Office Tower Y 8F 1-8-11, Harumi, Chuo-ku, Tokyo

+81-50-3850-5519

Maho.Moribe@fortrea.com

Complete

April. 28, 2022

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1. Subjects at least 18 years of age with PNH who have participated in an antecedent pegcetacoplan clinical trial. Subjects who received treatment with pegcetacoplan must have experienced clinical benefit and adequate tolerability in the opinion of the investigator.
Note: Subjects with PNH who completed an antecedent pegcetacoplan clinical trial without receiving pegcetacoplan (or without receiving pegcetacoplan 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 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, 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.

1. Subjects who have withdrawn from a pegcetacoplan clinical study and/or subjects who met study drug discontinuation criteria during a pegcetacoplan clinical study.
2. Any condition that could increase the subjects 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. Acute or active hepatitis B, hepatitis C, or HIV infection.
6. Hereditary complement deficiency.
7. History of bone marrow transplant.
8. Concurrent severe aplastic anemia (defined by bone marrow cellularity <25% [or 25% to 50% if less than 30% of residual cells are hematopoietic] and at least 2 of the following values: peripheral blood absolute neutrophil count <500/micro-L [<0.5 x 109/L], peripheral blood platelet count <20,000/micro-L, peripheral blood reticulocyte count <20,000/micro-L).
9. History of meningococcal disease.
10. Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab).
11. Pregnancy, breastfeeding, or positive pregnancy test.

Both

Paroxysmal Nocturnal Hemoglobinuria (PNH)

A subcutaneous dose of pegcetacoplan (APL-2) 1080 mg twice a week, 3 times a week or every 3 days

Incidence and severity of treatment-emergent adverse events

+81-263-35-4600

Oct. 25, 2019

No

the United States/South Korea/Australia/Malaysia/Hong Kong/Philippines/Singapore/Thailand/Peru/Colombia/Mexico/Canada/Bulgaria/Serbia/Belgium/France/Germany/Italy/the Netherlands/Russia/Spain/the United Kingdom