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 participants with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

An open-label, nonrandomized, multicenter extension study to evaluate the long-term safety and efficacy of Pegcetacoplan in participants with C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis

Mizuno Masashi

Nagoya University Hospital

65 Tsurumai-cho, Showa-ku, Nagoya City

mmizu@med.nagoya-u.ac.jp

Yoshi Shinoki

Worldwide Clinical Trials Japan K.K.

Shinagawa Intercity, Tower A, Level 28, 2-15-1, Konan, Minato-ku, Tokyo 108-6028

+81-3-6717-4360

SMP_APL314_jrct@worldwide.com

Not Recruiting

July. 23, 2024

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1. Participants must have completed participation in Study APL2-C3G-310 through the week 52 visit requirements.
2. Participants must have experienced clinical benefit from Pegcetacoplan while participating in the previous trial, in the opinion of the investigator.
3. Participants must remain on a stable regimen for C3G or IC-MPGN treatment according to the requirements of Study APL2-C3G-310.
4. Participants must have received vaccinations against S pneumoniae, N meningitidis (types A, C, W, Y, and B), and H influenzae (type B) according to the requirements of Study APL2-C3G-310 and agree to receive any additional vaccinations recommended according to ACIP recommendations for adults or children with complement deficiencies and/or immunocompromising conditions or other similar local applicable guidelines.
5. Female participants of childbearing potential, defined as any woman who has experienced menarche and who is not permanently sterile or postmenopausal, must have a negative urine pregnancy test at visit 1 and must agree to use protocol-defined methods of contraception for the duration of the study through at least 90 days after receiving the last dose of Pegcetacoplan.
6. Male participants must agree to use protocol-defined methods of contraception and agree to refrain from donating semen for the duration of the study through at least 90 days after receiving the last dose of Pegcetacoplan.
7. Participants above the legal age of consent, in accordance with local regulations, must be willing and able to provide informed consent. The legally authorized representative of participants under the legal age of consent must be willing and able to provide informed consent; where appropriate, participants under the legal age of consent must also give their assent to participation in the study.
8. Participants must be willing and able to self-administer Pegcetacoplan or have an identified caregiver who can perform the administration.

1. Female participants who are or are planning to become pregnant or who are currently breastfeeding and are unwilling to discontinue for the duration of the study and for at least 90 days after the final dose of study drug
2. Inability or unwillingness to cooperate with the requirements of the protocol
3. Any condition that, in the opinion of the investigator, creates an undue risk for the participant by participating in the study or is likely to confound interpretation of the study results
4. Evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator

Both

C3 GLOMERULOPATHY OR IMMUNE-COMPLEX MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS

All participants will receive SC infusions of Pegcetacoplan twice weekly. Adult participants and adolescent participants who weigh at least 50 kg will receive 20 mL SC infusions. Adolescent participants who weigh at least 35 kg but less than 50 kg will receive a reduced infusion volume (15 mL infusions). Adolescent participants who weigh at least 30 kg but less than 35 kg will receive a further reduced infusion volume (12 mL infusions). Participant weight will be assessed at each visit. If an adolescent participants weight has changed or if an adolescent participant has entered adulthood, the dose and infusion volume should be adjusted accordingly.

C562875

The log-transformed ratio of urine protein-to-creatinine ratio (uPCR) over time compared to pretreatment baseline

-Change from pretreatment in estimated glomerular filtration rate (eGFR) values over time
-The proportion of participants with a reduction in uPCR of at least 50% from the pretreatment value over time
-The proportion of participants with eGFR values that are stable or improved from pretreatment values over time
-The proportion of participants achieving proteinuria <1 g per day over time
-The change from pretreatment in the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) Scale score
-The time to progression to a composite clinical outcome (at least one of the following sustained doubling of serum creatinine, progression to chronic kidney disease stage 5 or end-stage renal disease, renal transplantation, or death from renal or cardiovascular disease)

+81-562-43-0500

May. 14, 2024

+81-95-819-7200

May. 14, 2024

May. 14, 2024

Yes

Requests for access to the deidentified data will be reviewed by Apellis External Research Committee.

Argentina/Australia/Austria/Belgium/Brazil/Canada/Czech Republic/France/Germany/Israel/Italy/Netherlands/South Korea/Spain/Switzerland/United Kingdom/United States