Clinical trial NCT05809531: 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
Official title: An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: C3G
IC-MPGN
C3 Glomerulopathy
C3 Glomerulonephritis
Complement 3 Glomerulopathy
Complement 3 Glomerulopathy (C3G)
Complement 3 Glomerulonephritis
Dense Deposit Disease
DDD
Membranoproliferative Glomerulonephritis
Membranoproliferative Glomerulonephritis (MPGN)
Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
Lead sponsor: Apellis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2023-04-12
Enrollment: 100
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This study is designed as a long-term extension to Study APL2-C3G-310, and is being conducted to establish the long-term safety and efficacy of pegcetacoplan in patients with C3 glomerulopathy (C3G) or immune-complex membranoproliferative glomerulonephritis (IC-MPGN).


Eligibility criteria: Inclusion Criteria:

* Completed participation in Study APL2-C3G-310 through the week 52 visit requirements
* Experienced clinical benefit from pegcetacoplan while participating in the previous trial, in the opinion of the investigator
* Must remain on a stable regimen for C3G or IC-MPGN treatment according to the requirements of Study APL2-C3G-310
* 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
* 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
* 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
* 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
* Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration

Exclusion Criteria:

* 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
* Inability or unwillingness to cooperate with the requirements of the protocol
* 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
* Evidence of ongoing drug or alcohol abuse or dependence, in the opinion of the investigator

Primary outcomes:
measure: Proportion of participants with a reduction in urine protein-to-creatinine ratio (uPCR) of at least 50% from the pretreatment value over time.
timeFrame: 2.5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Academic Medical Research Institute (01034)
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado (01037)
state: Colorado
city: Gainesville
country: United States
facility: University of Florida, Department of Pediatric Nephrology (01010)
state: Florida
city: Atlanta
country: United States
facility: Emory Investigational Drug Service (01021)
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa Hospital and Clinics (01030)
state: Iowa
city: Ann Arbor
country: United States
facility: University of Michigan Hospital (01008)
state: Michigan
city: Hackensack
country: United States
facility: Hackensack Meridian Health (01036)
state: New Jersey
city: Hyde Park
country: United States
facility: Cohen Children Hospital, Pediatric Pharmacy (01022)
state: New York
city: New York
country: United States
facility: Columbia Nephrology (01039)
state: New York
city: Portland
country: United States
facility: Oregon Health & Science Univ., Dept. Pediatrics (01038)
state: Oregon
city: Bethlehem
country: United States
facility: Northeast Clinical Research Center LLC (01009)
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital (01027)
state: Texas
city: Cordoba
country: Argentina
facility: Hospital Privado-Universitario de Cordoba (54004)
city: Cordoba
country: Argentina
facility: Clinica Privada Velez Sarsfield (54007)
city: Woolloongab
country: Australia
facility: Kidney Trials Unit, Princess Alexandra Hospital (61007)
state: Queensland
city: Fitzroy
country: Australia
facility: St. Vincent's Hospital Melbourne (61003)
city: Leuven
country: Belgium
facility: Catholic University of Leuven (32001)
city: Belo Horizonte
country: Brazil
facility: Santa Casa de Misericordia de Belo Horizonte (55017)
state: Minas Gerais
city: Porto Alegre
country: Brazil
facility: Irmandade da Santa Casa Misericordia Porto Alegre (55016)
state: RS
city: Botucatu
country: Brazil
facility: HC UNESP Botucatu (55010)
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre (55018)
city: Recife
country: Brazil
facility: Real Hospital Portugues de Beneficencia (55015)
city: Rio De Janeiro
country: Brazil
facility: Ruschel Medicina E Pesquisa Clinica (55012)
city: Sao Jose do Rio Preto
country: Brazil
facility: Hospital De Base (55014)
city: Sao Paulo
country: Brazil
facility: Univ Sao Paulo, Av. Dr Eneas Carvalho de Aguiar, 155 (55007)
city: São Paulo
country: Brazil
facility: Hospital do Rim - Fundacao Oswaldo Ramos (55005)
city: Prague
country: Czechia
facility: Institute for Clinical and Experimental Medicine (42001)
city: Bordeaux
country: France
facility: CHU de Bordeaux - Hopital Pellegrin (33010)
city: Nantes
country: France
facility: Institut de transplantation urologie-nephrologie (33011)
city: Petah Tikva
country: Israel
facility: Schneider Childrens Medical Center (97204)
city: Milano
country: Italy
facility: Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (39004)
city: Ranica
country: Italy
facility: Istituto di Ricerche Farmacologiche Mario Negri IRCCS (39003)
city: Rome
country: Italy
facility: Ospesale Pediatrico bambino Gesu (39005)
city: Nagasaki-shi
country: Japan
facility: Nagasaki University Hospital (81005)
state: Nagasaki
city: Hamamatsu
country: Japan
facility: Seirei Hamamatsu General Hospital (81004)
state: Shizuoka
city: Seoul
country: Korea, Republic of
facility: Yonsei University College of Medicine, Sinchon Severance Hospital (82002)
city: Soeul
country: Korea, Republic of
facility: Seoul National University Hospital (82003 & 82005)
city: Amsterdam
country: Netherlands
facility: AMC Apotheek Goederenontvangst (31002)
city: Groningen De Brug
country: Netherlands
facility: University Medical Center Groningen (31004)
city: Nijmegen
country: Netherlands
facility: Radboud University Medical Center (31003)
city: Barcelona
country: Spain
facility: Hosp. Universit. Materno-Infantil Vall d' Hebron (34006)
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre (34008)
city: Valencia
country: Spain
facility: Hospital Universitario Dr Peset (34009)
city: Lausanne
country: Switzerland
facility: CHUV Lausanne (41002)
city: London
country: United Kingdom
facility: Imperial College Hammersmith Hospital (44013)
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital (44011)
city: Nottingham
country: United Kingdom
facility: Nottingham University Hospitals NHS Trust (44012)
Study officials:
References: