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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05809531




Registration number
NCT05809531
Ethics application status
Date submitted
30/03/2023
Date registered
12/04/2023
Date last updated
12/03/2024

Titles & IDs
Public title
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
Scientific 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
Secondary ID [1] 0 0
2023-504625-39-00
Secondary ID [2] 0 0
APL2-C3G-314
Universal Trial Number (UTN)
Trial acronym
VALE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
C3G 0 0
IC-MPGN 0 0
C3 Glomerulopathy 0 0
C3 Glomerulonephritis 0 0
Complement 3 Glomerulopathy 0 0
Complement 3 Glomerulopathy (C3G) 0 0
Complement 3 Glomerulonephritis 0 0
Dense Deposit Disease 0 0
DDD 0 0
Membranoproliferative Glomerulonephritis 0 0
Membranoproliferative Glomerulonephritis (MPGN) 0 0
Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Kidney disease
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Pegcetacoplan

Experimental: Pegcetacoplan administered subcutaneously - Pegcetacoplan administered subcutaneously twice weekly according to protocol defined dosing regimen


Treatment: Drugs: Pegcetacoplan
Complement (C3) Inhibitor
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of participants with a reduction in urine protein-to-creatinine ratio (uPCR) of at least 50% from the pretreatment value over time.
Timepoint [1] 0 0
2.5 years

Eligibility
Key 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
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
29/05/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2027
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
St. Vincent's Hospital Melbourne (61003) - Fitzroy
Recruitment postcode(s) [1] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Iowa
Country [3] 0 0
United States of America
State/province [3] 0 0
Michigan
Country [4] 0 0
United States of America
State/province [4] 0 0
New Jersey
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
Brazil
State/province [7] 0 0
Minas Gerais
Country [8] 0 0
Brazil
State/province [8] 0 0
RS
Country [9] 0 0
Brazil
State/province [9] 0 0
Botucatu
Country [10] 0 0
Brazil
State/province [10] 0 0
Porto Alegre
Country [11] 0 0
Brazil
State/province [11] 0 0
Rio De Janeiro
Country [12] 0 0
Brazil
State/province [12] 0 0
São Paulo
Country [13] 0 0
Czechia
State/province [13] 0 0
Prague
Country [14] 0 0
France
State/province [14] 0 0
Bordeaux
Country [15] 0 0
Italy
State/province [15] 0 0
Milano
Country [16] 0 0
Italy
State/province [16] 0 0
Ranica
Country [17] 0 0
Korea, Republic of
State/province [17] 0 0
Seoul
Country [18] 0 0
Korea, Republic of
State/province [18] 0 0
Soeul
Country [19] 0 0
Netherlands
State/province [19] 0 0
Nijmegen
Country [20] 0 0
Spain
State/province [20] 0 0
Barcelona
Country [21] 0 0
Spain
State/province [21] 0 0
Madrid
Country [22] 0 0
Spain
State/province [22] 0 0
Valencia
Country [23] 0 0
Switzerland
State/province [23] 0 0
Lausanne
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Manchester
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Nottingham

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Apellis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/ct2/show/NCT05809531
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries