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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03531255




Registration number
NCT03531255
Ethics application status
Date submitted
9/05/2018
Date registered
21/05/2018
Date last updated
3/07/2023

Titles & IDs
Public title
Pegcetacoplan Long Term Safety and Efficacy Extension Study
Scientific title
An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegcetacoplan in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)
Secondary ID [1] 0 0
APL2-307
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
PNH 0 0
Condition category
Condition code

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

Experimental: 1,080 mg pegcetacoplan administered subcutaneously - 1,080mg pegcetacoplan administered subcutaneously twice weekly or every three days.


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

Outcomes
Primary outcome [1] 0 0
Incidence and severity of treatment-emergent adverse events
Timepoint [1] 0 0
Baseline to 2 Years

Eligibility
Key inclusion criteria
Inclusion criteria:

1. Subjects at least 18 years of age with PNH who have participated in a 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 a 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 caregiver will be allowed)
5. Women of childbearing potential (WOCBP) 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Subjects who have withdrawn from a pegcetacoplan clinical study.
2. Any condition that could increase the subject's 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 study data.
4. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
5. Known infection with hepatitis B, C, or HIV.
6. Hereditary complement deficiency.
7. History of bone marrow transplant.
8. Concurrent severe aplastic anemia (SAA), defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or anti-thymocyte globulin.
9. History of meningococcal disease.
10. Concomitant treatment with any complement inhibitor (eg, eculizumab, ravulizumab).
11. Pregnancy, breastfeeding, or positive pregnancy test.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
27/08/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2025
Actual
Sample size
Target
160
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3000 - Melbourne
Recruitment outside Australia
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United States of America
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California
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United States of America
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Colorado
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Florida
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Illinois
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Indiana
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North Carolina
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United States of America
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Ohio
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United States of America
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Tennessee
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Belgium
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Brussels
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Belgium
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Roeselare
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Bulgaria
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Sofia
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Canada
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Alberta
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Canada
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Ontario
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Colombia
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Valle Del Cauca
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France
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Chalon-sur-Saône
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France
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Lille
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France
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Paris
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France
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Pierre-Bénite
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France
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Pringy
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France
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Saint-Quentin
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Germany
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BW
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Germany
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NRW
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Germany
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Hamburg
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Hong Kong
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Hong Kong
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Japan
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Aichi
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Japan
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Nagano
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Japan
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Tokyo
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Japan
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Wakayama
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Japan
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Okayama
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Korea, Republic of
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Daejeon
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Malaysia
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Kuala Lumpur
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Malaysia
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Selangor
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Mexico
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Monterrey
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Peru
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Lima
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Philippines
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Metro Manila
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Russian Federation
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Saint Petersburg
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Russian Federation
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Tyumen
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Serbia
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Belgrade
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Singapore
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Singapore
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Spain
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Islas Canarias
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Spain
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Valencia
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Thailand
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Bangkok
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Thailand
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Chiang Mai
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Thailand
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Khon Kaen
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Thailand
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Pathum Thani
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Thailand
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Songkhla
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United Kingdom
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Leeds

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

Ethics approval
Ethics application status

Summary
Brief summary
This is an Open-label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed a pegcetacoplan study.
Trial website
https://clinicaltrials.gov/study/NCT03531255
Trial related presentations / publications
Public notes

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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03531255