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).

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 160

Inclusion Criteria

1. Subjects at least 18 years of age with PNH who have participated in a Pegcetacoplan (APL-2) clinical trial.
Subjects who received treatment with Pegcetacoplan (APL-2) must have experienced
clinical benefit and adequate tolerability in the opinion of the Investigator.
Note: Subjects with PNH who completed an Pegcetacoplan (APL-2) clinical trial without
receiving Pegcetacoplan (APL-2) (or without receiving Pegcetacoplan (APL-2) 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 (APL-2) 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 (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.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 129
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 31

Exclusion Criteria

1. Subjects who withdrew from a Pegcetacoplan (APL-2) clinical study clinical study and/or subjects who met study drug discontinuation criteria during 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 the 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

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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