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).

Phase 1

• Conditions: Paroxysmal Nocturnal Hemoglobinuria; MedDRA version: 21.1Level: LLTClassification code 10055629Term: Paroxysmal nocturnal hemoglobinuriaSystem Organ Class: 100000004857; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2019-001106-23-DE
• Lead Sponsor: Apellis Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-09-30
• Last Update Posted: 23 May 2022

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 an antecedent 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 antecedent 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, or within 14 days after starting treatment with Pegcetacoplan (APL-2). Unless documented evidence exists that subjects are non-responders 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 (APL-2) (administration by caregiver will be allowed).
5. Women of child-bearing potential, 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 128
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 32

Exclusion Criteria

1. Subjects who have withdrawn from an Pegcetacoplan (APL-2) 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 study data.
4. History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
5. Acute or active infection with hepatitis B, hepatitis C, or HIV infection.
6. Hereditary complement deficiency.
7. History of bone marrow transplant.
8. Concurrent severe aplastic anemia (defined by bone marrow cellularity <25% [or 25% to 50% if less than 30% of residual cells are hematopoietic] and at least 2 of the following values: peripheral blood absolute neutrophil count <500/µL [<0.5 × 109/L], peripheral blood platelet count <20,000/µL, peripheral blood reticulocyte count <20,000/µL).
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