An Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 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-ES
• Lead Sponsor: Apellis Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-01-14
• Last Update Posted: 23 October 2023

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 119

Inclusion Criteria

1. Subjects with PNH who have participated in an APL-2 clinical trial.
Subjects who received treatment with APL-2 must have experienced
clinical benefit in the opinion of the Investigator.
Note: Subjects with PNH who completed an APL-2 clinical trial without
receiving APL-2 (or without receiving 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 APL-2 therapy.
2. Willing and able to give written informed consent.
3. Women of child-bearing potential (WOCBP) 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 60 days after their last
dose of study drug.
4. 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 60 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 66
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 16

Exclusion Criteria

1. Subjects who have withdrawn from an APL-2 clinical study
2. Any condition that could increase the subject’s risk by participating in the study

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified