This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on theinvestigational medicine after you have been given it, and if this iscomparable to what is seen for the licensed medicine, eculizumab, inpatients with Paroxysmal Nocturnal Hemoglobinuria (PNH).This study will assess if the investigational medicine is safe and effective intreating PNH compared to the licensed medicine.

Phase 1

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

• Registration Number: EUCTR2017-001418-27-SI
• Lead Sponsor: Amgen Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-09-10
• Last Update Posted: 9 May 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

The study will enroll subjects with PNH who are stable on eculizumab treatment. Subjects cannot be enrolled or randomized before all inclusion criteria (including test results) are confirmed:
1. Men and women = 18 years of age
2. Historical diagnosis of PNH by documented flow cytometry
3. Administration of eculizumab for = 6 months and currently receiving 900 mg of eculizumab every 14 ± 2 days
4. Hemoglobin = 9.0 g/dL for at least 6 weeks prior to randomization
5. Lactate dehydrogenase (LDH) < 1.5 × the upper limit of normal at screening
6. Platelet count = 50 × 10^9/L
7. Absolute neutrophil count = 0.5 x 10^9/L (500/µL)
8. Subjects must be vaccinated against Neisseria meningitidis. Subjects must have been vaccinated or revaccinated according to current national guidelines for vaccination use.
9. Subjects must sign an institutional review board/independent ethics committee-approved informed consent form before participation in any procedures.
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 35
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion Criteria

If any of the following apply, the subject MUST NOT enter the study:
1. Known or suspected hereditary complement deficiency
2. Clinically significant cardiovascular disease (including myocardial infarction, unstable angina, symptomatic congestive heart failure [New York Heart Association = Class III], serious uncontrolled cardiac arrhythmia), peripheral vascular disease, cerebrovascular accident, or transient ischemic attack in the previous 6 months
3. Evidence of acute thrombosis (liver Doppler ultrasound of hepatic and portal veins)
4. Known to be positive for human immunodeficiency virus
5. Woman who is pregnant or breastfeeding
6. Woman of childbearing potential who does not consent to use a highly effective method of birth control (e.g., true abstinence, sterilization, birth control pills, Depo Provera injections, or contraceptive implants) during treatment and for an additional 5 months after the last administration of protocol-specified treatment
7. Man with a partner of childbearing potential who does not consent to use a highly effective method of birth control (eg, true abstinence, vasectomy, or a condom in combination with hormonal birth control or barrier methods used by the woman) during treatment and for an additional 5 months after the last administration of protocol-specified treatment
8. Subject is currently enrolled in or has not yet completed at least 30 days since ending other investigational device or drug study(s), or subject is receiving other investigational agent(s).
9. Subject has known sensitivity to any constituent of the products to be administered during the study, including mammalian cell-derived drug products.
10. History or evidence of clinically significant disorder, infection, condition, or disease that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion.
11. History of meningococcal infection
12. Presence or suspicion of active bacterial infection, or recurrent bacterial infection.
13. History of bone marrow transplantation
14. Red blood cell transfusion required within 12 weeks before randomization
15. Subject experienced = 2 breakthrough events, (ie, signs and symptoms of intravascular hemolysis, that require dose and/or schedule adjustments of eculizumab) in the previous 12 months before screening.

Study & Design

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