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 the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine.

Phase 1

• Conditions: Paroxysmal Nocturnal Hemoglobinuria (PNH); MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

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

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-12-11
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Not specified
• Target Recruitment: 40

Inclusion Criteria

The study will enroll participants with PNH who are stable on eculizumab treatment. All inclusion criteria must be met.
1. Men and women = 18 years of age
2. Historical diagnosis of PNH by documented flow cytometry (e.g., type III erythrocyte cells of = 10%)
3. Administration of eculizumab for = 6 months and currently receiving 900 mg of eculizumab every 14 ± 2 days
4. Haemoglobin = 9.0 g/dL for at least 6 weeks prior to randomisation
5. Lactate dehydrogenase (LDH) < 1.5 × the upper limit of normal at screening
6. Platelet count = 50 × 1 09/L
7. Absolute neutrophil (a type of white blood cell) count = 0.5 x 1 09/L (500/µL)
8. Participants must have been vaccinated against Neisseria meningitidis (meningococcus – a bacteria which causes meningitis). Participants must be vaccinated or revaccinated according to current national guidelines for vaccination use.
9. Participants must sign an 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: 0
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 (heart attack), unstable angina, symptomatic congestive heart failure [New York Heart Association = Class III], serious uncontrolled cardiac arrhythmia), peripheral vascular disease (a blood circulation disorder), cerebrovascular accident (stroke), or transient ischemic attack (mini stroke) 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 (HIV)
5. Women who are pregnant or breastfeeding
6. Women of childbearing potential who does not consent to use a highly effective method of birth control (eg, true abstinence, sterilisation, 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
WOCBP is defined as a woman who is fertile, following menarche, and until becoming postmenopausal unless permanently sterile. Permanent sterilisation methods include hysterectomy, bilateral salpingectomy, and bilateral
oophorectomy.
A postmenopausal state is defined as no menses for 12 months without an alternative medical cause. A high follicle stimulating hormone (FSH) level in the postmenopausal range may be used to confirm a postmenopausal state in women not using hormonal contraception or hormonal replacement therapy (HRT).
However, in the absence of 12 months of amenorrhea, a single FSH measurement is insufficient.
Females on HRT and whose menopausal status is in doubt will be required to use 1 of the non-hormonal highly effective contraception methods if they wish to continue their HRT during the study. Otherwise, they must discontinue HRT to allow confirmation of postmenopausal status before study enrollment.
7. Males with partners of childbearing potential who do 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. Participant is currently enrolled in or has not yet completed at least 30 days since ending other investigational device or drug study(s), or the participant is receiving other investigational agent(s).
9. Particpant has known sensitivity to any constituent of the products to be administered during the study, including mammalian cell-derived 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 participant 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

Study & Design

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