A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH

• Register: ClinicalTrials.gov
• Last refreshed on: 14 November 2022
• Main ID: NCT03818607
• Date of registration: 17/01/2019
• Prospective Registration: Yes
• Primary sponsor: Amgen
• Public title: A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH DAHLIA
• Scientific title: A Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Date of first enrolment: April 24, 2019
• Target sample size: 42
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03818607
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Czechia, Finland, France, Germany, Ireland, Italy, Netherlands, Norway, Portugal, Slovenia, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States

Contacts

• Name: MD
• Affiliation: Amgen

Key inclusion & exclusion criteria

Inclusion Criteria:

- Men and women = 18 years of age.

- Historical diagnosis of PNH.

- Administration of eculizumab for = 6 months and currently receiving 900 mg of
eculizumab.

- Hemoglobin = 9.0 g/dL for at least 6 weeks before randomization.

- Lactate dehydrogenase < 1.5 × the upper limit of normal at screening.

- Platelet count = 50 × 10^9/L.

- Absolute neutrophil count (ANC) > 0.5 x 10^9/L (500/µL).

- Participants must be vaccinated against Neisseria meningitidis.

- Participants must sign an IRB/IEC-approved ICF before participation in any procedures.

Exclusion Criteria:

- Known or suspected hereditary complement deficiency.

- 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.

- Evidence of acute thrombosis (liver Doppler ultrasound of hepatic and portal veins).

- Known to be positive for human immunodeficiency virus.

- Woman who is pregnant or breastfeeding.

- Participant is currently enrolled in or has not yet completed at least 30 days since
ending other investigational device or drug study(s), or participant is receiving
other investigational agent(s).

- Participant has known sensitivity to any of the products to be administered during the
study, including mammalian cell-derived drug products.

- History of meningococcal infection.

- Presence or suspicion of active bacterial infection, or recurrent bacterial infection.

- History of bone marrow transplantation.

- Red blood cell transfusion required within 12 weeks before randomization.

- Participant 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Intervention(s)

Drug: ABP 959

Drug: Eculizumab

Primary Outcome(s)

Hemolysis as measured by LDH (Crossover Comparison) [Time Frame: 18 months]

Hemolysis as measured by Lactate dehydrogenas (LDH) (Parallel Comparison) [Time Frame: 12 months]

Secondary Outcome(s)

Bilirubin [Time Frame: 18 months]

Haptoglobin [Time Frame: 18 months]

Total hemoglobin [Time Frame: 18 months]

Lactate dehydrogenase-time profile [Time Frame: 18 months]

Crossover comparison of hemolysis as measured by LDH [Time Frame: 18 months]

Pharmacokinetic area under the curve (AUC) of eculizumab [Time Frame: 18 months]

Serum-free hemoglobin [Time Frame: 18 months]

Total hemolytic complement (Total Complement) [Time Frame: 18 months]

Pharmacokinetic area under the curve (AUC) of ABP 959 [Time Frame: 18 months]

Degree of hemoglobinuria [Time Frame: 18 months]

Incidence of red blood cell transfusion [Time Frame: 18 months]

Type III erythrocytes [Time Frame: 18 months]

Secondary ID(s)

20150168

Source(s) of Monetary Support

Please refer to primary and secondary sponsors