Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00098280
• Date of registration: 03/12/2004
• Prospective Registration: No
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
• Scientific title: A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
• Date of first enrolment: December 2004
• Target sample size: 75
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00098280
• Study type: Interventional
• Study design: Endpoint Classification: Efficacy Study, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

United States

Key inclusion & exclusion criteria

INCLUSION CRITERIA - Screening Period:

Individuals at least 18 years of age;

Patients must have required at least 4 episodes of transfusions in the 12 months prior to
Visit 1 for anemia or anemia-related symptoms;

Presence of a GPI deficient red blood cell clone (type II cells) by flow cytometry of
greater than or equal to 10%;

Patients taking erythropoietin must be on a stable dose for 26 weeks prior to visit 1 and
the dose must be expected to remain stable during the Observation Period and the Treatment
Phase;

Patients taking immunosuppressants must be on a stable dose for at least 26 weeks prior to
Visit 1 and the dose must be expected to remain stable during the Observation period and
the Treatment Phase;

Patients taking corticosteroids must be on a stable dose for at least 4 weeks prior to
Visit 1. The dose level may be decreased during the study as clinically appropriate. If
the corticosteroids need to be increased, they may not be increased above the dose
reported at Visit 1;

Patients are allowed to take coumadin, but must be at a stable international normalized
ratio (INR) level for 4 weeks prior to Visit 1 and are expected to maintain a stable INR
level during the Observation Period and the Treatment Phase;

Patients taking iron supplements or folic acid must be on a stable dose for 4 weeks prior
to Visit 3 and be expected to remain stable during Observation Period and the Treatment
Phase;

Patients are allowed to take low molecular weight heparin, but must be on a stable dose
for 4 weeks prior to Visit 1 and the dose must be expected to remain stable during
Observation period and the Treatment Phase;

Patient must be willing and able to give written informed consent;

Patients must be vaccinated against N. meningitidis at Visit 1 or at least 14 days prior
to Visit 3;

Patient must avoid conception during the trial using a method that is most appropriate for
their physical state and culture.

INCLUSION CRITERIA - Observation Period:

Documented LDH level greater than or equal to 1.5 x upper limit of normal (ULN) either at
Visit 1 or during the Observation Period;

Patients who have received one PRBC transfusion during the Observation Period at a
hemoglobin value of less than or equal to 9 gm/dL with symptoms or at a hemoglobin value
of less than or equal to 7 gm/dL without symptoms, and also within 1.5 gm/dL of the mean
hemoglobin pre-transfusion value for the previous 12 months. This transfusion should be
in accordance with that patient's individual transfusion hemoglobin algorithm, and occur
within 48 hours of the hemoglobin sample that precipitated the transfusion;

Platelet count greater than or equal to 100,000/mm(3) either at Visit 1 or during the
Observation Period.

EXCLUSION CRITERIA:

Patients whose mean hemoglobin levels prior to transfusion over the previous 12 months is
greater than 10.5 gm/dL;

Absolute Neutrophil count less than or equal to 500/microL;

Presence or suspicion of active bacterial infection, in the opinion of the Investigator,
at Visit 3 or recurrent bacterial infections;

Known or suspected hereditary complement deficiency;

Participation in any other investigational drug trial or exposure to other investigational
agent, device, or procedure within 30 days prior to Visit 1;

Pregnant, breast-feeding, or intending to conceive during the course of the study,
including the Post-treatment Phase;

History of meningococcal disease;

History of bone marrow transplantation;

Any condition that, in the opinion of the Investigator, could increase the patient's risk
by participating in the study or confound the outcome of the study.



Age minimum: N/A
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Hemoglobinuria, Paroxysmal

Intervention(s)

Drug: Eculizumab

Secondary ID(s)

05-H-0048

050048

Source(s) of Monetary Support

Please refer to primary and secondary sponsors