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 - TRIUMPH

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2004-000646-20-SE
• Date of registration: 19/07/2004
• Prospective Registration: Yes
• Primary sponsor: ALEXION PHARMACEUTICALS INCORPORATED
• Public 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 - TRIUMPH
• 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 - TRIUMPH
• Date of first enrolment: 21/09/2004
• Target sample size: 75
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-000646-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: Single blind: Double blind: yes Parallel group: yes Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: yes Other:

Countries of recruitment

Ireland, Italy, Sweden

Key inclusion & exclusion criteria

Inclusion criteria:
1. Individuals at least 18 years of age
2. Patients must have required at least 4 episodes of transfusions in the 12 months prior to Visit 1 for anemia or anemia-related symptoms
3. Presence of a GPI deficient red blood cell clone (type III cells) by flow cytometry equal to or greater than 10%
4. Patients taking erythropoeitin 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
5. 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 rermain stable during the Observation Period and the Treatment Phase
6. Other criteria specified in the Protocol
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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Patients whose mean hemoglobin levels prior to transfusion over the previous 12 months is greater than 10.5 gm/dL
2. Absolute neutrophil count < 500 per microlitre
3. Presence or suspicion of active bacterial infection in the opinion of the investigator, at Visit 3 or recurrent bacterial infections
4. Known or suspected hereditary complement deficiency
5. Participation in any other drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to Visit 1
6. Pregnant, breast-feeding or intending to conceive during the course of the study, including the post-treatment phase
7. Any of the criteria stated in the Protocol



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0 Level: PT Classification code 10034042

Intervention(s)

Product Name: eculizumab
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: eculizumab
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Intravenous infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)

Primary end point(s): Co-primary endpoints for this study are: hemoglobin stablization and the number of PRBC units transfused during the treatment phase of the study to 26 weeks.

Main Objective: The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic paroxysmal hemoglobinuria (PNH)

Secondary Objective:

Secondary ID(s)

C04-001