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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00122330




Registration number
NCT00122330
Ethics application status
Date submitted
18/07/2005
Date registered
22/07/2005
Date last updated
4/12/2006

Titles & IDs
Public title
Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
Scientific title
Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients
Secondary ID [1] 0 0
TRIUMPH
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemoglobinuria, Paroxysmal 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
* Must have required at least 4 transfusions in the past 12 months
* PNH type III red blood cell (RBC) clone by flow cytometry of >10%
* Lactate dehydrogenase (LDH) level > 1.5 x upper limit of normal
* Platelet count > 100,000/mm3
* Patient taking erythropoietin must be on a stable dose for at least 26 weeks
* Patient taking immunosuppressants must be on a stable dose for at least 26 weeks
* Patient taking corticosteroids must be on a stable dose for at least 4 weeks
* Patient taking coumadin must be at a stable INR for at least 4 weeks
* Patient taking iron supplements or folic acid must be on a stable dose for 4 weeks
* Willing and able to give written informed consent
* Must avoid conception
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Mean hemoglobin level prior to transfusion over the previous 12 months is >10.5 gm/dl
* Absolute neutrophil count <500/ul
* Active bacterial infection
* Hereditary complement deficiency
* Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedures within 30 days
* Pregnant, breast-feeding, or intending to conceive
* History of meningococcal disease
* History of bone marrow transplantation

Study design
Purpose of the study
Educational/counselling/training
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/10/2004
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
Princess Alexandra Hospital, Oncology Haematology Radiation Department - Woolloongabba
Recruitment hospital [2] 0 0
Queen Elizabeth Hospital, Dept. of Haematology - Woodville South
Recruitment hospital [3] 0 0
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology - Parkville
Recruitment hospital [4] 0 0
The Royal Perth Hosptial, Department of Haematology/Level 2 - Perth
Recruitment postcode(s) [1] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [2] 0 0
5011 - Woodville South
Recruitment postcode(s) [3] 0 0
3050 - Parkville
Recruitment postcode(s) [4] 0 0
6000 - Perth
Recruitment outside Australia
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United States of America
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California
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United States of America
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Connecticut
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United States of America
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Florida
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United States of America
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Indiana
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United States of America
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Maryland
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United States of America
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Massachusetts
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United States of America
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Missouri
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United States of America
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New York
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United States of America
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North Carolina
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United States of America
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Ohio
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United States of America
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Pennsylvania
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Belgium
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Brussels
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Canada
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Alberta
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France
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Paris
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Germany
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Essen
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Germany
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Greifswald
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Germany
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Hannover
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Germany
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Homburg/Saar
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Germany
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Ulm
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Ireland
State/province [20] 0 0
Dublin
Country [21] 0 0
Italy
State/province [21] 0 0
Firenze
Country [22] 0 0
Italy
State/province [22] 0 0
Genova
Country [23] 0 0
Italy
State/province [23] 0 0
Milano
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Italy
State/province [24] 0 0
Napoli
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Italy
State/province [25] 0 0
Vicenza
Country [26] 0 0
Netherlands
State/province [26] 0 0
GA
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Sweden
State/province [27] 0 0
Lund
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Sweden
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Umea
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Switzerland
State/province [29] 0 0
CH
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United Kingdom
State/province [30] 0 0
Cornwall
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Belfast
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Leeds
Country [33] 0 0
United Kingdom
State/province [33] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Alexion Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The primary objective is to evaluate the safety and efficacy of eculizumab in transfusion dependent patients with hemolytic PNH.
Trial website
https://clinicaltrials.gov/study/NCT00122330
Trial related presentations / publications
Hillmen P, Young NS, Schubert J, Brodsky RA, Socie G, Muus P, Roth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. doi: 10.1056/NEJMoa061648.
Hill A, Rother RP, Wang X, Morris SM Jr, Quinn-Senger K, Kelly R, Richards SJ, Bessler M, Bell L, Hillmen P, Gladwin MT. Effect of eculizumab on haemolysis-associated nitric oxide depletion, dyspnoea, and measures of pulmonary hypertension in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2010 May;149(3):414-25. doi: 10.1111/j.1365-2141.2010.08096.x. Epub 2010 Mar 8.
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
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Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00122330