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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT00122317




Registration number
NCT00122317
Ethics application status
Date submitted
20/07/2005
Date registered
20/07/2005
Date last updated
11/02/2018

Titles & IDs
Public title
Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)
Scientific title
Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH
Secondary ID [1] 0 0
E05-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - eculizumab

Experimental: Eculizumab - 600 mg intravenous infusion every week x 4 then 900 mg iv every two weeks


Treatment: Drugs: eculizumab


Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Treatment-emergent Adverse Events
Timepoint [1] 0 0
From time of consent to a maximum of 2.5 years of study treatment
Secondary outcome [1] 0 0
Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve
Timepoint [1] 0 0
From time of first infusion through 24 months of study treatment
Secondary outcome [2] 0 0
Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline - The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Patients score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.
Timepoint [2] 0 0
From time of first infusion through 24 months of study treatment
Secondary outcome [3] 0 0
Incidence of Thrombosis After Eculizumab Infusion - Thrombosis was defined as occurrence of major adverse vascular events
Timepoint [3] 0 0
From time of first ever dose through last dose (up to 24 months of study treatment)

Eligibility
Key inclusion criteria
- Patients who have fully completed the TRIUMPH, SHEPHERD, or X03-001 studies

- TRIUMPH patients who have discontinued receiving investigational drug prior to the
last visit of the study due to lack of efficacy or exacerbation of symptoms of PNH and
have completed all monthly safety and efficacy procedures

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

- Patient must avoid conception during the trial
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Patients who have terminated early from the SHEPHERD or X03-001 studies

- Patients who have terminated early from the TRIUMPH study due to an adverse event

- Female who is pregnant, breast feeding, or intending to conceive during the course of
the study

- Any condition that could increase the patient's risk by participating in the study or
could confound the outcome of the study

Study design
Purpose of the study
Treatment
Allocation to intervention
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
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)
NSW,QLD,SA,WA
Recruitment hospital [1] 0 0
Royal North Shore Hospital, Haematology Department - Saint Leonards
Recruitment hospital [2] 0 0
Princess Alexandra Hospital, Oncology Haematology Radiation Department - Woolloongabba
Recruitment hospital [3] 0 0
The Queen Elizabeth Hospital, Haematology/Oncology Department - Woodville South
Recruitment hospital [4] 0 0
The Royal Perth Hospital, Department of Haematology/Level 2 - Perth
Recruitment hospital [5] 0 0
Royal Melbourne Hospital, Department of Clinical Haematology & Medical Oncology - Parkville
Recruitment postcode(s) [1] 0 0
2065 - Saint Leonards
Recruitment postcode(s) [2] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [3] 0 0
5011 - Woodville South
Recruitment postcode(s) [4] 0 0
6000 - Perth
Recruitment postcode(s) [5] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
Belgium
State/province [13] 0 0
Brussels
Country [14] 0 0
Canada
State/province [14] 0 0
Alberta
Country [15] 0 0
Canada
State/province [15] 0 0
Ontario
Country [16] 0 0
France
State/province [16] 0 0
Cedex
Country [17] 0 0
Germany
State/province [17] 0 0
Essen
Country [18] 0 0
Germany
State/province [18] 0 0
Hannover
Country [19] 0 0
Germany
State/province [19] 0 0
Homburg/Saar
Country [20] 0 0
Germany
State/province [20] 0 0
Ulm
Country [21] 0 0
Ireland
State/province [21] 0 0
Dublin
Country [22] 0 0
Italy
State/province [22] 0 0
Firenze
Country [23] 0 0
Italy
State/province [23] 0 0
Genova
Country [24] 0 0
Italy
State/province [24] 0 0
Milano
Country [25] 0 0
Italy
State/province [25] 0 0
Napoli
Country [26] 0 0
Italy
State/province [26] 0 0
Vicenza
Country [27] 0 0
Netherlands
State/province [27] 0 0
GA
Country [28] 0 0
Spain
State/province [28] 0 0
Barcelona
Country [29] 0 0
Spain
State/province [29] 0 0
Madrid
Country [30] 0 0
Sweden
State/province [30] 0 0
Stockholm
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Leeds
Country [32] 0 0
United Kingdom
State/province [32] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Alexion Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to evaluate the long-term safety of eculizumab in patients with
transfusion dependent hemolytic PNH.
Trial website
https://clinicaltrials.gov/show/NCT00122317
Trial related presentations / publications
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
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No data has been provided for results reporting
Summary results
Other publications