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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00122304




Registration number
NCT00122304
Ethics application status
Date submitted
19/07/2005
Date registered
22/07/2005
Date last updated
21/02/2007

Titles & IDs
Public title
Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
Scientific title
Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
Secondary ID [1] 0 0
C04-002
Secondary ID [2] 0 0
SHEPHERD
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
Treatment: Drugs - eculizumab

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

Outcomes
Primary outcome [1] 0 0
Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)
Timepoint [1] 0 0
Primary outcome [2] 0 0
Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.
Timepoint [2] 0 0
Secondary outcome [1] 0 0
Hemolysis measured by the change of LDH from baseline;
Timepoint [1] 0 0
Secondary outcome [2] 0 0
Quality of Life
Timepoint [2] 0 0

Eligibility
Key inclusion criteria
- PNH > 6 months

- Type III PNH red blood cell (RBC) clone by flow cytometry >10%

- At least one transfusion in the past 2 years but no more than 3 transfusions in the
past 12 months; or personal beliefs that preclude the use of transfusion with severe
hemolytic PNH

- Lactate dehydrogenase (LDH) >1.5 x upper limit of normal

- Must avoid conception

- Willing and able to give written informed consent
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Platelet count of <30,000/mm3

- Absolute neutrophil count <500/ul

- Active bacterial infection

- Hereditary complement deficiency

- History of bone marrow transplantation

- Participation in any other investigational drug trial or exposure to other
investigational agent, device or procedure within 30 days

- Pregnant, breast-feeding, or intending to conceive

- History of meningococcal disease

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
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
1/12/2004
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)
NSW,QLD,SA,WA
Recruitment hospital [1] 0 0
Royal North Shore Hospital, Haematology Department - St. Leonard
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, Dept. of Haematology/Level 2 - Perth
Recruitment hospital [5] 0 0
Royal Melbourne Hospital, Dept. of Clinical Haematology & Medical Oncology - Parkville
Recruitment postcode(s) [1] 0 0
2065 - St. Leonard
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
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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
State/province [5] 0 0
Maryland
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United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
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United States of America
State/province [8] 0 0
Missouri
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United States of America
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New Jersey
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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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Oregon
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United States of America
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Pennsylvania
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United States of America
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Utah
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Belgium
State/province [16] 0 0
Brussels
Country [17] 0 0
Canada
State/province [17] 0 0
Alberta
Country [18] 0 0
Canada
State/province [18] 0 0
Ontario
Country [19] 0 0
France
State/province [19] 0 0
Cedex
Country [20] 0 0
Germany
State/province [20] 0 0
Essen
Country [21] 0 0
Germany
State/province [21] 0 0
Hannover
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Germany
State/province [22] 0 0
Homburg/Saar
Country [23] 0 0
Germany
State/province [23] 0 0
Ulm
Country [24] 0 0
Ireland
State/province [24] 0 0
Dublin
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Italy
State/province [25] 0 0
Firenze
Country [26] 0 0
Italy
State/province [26] 0 0
Genova
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Italy
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Milano
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Italy
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Napoli
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Italy
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Vicenza
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Netherlands
State/province [30] 0 0
Nijmegen
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Spain
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Barcelona
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Spain
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Madrid
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Sweden
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Stockholm
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Sweden
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Uppsala
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Switzerland
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Basel
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United Kingdom
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Cornwall
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United Kingdom
State/province [37] 0 0
Leeds
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United Kingdom
State/province [38] 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 of eculizumab in patients with
transfusion-dependent hemolytic PNH
Trial website
https://clinicaltrials.gov/ct2/show/NCT00122304
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00122304