Skip to main content
OpenTrials
Completed

NCT Number: NCT00122317

Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Paroxysmal Hemoglobinuria, Nocturnal Anemia Anemia, Hemolytic Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinuria, Paroxysmal Hemolysis Myelodysplastic Syndromes Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Royal North Shore Hospital, Haematology Department, Saint Leonards, New South Wales, Australia

Loading trial locations.

About this study

An open-label extension study to evaluate long-term safety of eculizumab in PNH patients who had completed the TRIUMPH (C04-001), SHEPHERD (C04-002), and X03-001 studies.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

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

Treatment and study plan

Eculizumab

Drug

Other names: Soliris

Primary outcomes

  1. Incidence of Treatment-emergent Adverse Events

    Time frame: From time of consent to a maximum of 2.5 years of study treatment

Secondary outcomes

  1. Hemolysis as Measured by Change From Baseline in LDH Area Under the Curve

    Time frame: From time of first infusion through 24 months of study treatment

  2. Quality of Life as Measured by FACIT-Fatigue Scale Change From Baseline

    Time frame: From time of first infusion through 24 months of study treatment

    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.

  3. Incidence of Thrombosis After Eculizumab Infusion

    Time frame: From time of first ever dose through last dose (up to 24 months of study treatment)

    Thrombosis was defined as occurrence of major adverse vascular events

Sponsors and collaborators

Lead sponsor

Alexion Pharmaceuticals, Inc.

Industry

Registry information

Official study title

Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH

Important dates

Study start
2005
Primary completion
2008
Study completion
2008
First posted
Jul 22, 2005
Registry last updated
Mar 13, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.