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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00122317
• Date of registration: 20/07/2005
• Prospective Registration: No
• Primary sponsor: Alexion Pharmaceuticals
• 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
• Date of first enrolment: May 2005
• Target sample size: 187
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00122317
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Spain, Sweden, United Kingdom, United States

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Paroxysmal Hemoglobinuria, Nocturnal

Intervention(s)

Drug: eculizumab

Primary Outcome(s)

Incidence of Treatment-emergent Adverse Events [Time Frame: From time of consent to a maximum of 2.5 years of study treatment]

Secondary Outcome(s)

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]

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]

Incidence of Thrombosis After Eculizumab Infusion [Time Frame: From time of first ever dose through last dose (up to 24 months of study treatment)]

Secondary ID(s)

E05-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 05/06/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00122317