Not Applicable

• Register: EUCTR
• Last refreshed on: 8 May 2012
• Main ID: EUCTR2009-010402-11-Outside-EU/EEA
• Date of registration: 30/04/2012
• Prospective Registration: Yes
• Primary sponsor: ALEXION PHARMACEUTICALS, INC.
• Public title: Not Applicable
• Scientific title: AN OPEN-LABEL MULTI-CENTER STUDY OF ECULIZUMAB IN CHILDREN AND ADOLESCENTS WITH A DIAGNOSIS OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
• Target sample size: 6
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-010402-11
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: ALEXION EUROPE SAS

• Name: European Clinical Trial Information
• Affiliation: ALEXION EUROPE SAS

Key inclusion & exclusion criteria

Inclusion criteria:
•Subjects between 2-17 years;
•Diagnosis of PNH;
•subjects with = 5% glycosylphosphatidylinositol (GPI) deficient red blood cells or granulocytes as confirmed by flow cytometry;
•Written Informed Consent from parent/guardian;
•Negative pregnancy Test for Women of Child Bearing Potential;
•Sexually active females must document reliable and medically approved method of birth control;
•Patient must be vaccinated against N.meningitides at least 14 days prior to to study drug initiation or receive antibiotics for 14 days after the vaccination;

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
•Prior Eculizumab treatment;
•Presence of suspicion of active bacterial infection;
•Participation in another concurrent clinical trial;
•History of meningococcal disease;
•Pregnant, breast feeding or intending to conceive during study including Follow Up period.
•Any other condition that can confound the outcome of the study


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Body processes [G] - Immune system processes [G12]

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA
MedDRA version: 14.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders

Intervention(s)

Trade Name: SOLIRIS
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: ECULIZUMAB
CAS Number: 219685-50-4
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

Main Objective: PK/PD

Primary end point(s): To study PK/PD of Eculizumab doses in pediatric and adolescent PNH patients

Secondary Objective: Safety and Efficacy

Timepoint(s) of evaluation of this end point: PK and PD samples will be collected before and after completing the administration of the investigational product at each visit

Secondary Outcome(s)

Secondary end point(s): Safety and Efficacy determination of the chosen dosing regimen based on the age of the patient

Timepoint(s) of evaluation of this end point: 12 weeks

Secondary ID(s)

M07-005

Source(s) of Monetary Support

ALEXION PHARMACEUTICALS, INC.