Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent PNH Subjects

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Alexion Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT00867932

First received: March 23, 2009

Last updated: November 17, 2011

Last verified: November 2009

History of Changes

Tracking Information
First Received Date ^ICMJE	March 23, 2009
Last Updated Date	November 17, 2011
Start Date ^ICMJE	May 2009
Primary Completion Date	May 2011 (final data collection date for primary outcome measure)
Current Primary Outcome Measures ^ICMJE (submitted: March 23, 2009)	To study PK/PD of Eculizumab doses in pediatric and adolescent PNH patients [ Time Frame: 15 months ] [ Designated as safety issue: Yes ]
Original Primary Outcome Measures ^ICMJE	Same as current
Change History	Complete list of historical versions of study NCT00867932 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ^ICMJE (submitted: March 23, 2009)	Safety and Efficacy determination of the chosen dosing regimen based on the age of the patient. [ Time Frame: 15 months ] [ Designated as safety issue: Yes ]
Original Secondary Outcome Measures ^ICMJE	Same as current
Current Other Outcome Measures ^ICMJE
Original Other Outcome Measures ^ICMJE

Descriptive Information
Brief Title ^ICMJE	Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent PNH Subjects
Official Title ^ICMJE	An Open Label Multi Center Study of Eculizumab in Children and Adolescents With Diagnosis of Paroxysmal Nocturnal Hemoglobinuria
Brief Summary	The purpose of this study is to determine appropriate dose regimens of Eculizumab in pediatric and/or adolescent patients with paroxysmal Nocturnal hemoglobinuria (PNH).
Detailed Description	This is an open label, multicenter study of eculizumab administered intravenously (IV) to approximately 6 to 8 pediatric and adolescent patients with PNH. Patients will be stratified into one of three cohorts based on age. Cohort 1: age 2-7 years Cohort 2: age 8-11 years Cohort 3: age 12 -17 years Depending on the age of the patient, induction and maintenance doses vary as follows: Cohort 1: Induction: 300mg/week x 4 weeks, then 450mg/week x 1 week; Cohort 1: Maintenance: 450mg every 2 weeks. Cohort 2: Induction: 300mg/week X 4 weeks then 600 mg/week x 1 week; Cohort 2: Maintenance: 600mg every 2 weeks Cohort 3: Induction: 600 mg/week x 4 weeks then 900mg/wee x 1 week; Cohort 3: maintenance: 900 mg every 2 weeks
Study Type ^ICMJE	Interventional
Study Phase	Phase 1 Phase 2
Study Design ^ICMJE	Allocation: Non-Randomized Endpoint Classification: Pharmacokinetics/Dynamics Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Condition ^ICMJE	Hemoglobinuria, Paroxysmal
Intervention ^ICMJE	Drug: Eculizumab Eculizumab as an IV infusion. Depending on the age of the patient, induction and maintenance doses vary as follows: Cohort 1 (ages 2-7 years): Induction: 300mg/week x 4 weeks, then 450mg/week x 1 week; Cohort 1: Maintenance: 450mg every 2 weeks for 12 weeks, and a 8 week follow up period Cohort 2: (ages 8 to 11 years)Induction: 300mg/week X 4 weeks then 600 mg/week x 1 week; Cohort 2: Maintenance: 600mg every 2 weeks for 12 weeks and a 8 week follow up period Cohort 3: (ages 12 to 17 years) Induction: 600 mg/week x 4 weeks then 900mg/wee x 1 week; Cohort 3: maintenance: 900 mg every 2 weeks for 12 weeks and a 8 week follow up period.
Study Arm (s)
Publications *
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information
Recruitment Status ^ICMJE	Completed
Enrollment ^ICMJE	7
Completion Date	November 2011
Primary Completion Date	May 2011 (final data collection date for primary outcome measure)
Eligibility Criteria ^ICMJE	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; 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.
Gender	Both
Ages	2 Years to 17 Years
Accepts Healthy Volunteers	No
Contacts ^ICMJE	Contact information is only displayed when the study is recruiting subjects
Location Countries ^ICMJE	United States

Administrative Information
NCT Number ^ICMJE	NCT00867932
Other Study ID Numbers ^ICMJE	M07-005
Has Data Monitoring Committee	No
Responsible Party	Alexion Pharmaceuticals
Study Sponsor ^ICMJE	Alexion Pharmaceuticals
Collaborators ^ICMJE
Investigators ^ICMJE
Information Provided By	Alexion Pharmaceuticals
Verification Date	November 2009
^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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