Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients

NCT01192399 | Completed Phase 2 Results FDA Drug

• 1 other identifier: C07-001
• Study Type: interventional
• Target: 29
• Locations: 0 countries
• Sites: N/A

Brief Summary

A study to assess the safety and efficacy of eculizumab in Japanese patients with hemolytic PNH.

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

Hemolytic Paroxysmal Nocturnal Hemoglobinuria; Eculizumab; PNH; Japan

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Lactate Dehydrogenase

  Baseline, Week 12

Secondary Outcomes (6)

• Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Total Score

  Baseline, Week 12

• Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Count

  Baseline, Week 12

• Number of Units of Packed Red Blood Cells (pRBCs) Transfused

  12 weeks pre-treatment, baseline, 12 weeks post-treatment

• Change From Baseline in Lactate Dehydrogenase (LDH) Area Under the Curve (AUC)

  Baseline to Week 12

• Change From Baseline in Plasma Free Hemoglobin

  Baseline, Week 12

Study Arms (1)

Eculizumab

EXPERIMENTAL

Eculizumab intravenous infusions every week x 4 doses, then 900 mg 1 week later for 1 dose, then 900 mg every 2 weeks for 4 doses

Biological: Eculizumab

Interventions

Eculizumab BIOLOGICAL

Also known as: Soliris

Eculizumab

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Japanese individuals at least 12 years of age
• Diagnosis of PNH \> 6 months
• At least one transfusion in the past 2 years for anemia or anemia-related symptoms
• LDH level ≥ 1.5 x upper limit of normal within 12 weeks
• Presence of a glycosylphosphatidylinositol (GPI)-deficient red blood cell (RBC) clone (type III cells) by flow cytometry of ≥10%
• Negative serum pregnancy test for women of child-bearing potential

You may not qualify if:

• Platelet count \< 30,000/µL
• Absolute neutrophil count ≤ 500/µL
• Known or suspected hereditary complement deficiency
• History of hematopoietic stem cell transplant
• History of meningococcal disease

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Related Publications (1)

• Kanakura Y, Ohyashiki K, Shichishima T, Okamoto S, Ando K, Ninomiya H, Kawaguchi T, Nakao S, Nakakuma H, Nishimura J, Kinoshita T, Bedrosian CL, Valentine ME, Khursigara G, Ozawa K, Omine M. Safety and efficacy of the terminal complement inhibitor eculizumab in Japanese patients with paroxysmal nocturnal hemoglobinuria: the AEGIS clinical trial. Int J Hematol. 2011 Jan;93(1):36-46. doi: 10.1007/s12185-010-0748-9. Epub 2011 Jan 12.

  PMID: 21222185 RESULT

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Results Point of Contact

• Title: Alexion Pharmaceuticals, Inc
• Organization: Alexion Pharmaceuticals, Inc

clinicaltrials@alexion.com

Study Officials

• Stephen Squinto, PhD

  Alexion Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2010
• First Posted: September 1, 2010
• Study Start: November 1, 2007
• Primary Completion: June 1, 2008
• Study Completion: June 1, 2008
• Last Updated: March 13, 2018
• Results First Posted: March 13, 2018

Record last verified: 2018-02

Data Sharing

• IPD Sharing: Will not share