Eculizumab to Enable Renal Transplantation in Patients With History of Catastrophic Antiphospholipid Antibody Syndrome

Phase 2

Completed

• Conditions: Antiphospholipid Antibody Syndrome; End Stage Renal Disease
• Interventions: Drug: Eculizumab

• Registration Number: NCT01029587
• Lead Sponsor: Johns Hopkins University

Brief Summary

Catastrophic Antiphospholipid Antibody Syndrome (CAPS) is a rare condition in which life-threatening blood clots form in multiple organs simultaneously and can lead to multi-organ system failure and death. The causes of CAPS are not entirely understood, but CAPS episodes are often triggered by stressful events such as infections, surgery, or trauma. For patients who survive an episode of CAPS, permanent kidney failure is not uncommon because the kidneys are the organ system most frequently affected in CAPS. Kidney transplantation is the treatment of choice for end-stage kidney disease, but patients with a history of CAPS are exceptionally high-risk kidney transplant recipients because the chance that surgery itself could trigger a life-threatening or transplant-threatening episode of CAPS is significant. As a result, patients with CAPS are not generally considered candidates for transplantation. Despite this, these patients have a severely decreased life-expectancy on dialysis and their long-term survival and quality of life would be greatly increased by a successful kidney transplant. In this trial, a drug called eculizumab will be tested for its ability to prevent CAPS after kidney transplantation in patients with a prior history of CAPS. Eculizumab is an inhibitor of the complement system, which is believed to be important in generating the inflammatory environment that leads to diffuse clotting of blood vessels in CAPS. The investigators hypothesize that by blocking the complement cascade using eculizumab, in conjunction with blocking the coagulation system, that kidney transplantation can be safely and successfully performed in patients with a history of CAPS.

Detailed Description

Not available

Study Timeline

• Study Start: 2009-11
• Study First Submitted: 2009-12-09
• Study First Submitted QC: 2009-12-09
• Study First Posted: 2009-12-10
• Primary Completion: 2010-05-06
• Study Completion: 2010-05-06
• Status Verified: 2020-10
• Results First Submitted: 2020-10-01
• Results First Submitted QC: 2020-10-01
• Last Update Submitted: 2020-10-01
• Results First Posted: 2020-10-26
• Last Update Posted: 2020-10-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 1

Inclusion Criteria

• Age 18 years or older
• History of Catastrophic Antiphospholipid Antibody Syndrome (CAPS)
• End-stage renal disease

Exclusion Criteria

• Any contraindications to transplantation other than CAPS
• Pregnant women
• Women who intend to become pregnant over the study period
• Ongoing or untreated meningococcal infections
• History of serious adverse reaction to eculizumab

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Eculizumab	Eculizumab	Patients will receive eculizumab in conjunction with systemic anticoagulation before and after kidney transplant operation

Primary Outcome Measures

Name	Time	Method
Number of Patients With Prevention of Catastrophic Antiphospholipid Antibody Syndrome (CAPS) After Kidney Transplant	6 months

Secondary Outcome Measures

Name	Time	Method
Number of Patients Who Survive	6 months
Number of Patients With Kidney Transplant Graft Survival	6 months

Trial Locations

Locations (1)

Johns Hopkins University School of Medicine; 🇺🇸 Baltimore, Maryland, United States