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

Johns Hopkins University

Status and phase: Completed; Phase 2

Conditions: Antiphospholipid Antibody Syndrome; End Stage Renal Disease

Treatments: Drug: Eculizumab

Study type: Interventional

Funder types: Other

Identifiers

NCT01029587

NA_00029904

Details and patient eligibility

About

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.

Enrollment: 1 patient

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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1 participants in 1 patient group

Eculizumab

Experimental group

Description:

Patients will receive eculizumab in conjunction with systemic anticoagulation before and after kidney transplant operation

Treatment:

Drug: Eculizumab