Clinical trial NCT01029587: Eculizumab to Enable Renal Transplantation in Patients With History of Catastrophic Antiphospholipid Antibody Syndrome
Official title: Eculizumab to Enable Renal Transplantation in Patients With History of Antiphospholipid Antibody Syndrome or Catastrophic Antiphospholipid Antibody Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Antiphospholipid Antibody Syndrome
End Stage Renal Disease
Keywords: CAPS
Kidney transplant
Renal transplant
Catastrophic Antiphospholipid Antibody Syndrome
Lead sponsor: Johns Hopkins University
Sponsor class: OTHER
First posted: 2009-12-10
Enrollment: 1
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of Patients With Prevention of Catastrophic Antiphospholipid Antibody Syndrome (CAPS) After Kidney Transplant
timeFrame: 6 months
Secondary outcomes:
measure: Number of Patients With Kidney Transplant Graft Survival
timeFrame: 6 months
measure: Number of Patients Who Survive
timeFrame: 6 months
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Johns Hopkins University School of Medicine
state: Maryland
Study officials:
affiliation: Johns Hopkins University
name: Benjamin Philosophe, MD, PHD
role: PRINCIPAL_INVESTIGATOR
References: