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

End Stage Renal Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01029587
• Other study ID #: NA_00029904
• Status: Completed
• Phase: Phase 2
• Start date: November 2009
• Est. completion date: May 6, 2010

Study information

• Verified date: October 2020
• Source: Johns Hopkins University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 1
• Est. completion date: May 6, 2010
• Est. primary completion date: May 6, 2010
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Antiphospholipid Antibody Syndrome
• Antiphospholipid Syndrome
• End Stage Renal Disease
• Kidney Failure, Chronic
• Syndrome

Intervention

Drug:
• Eculizumab
Eculizumab will be administered by intravenous infusion. Eculizumab will be administered at a dose of 1200mg by intravenous (IV) infusion on the day of or on the day prior to kidney transplantation, and at a dose of 900mg IV on post-operative day 1. Subsequently, the post-operative dosing regimen would be comprised of an induction phase of weekly doses of 900mg IV per dose followed by a maintenance phase of every other week dosing of 1200mg IV per dose. The weekly induction dosing regimen would begin on postoperative day 8, and would continue for three doses (specifically, doses of 900mg IV would be given on postoperative days 8, 15, and 22). The maintenance phase of dosing would begin with a dose of 1200mg on postoperative day 29, and would continue for a total of 5 doses (specifically, doses of 1200mg IV would be given on postoperative days 29, 43, 47, 72, and 85). In most cases, eculizumab would be discontinued after the 5th maintenance dose.

Locations

Country	Name	City	State
United States	Johns Hopkins University School of Medicine	Baltimore	Maryland

Sponsors (1)

Lead Sponsor	Collaborator
Johns Hopkins University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Patients With Prevention of Catastrophic Antiphospholipid Antibody Syndrome (CAPS) After Kidney Transplant		6 months
Secondary	Number of Patients With Kidney Transplant Graft Survival		6 months
Secondary	Number of Patients Who Survive		6 months