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

NCT01029587 | Completed Phase 2 Results

• 1 other identifier: NA_00029904
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

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.

Conditions

Antiphospholipid Antibody Syndrome; End Stage Renal Disease

Keywords

CAPS; Kidney transplant; Renal transplant; Catastrophic Antiphospholipid Antibody Syndrome

Outcome Measures

Primary Outcomes (1)

• Number of Patients With Prevention of Catastrophic Antiphospholipid Antibody Syndrome (CAPS) After Kidney Transplant

  6 months

Secondary Outcomes (2)

• Number of Patients With Kidney Transplant Graft Survival

  6 months

• Number of Patients Who Survive

  6 months

Study Arms (1)

Eculizumab

EXPERIMENTAL

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

Drug: Eculizumab

Interventions

Eculizumab DRUG

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.

Also known as: Soliris

Eculizumab

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Johns Hopkins University: lead

Study Sites (1)

Johns Hopkins University School of Medicine

Baltimore, Maryland, 21205, United States

Location

MeSH Terms

Conditions

Antiphospholipid Syndrome; Kidney Failure, Chronic

Interventions

eculizumab

Condition Hierarchy (Ancestors)

Autoimmune Diseases; Immune System Diseases; Renal Insufficiency, Chronic; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Benjamin Philosophe
• Organization: Johns Hopkins University

bphilosophe@jhmi.edu

443-287-7178

Study Officials

• Benjamin Philosophe, MD, PHD

  Johns Hopkins University

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

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

Study Record Dates

• First Submitted: December 9, 2009
• First Posted: December 10, 2009
• Study Start: November 1, 2009
• Primary Completion: May 6, 2010
• Study Completion: May 6, 2010
• Last Updated: October 26, 2020
• Results First Posted: October 26, 2020

Record last verified: 2020-10

Locations

US (1)