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

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ClinicalTrials.gov Identifier: NCT01029587
Recruitment Status : Completed
First Posted : December 10, 2009
Results First Posted : October 26, 2020
Last Update Posted : October 26, 2020
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Antiphospholipid Antibody Syndrome End Stage Renal Disease Drug: Eculizumab Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Eculizumab to Enable Renal Transplantation in Patients With History of Antiphospholipid Antibody Syndrome or Catastrophic Antiphospholipid Antibody Syndrome
Actual Study Start Date : November 2009
Actual Primary Completion Date : May 6, 2010
Actual Study Completion Date : May 6, 2010

Arm Intervention/treatment
Experimental: Eculizumab
Patients will receive eculizumab in conjunction with systemic anticoagulation before and after kidney transplant operation
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.
Other Name: Soliris

Primary Outcome Measures :
  1. Number of Patients With Prevention of Catastrophic Antiphospholipid Antibody Syndrome (CAPS) After Kidney Transplant [ Time Frame: 6 months ]

Secondary Outcome Measures :
  1. Number of Patients With Kidney Transplant Graft Survival [ Time Frame: 6 months ]
  2. Number of Patients Who Survive [ Time Frame: 6 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01029587

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United States, Maryland
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
Sponsors and Collaborators
Johns Hopkins University
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Principal Investigator: Benjamin Philosophe, MD, PHD Johns Hopkins University
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Responsible Party: Johns Hopkins University
ClinicalTrials.gov Identifier: NCT01029587    
Other Study ID Numbers: NA_00029904
First Posted: December 10, 2009    Key Record Dates
Results First Posted: October 26, 2020
Last Update Posted: October 26, 2020
Last Verified: October 2020
Keywords provided by Johns Hopkins University:
Kidney transplant
Renal transplant
Catastrophic Antiphospholipid Antibody Syndrome
Additional relevant MeSH terms:
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Kidney Failure, Chronic
Antiphospholipid Syndrome
Pathologic Processes
Kidney Diseases
Urologic Diseases
Renal Insufficiency, Chronic
Renal Insufficiency
Autoimmune Diseases
Immune System Diseases
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs