Eculizumab Therapy for Dense Deposit Disease and C3 Nephropathy
This study is ongoing, but not recruiting participants.
Sponsor:
Columbia University
Collaborator:
Alexion Pharmaceuticals
Information provided by (Responsible Party):
Gerald B. Appel, Columbia University
ClinicalTrials.gov Identifier:
NCT01221181
First received: October 13, 2010
Last updated: August 1, 2012
Last verified: August 2012
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Purpose
This is an investigator initiated, single site study to determine whether eculizumab, a drug approved by the FDA for another indication, is a safe and effective treatment for dense deposit disease (DDD) and C3 nephropathy.
All enrolled subjects will receive eculizumab treatment for one year. There will be 29 study visits over 53 weeks. The goal is to determine whether this treatment will improve kidney function, as evidenced by less protein in the urine and improved lab results.
An EXTENSION TREATMENT PHASE has been added. After completing the study, patients are followed with labs every four weeks as per standard of care. If labs suggest a relapse, they will be allowed to continue on therapy at the previous dosage until this drug receives FDA approval for this indication. Treatment intervals and dosage are identical to the original study.
| Condition | Intervention | Phase |
|---|---|---|
|
Dense Deposit Disease Membranoproliferative Glomerulonephritis |
Drug: Eculizumab |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Eculizumab Therapy for Dense Deposit Disease and C3 Nephropathy |
Resource links provided by NLM:
Genetics Home Reference related topics:
dense deposit disease
Drug Information available for:
Eculizumab
U.S. FDA Resources
Further study details as provided by Columbia University:
Primary Outcome Measures:
- Complete remission [ Time Frame: after 1 year of therapy ] [ Designated as safety issue: No ]The primary end point is complete remission, defined as remission of proteinuria to <500 mg/day with normal renal function.
Secondary Outcome Measures:
- Partial remission [ Time Frame: after 1 year of therapy ] [ Designated as safety issue: No ]Partial remission, a secondary endpoint, will be defined as reduction in proteinuria by at least 50% and to <2 g/day with stable renal function (i.e. no more than a 20% increase in serum creatinine).
- Improvement in histologic parameters [ Time Frame: after 1 year of therapy ] [ Designated as safety issue: No ]Improvement in histologic parameters, including percentage of non-affected glomeruli, interstitial fibrosis, intensity of C3 staining of immunoflourescence, and amount of electron dense deposits by electron microscopy.
| Estimated Enrollment: | 6 |
| Study Start Date: | August 2010 |
| Estimated Study Completion Date: | December 2013 |
| Estimated Primary Completion Date: | August 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Eculizumab
Eculizumab 900 mg IV one a week for 4 weeks, 1200 mg IV week 5, then 1200 mg IV every 2 weeks through week 53.
|
Drug: Eculizumab
900 mg IV once a week x 4 weeks, 1200 mg IV week 5, then 1200 mg IV every 2 weeks through week 53
Other Name: Eculizumab (Soliris®)
|
Show Detailed Description Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Adult patients with biopsy proven DDD or C3 nephropathy, at least 18 years of age
- 24-hour urine protein > 1000 mg/day, urine protein:creatinine ratio > 1.0, or acute renal failure (defined as > 50% increase in serum creatinine from baseline)
- Willing and able to sign informed consent
- Patients of childbearing age must agree to use birth control
- Patients must be willing to be vaccinated against meningococcal disease or have documentation of previous vaccination against meningococcal disease
Exclusion Criteria:
- Patients under 18 years of age
- Patients unable to sign informed consent
- Patients having received rituximab or another monoclonal antibody within 6 months of the trial
- Patients currently taking and unable to discontinue other immunomodulatory therapies (e.g. cyclosporine, high-dose steroids, mycophenolate mofetil) unless these other therapies are indicated for prophylaxis against transplant rejection (e.g. stable doses of mycophenolate mofetil and/or calcineurin inhibitor). Patients on chronic steroid therapy who are unable to taper down to <10 mg/day will be excluded.
- Patients of childbearing age who refuse to use birth control
- Patients with a baseline eGFR less than 30 ml/min/1.73m2
- Patients with other renal diseases (e.g. diabetic nephropathy, renal vascular disease) that would interfere with interpretation of the study.
- Patients with comorbid conditions that would interfere with completion of the trial (malignancies, CHF, recent myocardial infarction).
- Patients with known contraindications to the use of eculizumab, including refusal to receive N. meningitidis vaccine prior to therapy
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01221181
Locations
| United States, New York | |
| Columbia University Medical Center, Nephrology Clinical Research Center | |
| New York, New York, United States, 10032 | |
| Columbia University Medical Center, Glomerular Center | |
| New York, New York, United States, 10032 | |
Sponsors and Collaborators
Columbia University
Alexion Pharmaceuticals
Investigators
| Principal Investigator: | Gerald B Appel, MD | Columbia University |
More Information
No publications provided
| Responsible Party: | Gerald B. Appel, Professor of Clinical Medicine, Nephrology, Columbia University |
| ClinicalTrials.gov Identifier: | NCT01221181 History of Changes |
| Other Study ID Numbers: | AAAF2403 |
| Study First Received: | October 13, 2010 |
| Last Updated: | August 1, 2012 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by Columbia University:
|
Dense deposit disease C3 nephropathy Eculizumab |
Additional relevant MeSH terms:
|
Glomerulonephritis Glomerulonephritis, Membranoproliferative Kidney Diseases |
Nephritis Urologic Diseases Immune System Diseases |
ClinicalTrials.gov processed this record on May 21, 2013