Trial Outcomes & Findings for Eculizumab Therapy for Dense Deposit Disease and C3 Nephropathy (NCT NCT01221181)

Last Updated: 2019-02-19

Results Overview

This is designed to measure response to eculizumab through clinical and histological data, including a reduction in serum creatinine or in proteinuria, or histopathologic improvement. Any reduction in serum creatinine and/or proteinuria was included in our descriptive analysis.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

6 participants

Primary outcome timeframe

One year

Results posted on

2019-02-19

Participant Flow

Participant milestones

Participant milestones
Measure	Eculizumab Patients will receive Eculizumab and be observed for 60 minutes after the first 5 infusions, then 30 minutes after all subsequent infusions.
Overall Study STARTED	6
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Eculizumab Patients will receive Eculizumab and be observed for 60 minutes after the first 5 infusions, then 30 minutes after all subsequent infusions.
Overall Study Lost to Follow-up	1

Baseline Characteristics

Eculizumab Therapy for Dense Deposit Disease and C3 Nephropathy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Eculizumab n=6 Participants Patients will receive Eculizumab and be observed for 60 minutes after the first 5 infusions, then 30 minutes after all subsequent infusions.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	23.5 years n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Creatinine	1.75 mg/dl n=5 Participants

PRIMARY outcome

Timeframe: One year

Outcome measures

Outcome measures
Measure	Eculizumab n=6 Participants 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. 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. Patients will be observed for 60 minutes after the first 5 infusions, then 30 minutes after all subsequent infusions. Patients will not be allowed to take other immunomodulatory therapies during the study period but will continue on their other non-immunomodulatory therapies (e.g. ACE inhibitors, -statins, aspirin) without modifications unless clinically indicated. All patients, if unvaccinated, will be given N. meningitidis vaccine at least two weeks prior to first eculizumab exposure. All female patients of childbearing potential will be asked to use adequate contraception methods during treatment and up to 5 months following discontinuation of eculizumab treatment.
Number of Patients With Change in Proteinuria or Serum Creatinine Over Treatment Period	5 Participants

Adverse Events

Eculizumab

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Andrew Bomback, MD, MPH

Columbia University Medical Center

Phone: 212-305-0320

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place