Trial Outcomes & Findings for Renal AL Amyloid Involvement and NEOD001 (NCT NCT03168906)

NCT ID: NCT03168906

Last Updated: 2020-09-09

Results Overview

A renal response is a ≥ 30% reduction in proteinuria in the absence of a ≥ 25% decrease in eGFR. A confirmed renal response is one that has been documented as present one month after 12 monthly treatments.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Baseline to 13 Months

Results posted on

2020-09-09

Participant Flow

Participant milestones

Participant milestones
Measure	NEOD001 Study Drug given IV every 28 days at 24mg/kg NEOD001: NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates	Placebo Placebo Placebo: Saline bag
Overall Study STARTED	6	6
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	6	6

Reasons for withdrawal

Reasons for withdrawal
Measure	NEOD001 Study Drug given IV every 28 days at 24mg/kg NEOD001: NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates	Placebo Placebo Placebo: Saline bag
Overall Study Lack of Efficacy	6	6

Baseline Characteristics

Renal AL Amyloid Involvement and NEOD001

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	NEOD001 n=6 Participants Study Drug given IV every 28 days at 24mg/kg NEOD001: NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates	Placebo n=6 Participants Placebo Placebo: Saline bag	Total n=12 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants	5 Participants n=7 Participants	9 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Age, Continuous	60 years n=5 Participants	54.5 years n=7 Participants	57.5 years n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants	4 Participants n=7 Participants	8 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants	5 Participants n=7 Participants	10 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to 13 Months

Population: Trial was closed prior to completion, therefore data were not collected.

A renal response is a ≥ 30% reduction in proteinuria in the absence of a ≥ 25% decrease in eGFR. A confirmed renal response is one that has been documented as present one month after 12 monthly treatments.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline

Population: Trial was closed prior to completion, therefore data were not collected.

The aim of this study is to assess the performance of CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) creatinine in comparison to iothalamate clearance measured in AL amyloidosis patients. Iothalamate will be given subcutaneously. Urine and plasma samples will then be obtained. All laboratory tests for samples obtained for GFR measurement will be performed at Mayo Clinic Rochester. Quantification of iothalamate in urine and plasma will be performed using a tandem mass spectrometric method.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to 13 Months

Population: Trial was closed prior to completion, therefore data were not collected.

Months to Chronic Kidney Disease level 4 or 5

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to 13 Months

Population: Trial was closed prior to completion, therefore data were not collected.

Months to estimated Glomerular Filtration Rate ≤ 15 or dialysis

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to 13 Months

Population: Trial was closed prior to completion, therefore data were not collected.

Months to doubling of serum creatinine

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to 13 Months

Population: Trial was closed prior to completion, therefore data were not collected.

Months to ≥ 40% reduction in estimated glomerular filtration rate

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to 26 months

Population: Trial was closed prior to completion, therefore data were not collected.

A renal response is a ≥ 30% decrease in proteinuria or drop of proteinuria below 0.5 g/24h in the absence of renal progression.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline to 26 months

Population: Trial was closed prior to completion, therefore data were not collected.

Death at 26 months from Baseline due to any cause

Outcome measures

Outcome data not reported

Adverse Events

NEOD001

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	NEOD001 n=6 participants at risk Study Drug given IV every 28 days at 24mg/kg NEOD001: NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates	Placebo n=6 participants at risk Placebo Placebo: Saline bag
General disorders Chest pain	16.7% 1/6 • Number of events 1 • 1 month	0.00% 0/6 • 1 month
Nervous system disorders Left arm pain	16.7% 1/6 • Number of events 1 • 1 month	0.00% 0/6 • 1 month
Musculoskeletal and connective tissue disorders Right hip pain	16.7% 1/6 • Number of events 1 • 1 month	0.00% 0/6 • 1 month
General disorders Fatigue	33.3% 2/6 • Number of events 2 • 1 month	0.00% 0/6 • 1 month
Injury, poisoning and procedural complications Concussion	16.7% 1/6 • Number of events 1 • 1 month	0.00% 0/6 • 1 month
Renal and urinary disorders Dysuria	16.7% 1/6 • Number of events 1 • 1 month	0.00% 0/6 • 1 month
Gastrointestinal disorders Nausea	16.7% 1/6 • Number of events 1 • 1 month	0.00% 0/6 • 1 month
General disorders Upper and lower extremity swelling	0.00% 0/6 • 1 month	16.7% 1/6 • Number of events 1 • 1 month
Gastrointestinal disorders Constipation	0.00% 0/6 • 1 month	16.7% 1/6 • Number of events 1 • 1 month
Respiratory, thoracic and mediastinal disorders Coughing spells	0.00% 0/6 • 1 month	16.7% 1/6 • Number of events 1 • 1 month

Additional Information

Jaime Chisholm

Tufts Medical Center

Phone: 617-636-5409

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place