Trial Outcomes & Findings for Human Immune Globulin in Treating Patients With Primary Amyloidosis That is Causing Heart Dysfunction (NCT NCT00547365)

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

10 participants

Primary outcome timeframe

Up to 1 year

Results posted on

2013-09-19

Participant Flow

Overall study length - 2007-2011; Location - medical clinic

Patients with primary light-chain (AL)-associated amyloidosis that caused heart dysfunction were on study.

Participant milestones

Participant milestones
Measure	Human Immune Globulin Intravenous (IGIV) The therapeutic potential of human immune globulin intravenous (IGIV)was evaluated in patients with cardiac-associated AL amyloidosis. Patients received, via intravenous infusion, 30-40 gm of IGIV (depending on body weight) weekly for 3 months and then every other week for the next 9 months.The total time to complete the study was \~1 yr.
Overall Study STARTED	10
Overall Study COMPLETED	2
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Reasons for withdrawal
Measure	Human Immune Globulin Intravenous (IGIV) The therapeutic potential of human immune globulin intravenous (IGIV)was evaluated in patients with cardiac-associated AL amyloidosis. Patients received, via intravenous infusion, 30-40 gm of IGIV (depending on body weight) weekly for 3 months and then every other week for the next 9 months.The total time to complete the study was \~1 yr.
Overall Study Physician Decision	1
Overall Study Lost to Follow-up	1
Overall Study Protocol Violation	2
Overall Study death (not related to study)	4

Baseline Characteristics

Human Immune Globulin in Treating Patients With Primary Amyloidosis That is Causing Heart Dysfunction

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Human Immune Globulin Intravenous (IGIV) n=10 Participants
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=93 Participants
Age, Categorical >=65 years	6 Participants n=93 Participants
Sex: Female, Male Female	5 Participants n=93 Participants
Sex: Female, Male Male	5 Participants n=93 Participants
Region of Enrollment United States	10 participants n=93 Participants

PRIMARY outcome

Timeframe: Up to 1 year

Population: All patients who had at least one infusion of human immune globulin intravenous.

Outcome measures

Outcome measures
Measure	Human Immune Globulin Intravenous (IGIV) n=10 Participants Immune globulin intravenous (IGIV) was administered to patients with cardiac-dominant AL amyloidosis in order to determine its therapeutic potential or possible toxicity when given to subjects weekly for 3 months and then every other week for the next 9 months. Response was evaluated by changes in serum anti-fibril antibody levels, changes in BNP (B-type natriuretic peptide) levels and IVS (interventricular septum) thickness.
Tolerance for Human Immune Globulin Intravenous (IGIV), as Reflected by the Number and Severity of Toxicity Incidents Occurring in Ten Patients Receiving at Least One Infusion of IGIV.	0 events

PRIMARY outcome

Timeframe: Up to 1 year

Population: Two of ten patients with AL cardiac involvement who received IGIV infusions were analyzed (other eight individuals were removed from study before completion due to death/conditions unrelated to IGIV, loss to follow-up, or physician decision).

Positive clinical response was defined by improvement in heart function in participating patients with cardiac-dominant AL amyloidosis, as demonstrated by increased serum anti-fibril immunoglobulin G (IgG) antibody levels and reduction (or no evident progression) in amyloid burden.

Outcome measures

Outcome measures
Measure	Human Immune Globulin Intravenous (IGIV) n=2 Participants Immune globulin intravenous (IGIV) was administered to patients with cardiac-dominant AL amyloidosis in order to determine its therapeutic potential or possible toxicity when given to subjects weekly for 3 months and then every other week for the next 9 months. Response was evaluated by changes in serum anti-fibril antibody levels, changes in BNP (B-type natriuretic peptide) levels and IVS (interventricular septum) thickness.
Clinical Response of Patients With Cardiac-dominant AL Amyloidosis Given Human Immune Globulin Intravenous (IGIV)	1 participants with positive response

Adverse Events

Human Immune Globulin Intravenous (IGIV)

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

Alan Solomon, MD

University of Tennessee Graduate School of Medicine

Phone: 865-305-9167

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place