Clinical trial NCT00547365: Human Immune Globulin in Treating Patients With Primary Amyloidosis That is Causing Heart Dysfunction
Official title: Therapeutic Potential of Human Immune Globulin Intravenous (IGIV) in Patients With Cardiac-Associated Light Chain (AL) Amyloidosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Multiple Myeloma
Plasma Cell Neoplasm
Keywords: primary systemic amyloidosis
Lead sponsor: University of Tennessee
Sponsor class: OTHER
First posted: 2007-10-22
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: RATIONALE: Antibodies, such as human immune globulin, can block the growth of abnormal cells in different ways. Some block the ability of abnormal cells to grow and spread. Others find abnormal cells and help kill them or carry cell-killing substances to them. Giving human immune globulin may be effective in treating patients with primary amyloidosis that is causing heart dysfunction.

PURPOSE: This phase I/II trial is studying the side effects and best dose of human immune globulin and to see how well it works in treating patients with primary amyloidosis that is causing heart dysfunction.

Detailed description: OBJECTIVES:

* Establish the maximum tolerated dose of human immune globulin intravenous (IGIV) given weekly for the first 3 months and then bi-weekly for 9 additional months in patients with cardiac-associated primary light chain-associated (AL) amyloidosis.
* Determine the safety, pharmokinetics, and therapeutic efficacy as evidenced by titers of serum fibril-reactive immunoglobulin G (IgG) antibodies pre- and post-IGIV infusions.
* Demonstrate stable or improved organ function.

OUTLINE: Patients receive human immune globulin IV (IGIV) once weekly for 3 months and then once biweekly for 9 months, for a total of 12 months in the absence of disease progression or unacceptable toxicity.

Patients undergo blood sample collection to measure serum anti-fibril antibody titers pre- and post- IGIV infusion for assessing safety and response to treatment.

Eligibility criteria: Inclusion criteria:

* Confirmed diagnosis of cardiac-associated primary (AL) amyloidosis based on accepted clinical and laboratory criteria
* Patients must have heart involvement as evidenced by elevated serum brain natriuretic peptide (BNP), troponin levels, and/or 2D echocardiography evidence of a thickened intraventricular septum (IVS).
* Life expectancy \> 3 months
* Prior or concurrent chemotherapy or other drug-based anti-AL regimes allowed

Exclusion criteria:

* Non-AL amyloidosis
* New York Heart Association (NYH) class IV heart disease
* Significant comorbidity (e.g., uncontrolled infection, diabetes, or other serious illnesses)

Primary outcomes:
measure: 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.
timeFrame: Up to 1 year
measure: Clinical Response of Patients With Cardiac-dominant AL Amyloidosis Given Human Immune Globulin Intravenous (IGIV)
timeFrame: Up to 1 year
Secondary outcomes:
Other outcomes:
Locations:
city: Knoxville
country: United States
facility: Baptist Regional Cancer Center at Baptist Riverside
state: Tennessee
city: Powell
country: United States
facility: St. Mary's Medical Center
state: Tennessee
Study officials:
affiliation: St. Mary's Medical Center
name: Alan Solomon, MD
role: STUDY_CHAIR
References: