Intravenous rhMBL in Patients With Multiple Myeloma Receiving Chemotherapy Followed by Stem Cell Transplant

Multi-Center,Open-Label,Randomized,Phase 1B Study Evaluating Safety & Tolerability of Intravenous rhMBL in Pts With Multiple Myeloma Receiving Melphalan-Based High-Dose Chemotherapy Followed by Autologous Hematopoietic Stem Cell Transplant

MBL deficient patients enrolled in this protocol are scheduled to be treated with melphalan-based high-dose chemotherapy followed by autologous hematopoietic stem cell transplant (HSCT) for their multiple myeloma. Patients are randomized to 0.5 mg/kg, 1.0 mg/kg, or no rhMBL.

Study Overview

• Status: Completed
• Conditions: Multiple Myeloma
• Intervention / Treatment: Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)

Detailed Description

MBL deficient patients will be randomized in a ratio of 2:2:1 to receive up to 4 weekly i.v. infusions of rhMBL at a dose of 0.5 mg/kg or 1.0 mg/kg, or standard anti-infectious prophylactic therapy alone. A total of 20 patients will be treated in each of the rhMBL arms, and 10 patients will receive best standard supportive prophylactic therapy but not rhMBL. All patients are to receive anti-infectious prophylactic supportive therapy as per institutional standards.

• Study Type: Interventional
• Enrollment (Actual): 46
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • University of Arkansas for Medical Sciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Major Inclusion Criteria: Patients must meet all of the following criteria to be eligible for enrollment into the study:

• Capable of understanding the protocol requirements and risks and providing written informed consent.
• Histologically or cytologically confirmed diagnosis of multiple myeloma.
• Mannose-binding lectin level <300 ng/mL.
• Age ≥18 years old.
• Score of 0 to 2 on the Zubrod performance status scale.
• Patient is scheduled to receive melphalan-based high-dose chemotherapy and autologous HSCT for the treatment of multiple myeloma.

Exclusion Criteria:

• Concurrent serious medical illness that could potentially interfere with protocol compliance.
• Concurrent or previous malignancy associated with a poor prognosis.
• Known chronic infectious disease, such as acquired immunodeficiency syndrome (AIDS) or hepatitis (for hepatitis and human immunodeficiency virus [HIV] will not be performed).
• Positive screening pregnancy test or is breast-feeding.
• Female or male patient of reproductive capacity unwilling to use methods appropriate to prevent pregnancy during the course of this protocol.
• Known or clinically suspected active brain metastases.
• Current participation in another clinical study with an investigational agent and/or use of an investigational drug.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
OTHER: 0 mg/kg	Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL); Intravenous (i.v.) administration for 4 weeks. Patients will be randomized to one of three cohorts: 0 mg/kg; 0.5 mg/kg, or 1.0 mg.kg
OTHER: 0.5 mg/kg	Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL); Intravenous (i.v.) administration for 4 weeks. Patients will be randomized to one of three cohorts: 0 mg/kg; 0.5 mg/kg, or 1.0 mg.kg
OTHER: 1.0 mg/kg	Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL); Intravenous (i.v.) administration for 4 weeks. Patients will be randomized to one of three cohorts: 0 mg/kg; 0.5 mg/kg, or 1.0 mg.kg

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Safety and tolerability of rhMBL	2 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Pharmacokinetic (PK) of rhMBL	2 months
Pharmacodynamics (PD) of rhMBL	2 months
Immunogenicity of rhMBL, incidence of infectious complications	2 months

Collaborators and Investigators

• Sponsor: Enzon Pharmaceuticals, Inc.
• Investigators: Principal Investigator: Elias Anaissie, MD, University of Arkansas

Study record dates

Study Major Dates

• Study Start: September 1, 2006
• Primary Completion (ACTUAL): February 1, 2009
• Study Completion (ACTUAL): May 1, 2009

Study Registration Dates

• First Submitted: October 13, 2006
• First Submitted That Met QC Criteria: October 13, 2006
• First Posted (ESTIMATE): October 17, 2006

Study Record Updates

• Last Update Posted (ESTIMATE): December 5, 2011
• Last Update Submitted That Met QC Criteria: December 2, 2011
• Last Verified: December 1, 2011

More Information

Terms related to this study

• Keywords: Multiple Myeloma; Stem Cell Transplant; rhMBL
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Immunoproliferative Disorders; Hematologic Diseases; Hemorrhagic Disorders; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Multiple Myeloma; Neoplasms, Plasma Cell; Physiological Effects of Drugs; Immunologic Factors; Coagulants; Agglutinins; Lectins
• Other Study ID Numbers: EZN-2232-01