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

• ClinicalTrials.gov Identifier: NCT00388999
• Recruitment Status: Completed
• First Posted: October 17, 2006
• Last Update Posted: December 5, 2011
• Sponsor: Enzon Pharmaceuticals, Inc.
• Information provided by: Enzon Pharmaceuticals, Inc.

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Multiple Myeloma	Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)	Phase 1

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 46 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: 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
• Study Start Date: September 2006
• Actual Primary Completion Date: February 2009
• Actual Study Completion Date: May 2009

Arms and Interventions

Arm	Intervention/treatment
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
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
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

Outcome Measures

Primary Outcome Measures :

1. Safety and tolerability of rhMBL [ Time Frame: 2 months ]

Secondary Outcome Measures :

1. Pharmacokinetic (PK) of rhMBL [ Time Frame: 2 months ]
2. Pharmacodynamics (PD) of rhMBL [ Time Frame: 2 months ]
3. Immunogenicity of rhMBL, incidence of infectious complications [ Time Frame: 2 months ]

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Locations

United States, Arkansas

University of Arkansas for Medical Sciences

Little Rock, Arkansas, United States, 72205

Sponsors and Collaborators

Enzon Pharmaceuticals, Inc.

Investigators

• Principal Investigator: Elias Anaissie, MD; University of Arkansas

More Information

• Responsible Party: Nazish Huq, Clinical Project Manager, Enzon Pharmaceuticals, Inc
• ClinicalTrials.gov Identifier: NCT00388999
• Other Study ID Numbers: EZN-2232-01
• First Posted: October 17, 2006
• Last Update Posted: December 5, 2011
• Last Verified: December 2011

Keywords provided by Enzon Pharmaceuticals, Inc.:

Multiple Myeloma; Stem Cell Transplant; rhMBL

Additional relevant MeSH terms:

Multiple Myeloma; Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases