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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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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

Conditions
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Multiple Myeloma

Intervention / Treatment
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* Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)
* Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)
* Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: 0 mg/kg<br> | Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)<br>* 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<br>|
| Other: 0.5 mg/kg<br> | Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)<br>* 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<br>|
| Other: 1.0 mg/kg<br> | Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)<br>* 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<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of rhMBL |  | 2 months | 

Secondary Outcome Measures

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

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Multiple Myeloma, Stem Cell Transplant, rhMBL