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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 0 mg/kg | Other |
| |
| 0.5 mg/kg | Other |
| |
| 1.0 mg/kg | Other |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) | Drug | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability of rhMBL | 2 months |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetic (PK) of rhMBL | 2 months | |
| Pharmacodynamics (PD) of rhMBL | 2 months | |
| Immunogenicity of rhMBL, incidence of infectious complications |
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Major Inclusion Criteria: Patients must meet all of the following criteria to be eligible for enrollment into the study:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Elias Anaissie, MD | University of Arkansas | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205 | United States |
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| Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) | Drug | 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 |
|
| Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) | Drug | 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 |
|
| 2 months |
| ID | Term |
|---|---|
| D009101 | Multiple Myeloma |
| ID | Term |
|---|---|
| D054219 | Neoplasms, Plasma Cell |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D020141 | Hemostatic Disorders |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D010265 | Paraproteinemias |
| D001796 | Blood Protein Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006474 | Hemorrhagic Disorders |
| D008232 | Lymphoproliferative Disorders |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
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