Clinical trial NCT00388999: Intravenous rhMBL in Patients With Multiple Myeloma Receiving Chemotherapy Followed by Stem Cell Transplant
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Multiple Myeloma
Keywords: Multiple Myeloma
Stem Cell Transplant
rhMBL
Lead sponsor: Enzon Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2006-10-17
Enrollment: 46
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility 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.

Primary outcomes:
measure: Safety and tolerability of rhMBL
timeFrame: 2 months
Secondary outcomes:
measure: Pharmacokinetic (PK) of rhMBL
timeFrame: 2 months
measure: Pharmacodynamics (PD) of rhMBL
timeFrame: 2 months
measure: Immunogenicity of rhMBL, incidence of infectious complications
timeFrame: 2 months
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
Study officials:
affiliation: University of Arkansas
name: Elias Anaissie, MD
role: PRINCIPAL_INVESTIGATOR
References: