CancerPhase 1NCT00520325
A Study of Safety, Tolerability, and Pk of rhMBL in Pediatric Hematology/Oncology Pts With Fever and Neutropenia
A Phase 1 study testing Intravenous recombinant human mannose binding lectin in people with Cancer and Hematologic Diseases, run by Enzon Pharmaceuticals, Inc..
Trial summary
The goal of this clinical research study is to find the dose of EZN-2232 that can be given to MBL deficient pediatric cancer patients undergoing chemotherapy. The pharmacokinetics, pharmacodynamics, and safety of the study drug will also be studied. Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Cancer, Hematologic Diseases, Fever
- What is being tested?
- Intravenous recombinant human mannose binding lectin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jun 2007to Aug 2009
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 17 years
- All sexes
- No healthy volunteers
- Child
Inclusion Criteria: Patients must meet all of the following criteria to be eligible for enrollment into the study:
- Children ages 2 to 17 receiving chemotherapy for hematological/oncological diseases.
- Patients must have documented MBL levels <300 ng/mL in serum as evaluated in the screening protocol.
- Fever defined as an oral temperature equivalent of >100.4°F (38°C).
- Neutropenia defined as an absolute neutrophil count ≤1,000/mm3 with the anticipation that the counts will fall below 500/mm3.
- Receiving broad spectrum antibiotics for fever and neutropenia.
- Patients with age-adjusted normal serum creatinine OR a creatinine clearance >60 mL/min/1.73m2.
- Informed consent of the patient, parent or legally authorized representative obtained prior to entry.
- Reliable and willing to make themselves available for the duration of the study and to abide by the study restrictions.
Exclusion Criteria: Patients meeting any of the following exclusion criteria will not be eligible for enrollment.
- Patients receiving the preparative regimen for a bone marrow or hematopoietic stem cell transplantation.
- Concurrent serious illness in the opinion of the principal investigator that could potentially interfere with protocol compliance.
- Patient is pregnant, breast feeding or planning a pregnancy during the course of the study.
- Sexually active male and female patients not using an acceptable barrier method of contraception or practicing abstinence throughout the study and for at least 30 days after receiving their last treatment unless the patient is surgically or medically sterile.
- Patients with moderate or severe liver disease, as defined by:
AST or ALT >5 times upper limit of normal (ULN) OR Total bilirubin >2.5 times ULN.
- Patients with poor venous access that would preclude intravenous drug delivery or multiple blood draws.
- Current participation in another clinical study with an investigational agent and/or use of an investigational drug (not including investigational use of an FDA approved drug) in the 30 days before scheduled administration of rhMBL.
- Known allergic reactions to MBL or other human plasma products.
- Patients on hemodialysis unable to tolerate the volume of IV fluid on non-dialysis days.
About this study
Official title: A Multi-Center Study of the Safety, Tolerability, Pharmacokinetics, and Dose Escalation of Intravenous Recombinant Human Mannose-Binding-Lectin (rhMBL) in MBL Deficient Pediatric Hematology/Oncology Patients With Fever and Neutropenia
The goal of this clinical research study is to find the dose of EZN-2232 that can be given to MBL deficient pediatric cancer patients undergoing chemotherapy. The pharmacokinetics, pharmacodynamics, and safety of the study drug will also be studied.
Read the detailed description
MBL deficient patients will be randomized in a ratio of 1:1 to receive a single dose of either 0.5 mg/kg or 1.0 mg/kg of intravenous rhMBL. A total of 24 patients will be treated in each of the rhMBL arms, (12 in the 0.5 mg/kg and 12 in the 1.0 mg/kg. All patients are to receive anti-infectious prophylactic supportive therapy as per institutional standards
- Conditions
- Cancer, Hematologic Diseases, Fever, Neutropenia
- Being tested
- Intravenous recombinant human mannose binding lectin
- Keywords
- MBL deficient oncologic hematologic pediatric fever neutropenia
- Medical subject headings
- Neoplasms, Hematologic Diseases, Fever, Neutropenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravenous recombinant human mannose binding lectinDrug
Intravenous(i.v.) administration for one dose. 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 lectinDrug
Intravenous(i.v.) administration for one dose. Patients will be randomized to one of three cohorts: 0 mg/kg; 0.5 mg/kg, or 1.0 mg.kg
| Study group | Role | What they receive |
|---|---|---|
| 0.5 mg/kg | Other | Not described Receives: Drug: Intravenous recombinant human mannose binding lectin |
| 1.0 mg/kg | Other | Not described Receives: Drug: Intravenous recombinant human mannose binding lectin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the safety and tolerability of intravenous rhMBL administered at dosages of 0.5 and 1.0 mg/kg to MBL deficient pediatric hematology/oncology patients during episodes of fever and neutropenia.
Measured: 1 month
Secondary outcomes (1)
Determine the PK profile of intravenous rhMBL as a single dose escalation cohort design in MBL deficient pediatric oncology patients during episodes of fever and neutropenia.
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Aug 2007 (estimated)
- Study startJun 2007
- Primary completionJun 2009
- Study completionAug 2009
Why it stopped: Sponsor decision to terminate the study
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CancerStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Enzon Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Thomas Walsh, MD, Principal investigator, NIH/National Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00520325
- Sponsor's protocol ID
- EZN-2232-03
- Other IDs
- NCI # 07-C-0027
- Status last verified
- Feb 2009
- First submitted
- 23 Aug 2007
- Last updated
- 5 Sep 2012
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center Study of the Safety, Tolerability, Pharmacokinetics, and Dose Escalation of Intravenous Recombinant Human Mannose-Binding-Lectin (rhMBL) in MBL Deficient Pediatric Hematology/Oncology Patients With Fever and Neutropenia
- ClinicalTrials.gov ID
- NCT00520325
- Acronym / sponsor ID
- EZN-2232-03
- Conditions
- Cancer, Hematologic Diseases, Fever, Neutropenia
- Keywords
- MBL deficient oncologic hematologic pediatric fever neutropenia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Sponsor decision to terminate the study
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Enzon Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Jun 2007
- Primary completion
- Jun 2009 (actual)
- Study completion
- Aug 2009 (actual)
- First posted
- 24 Aug 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is to find the dose of EZN-2232 that can be given to MBL deficient pediatric cancer patients undergoing chemotherapy. The pharmacokinetics, pharmacodynamics, and safety of the study drug will also be studied.
Detailed description
MBL deficient patients will be randomized in a ratio of 1:1 to receive a single dose of either 0.5 mg/kg or 1.0 mg/kg of intravenous rhMBL. A total of 24 patients will be treated in each of the rhMBL arms, (12 in the 0.5 mg/kg and 12 in the 1.0 mg/kg. All patients are to receive anti-infectious prophylactic supportive therapy as per institutional standards
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 0.5 mg/kg | Other | Drug: Intravenous recombinant human mannose binding lectin |
| 1.0 mg/kg | Other | Drug: Intravenous recombinant human mannose binding lectin |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Intravenous recombinant human mannose binding lectinIntravenous(i.v.) administration for one dose. Patients will be randomized to one of three cohorts: 0 mg/kg; 0.5 mg/kg, or 1.0 mg.kg | Drug | 0.5 mg/kg |
| Intravenous recombinant human mannose binding lectinIntravenous(i.v.) administration for one dose. Patients will be randomized to one of three cohorts: 0 mg/kg; 0.5 mg/kg, or 1.0 mg.kg | Drug | 1.0 mg/kg |
Primary outcome
Determine the safety and tolerability of intravenous rhMBL administered at dosages of 0.5 and 1.0 mg/kg to MBL deficient pediatric hematology/oncology patients during episodes of fever and neutropenia.
Time frame: 1 month
1 secondary outcome
Determine the PK profile of intravenous rhMBL as a single dose escalation cohort design in MBL deficient pediatric oncology patients during episodes of fever and neutropenia.
Time frame: 1 month
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2012.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Thomas Walsh, MD principal investigator
NIH/National Cancer Institute
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intravenous recombinant human mannose binding lectin in people with Cancer and Hematologic Diseases, run by Enzon Pharmaceuticals, Inc..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 5 Sep 2012. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine the safety and tolerability of intravenous rhMBL administered at dosages of 0.5 and 1.0 mg/kg to MBL deficient pediatric hematology/oncology patients during episodes of fever and neutropenia. (time frame: 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Sep 2012; AzTrials last refreshed its copy on 8 Nov 2026.