Liver TransplantationPhase 1NCT00415311
A Multicenter Study Evaluating the Safety and Tolerability of Intravenous rhMBL in Liver Transplant Recipients.
A Phase 1 study testing Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) in people with Liver Transplantation, run by Enzon Pharmaceuticals, Inc. at 4 sites in United States.
Trial summary
This is a multicenter, open-label, randomized, Phase 1B study evaluating liver transplant recipients receiving rhMBL (2 cohorts) or without rhMBL (1 cohort). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Liver Transplantation
- What is being tested?
- Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 4 sitesUnited States
- Study dates
- Dec 2006to Dec 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
- Scheduled to undergo OLT or LRD liver transplantation (single organ). Split grafts will not be allowed from OLT donors.
- Donor has an MBL genotype of A/O or O/O.
- Age ≥18 years old.
- Willing to receive transfusions of blood products.
Any patient who has given informed consent to participate in the clinical study and who meets all entry criteria for the study may participate in the genetic part of the study.
Exclusion Criteria: Patients meeting any of the following exclusion criteria will not be eligible for enrollment.
- Concurrent serious medical illness, in the judgment of the principal investigator (PI), which could potentially interfere with protocol compliance.
- Positive screening pregnancy test or is breast-feeding.
- Female or male patient of reproductive capacity unwilling to use methods appropriate to prevent pregnancy during this study.
- Any condition that, in the opinion of the PI or Enzon makes the patient unsuitable for the study.
- Current participation in another clinical study with an investigational agent and/or use of an investigational drug (not including investigational use of an approved drug) in the 30 days before the first dose of rhMBL.
- Prior liver transplants.
- Systemic chemotherapy within 1 year before liver transplantation.
- Serum creatinine >5 mg/dL.
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Vanderbilt University
Nashville, Tennessee, 372321
Mount Sinai School of Medicine
New York, New York, 10029
Nebraska Medical Center
Omaha, Nebraska, 68198-7400
University of California San Francisco
San Francisco, California, 94143
About this study
Official title: A Multicenter, Open-Label, Randomized, Phase 1B Study Evaluating the Safety and Tolerability of Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) in Liver Transplant Recipients
This is a multicenter, open-label, randomized, Phase 1B study evaluating liver transplant recipients receiving rhMBL (2 cohorts) or without rhMBL (1 cohort).
Read the detailed description
This is a multicenter, open-label, randomized, Phase 1B study evaluating liver transplant recipients receiving rhMBL (2 cohorts) or without rhMBL (1 cohort).
Patients will have received an orthotopic liver transplant (OLT) or a living related donor (LRD) liver transplant. Patients in all cohorts are to receive immunosuppressant therapy and anti-infectious prophylactic supportive therapy according to institutional standards.
The donor's mannose-binding lectin (MBL) genotype will be evaluated to determine the liver transplant recipient's study eligibility. For recipients receiving an OLT, a sample of liver tissue or lymph nodes will be collected from the donor liver at the time of organ harvest for MBL genotyping. For recipients receiving a LRD transplant, the MBL genotype of the LRD will be determined in a companion protocol, "Screening Protocol to Evaluate Mannose-Binding Lectin (MBL) Genotype in Living Related Donors for Liver Transplant Recipients." A recipient whose donor has an A/O or O/O MBL genotype will be eligible to participate in this study.
Patients will be randomized in a 2:2:1 ratio to receive up to 8 intravenous (i.v.) infusions of rhMBL at a dose of 0.5 or 1.0 mg/kg, or to receive no rhMBL, respectively. Approximately 20 patients will be treated in each of the 2 rhMBL arms, and approximately 10 patients will be treated with standard immunosuppressive agents and anti-infectious prophylaxis but not with rhMBL.
Cohort 1
- Number of Patients 20
- rhMBL (mg/kg) 0.5
Cohort 2
- Number of Patients 20
- rhMBL (mg/kg) 1.0
Cohort 3
- Number of Patients 10
- rhMBL (mg/kg) None
- Conditions
- Liver Transplantation
- Being tested
- Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)
- Keywords
- Liver Transplantation, rhMBL
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)Drug
Intravenous(i.v.) administration for 8 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 8 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 8 weeks. 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 mg/kg | Other | Not described Receives: Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) |
| 0.5 mg/kg | Other | Not described Receives: Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) |
| 1.0 mg/kg | Other | Not described Receives: Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the safety and tolerability of rhMBL administered i.v. at doses of 0.5 and 1.0 mg/kg, as 8 weekly doses over 8 weeks, in the setting of liver transplantation
Measured: 3 months
Secondary outcomes (4)
Determine the pharmacokinetic (PK) profile of rhMBL
Measured: 3 months
Determine the pharmacodynamics (PD) (complement deposition) of rhMBL
Measured: 3 months
Determine the immunogenicity of rhMBL by testing for the presence of anti-rhMBL antibodies
Measured: 3 months
Detect preliminary evidence of activity of rhMBL by analyzing the incidence of infectious complications.
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Dec 2006 (estimated)
- Study startDec 2006
- Primary completionJun 2009
- Study completionDec 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 Liver TransplantationStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Enzon Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Alison Freifeld, MD, Principal investigator, University of Nebraska Medical Center, 985400 Nebraska Medical Center, Omaha, NE 68198-5400
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00415311
- Sponsor's protocol ID
- EZN-2232-02
- Status last verified
- Aug 2011
- First submitted
- 20 Dec 2006
- Last updated
- 9 Aug 2011
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-Label, Randomized, Phase 1B Study Evaluating the Safety and Tolerability of Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) in Liver Transplant Recipients
- ClinicalTrials.gov ID
- NCT00415311
- Acronym / sponsor ID
- EZN-2232-02
- Conditions
- Liver Transplantation
- Keywords
- Liver Transplantation, rhMBL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision to terminate the study
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Enzon Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2006
- Primary completion
- Jun 2009 (actual)
- Study completion
- Dec 2009 (actual)
- First posted
- 22 Dec 2006 (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.
This is a multicenter, open-label, randomized, Phase 1B study evaluating liver transplant recipients receiving rhMBL (2 cohorts) or without rhMBL (1 cohort).
Detailed description
This is a multicenter, open-label, randomized, Phase 1B study evaluating liver transplant recipients receiving rhMBL (2 cohorts) or without rhMBL (1 cohort).
Patients will have received an orthotopic liver transplant (OLT) or a living related donor (LRD) liver transplant. Patients in all cohorts are to receive immunosuppressant therapy and anti-infectious prophylactic supportive therapy according to institutional standards.
The donor's mannose-binding lectin (MBL) genotype will be evaluated to determine the liver transplant recipient's study eligibility. For recipients receiving an OLT, a sample of liver tissue or lymph nodes will be collected from the donor liver at the time of organ harvest for MBL genotyping. For recipients receiving a LRD transplant, the MBL genotype of the LRD will be determined in a companion protocol, "Screening Protocol to Evaluate Mannose-Binding Lectin (MBL) Genotype in Living Related Donors for Liver Transplant Recipients." A recipient whose donor has an A/O or O/O MBL genotype will be eligible to participate in this study.
Patients will be randomized in a 2:2:1 ratio to receive up to 8 intravenous (i.v.) infusions of rhMBL at a dose of 0.5 or 1.0 mg/kg, or to receive no rhMBL, respectively. Approximately 20 patients will be treated in each of the 2 rhMBL arms, and approximately 10 patients will be treated with standard immunosuppressive agents and anti-infectious prophylaxis but not with rhMBL.
Cohort 1
- Number of Patients 20
- rhMBL (mg/kg) 0.5
Cohort 2
- Number of Patients 20
- rhMBL (mg/kg) 1.0
Cohort 3
- Number of Patients 10
- rhMBL (mg/kg) None
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 0 mg/kg | Other | Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) |
| 0.5 mg/kg | Other | Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) |
| 1.0 mg/kg | Other | Drug: Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 Lectin (rhMBL)Intravenous(i.v.) administration for 8 weeks. Patients will be randomized to one of three cohorts: 0 mg/kg; 0.5 mg/kg, or 1.0 mg.kg | Drug | 0 mg/kg |
| Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL)Intravenous(i.v.) administration for 8 weeks. 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 Lectin (rhMBL)Intravenous(i.v.) administration for 8 weeks. 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 rhMBL administered i.v. at doses of 0.5 and 1.0 mg/kg, as 8 weekly doses over 8 weeks, in the setting of liver transplantation
Time frame: 3 months
4 secondary outcomes
Determine the pharmacokinetic (PK) profile of rhMBL
Time frame: 3 months
Determine the pharmacodynamics (PD) (complement deposition) of rhMBL
Time frame: 3 months
Determine the immunogenicity of rhMBL by testing for the presence of anti-rhMBL antibodies
Time frame: 3 months
Detect preliminary evidence of activity of rhMBL by analyzing the incidence of infectious complications.
Time frame: 3 months
Results and safety
The study was stopped early (reason given: Sponsor decision to terminate the study). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2011.
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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.
Alison Freifeld, MD principal investigator
University of Nebraska Medical Center, 985400 Nebraska Medical Center, Omaha, NE 68198-5400
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Intravenous Recombinant Human Mannose-Binding Lectin (rhMBL) in people with Liver Transplantation, run by Enzon Pharmaceuticals, Inc. at 4 sites in United States.
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: stopped early, as of the sponsor's last update on 9 Aug 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
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 stopped early (reason given: Sponsor decision to terminate the study). Results are not available in the source record.
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 rhMBL administered i.v. at doses of 0.5 and 1.0 mg/kg, as 8 weekly doses over 8 weeks, in the setting of liver transplantation (time frame: 3 months). 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 9 Aug 2011.