MBL-DeficientPhase 2NCT00138736
Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia
A Phase 2 study testing Mannan Binding Lectin (MBL) and Mannan Binding Lectin in people with MBL-Deficient and Neutropenia, run by Prothya Biosolutions at 2 sites in Netherlands.
Trial summary
The pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied. Read more about this study
- Who is it for?
- Ages Up to 12 years
- Conditions the study is for
- MBL-Deficient, Neutropenia
- What is being tested?
- Mannan Binding Lectin (MBL) and Mannan Binding Lectin
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesNetherlands
- Study dates
- Apr 2004to Oct 2006
- Phase
- Phase 2
- 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 Up to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children ages 0 - 12 years, during chemotherapy, and expected to become neutropenic
- MBL deficiency by genotype or phenotype (< 100 ng/ml)
- Informed consent and assent of patient and/or legal representative
Who cannot join
- Inability or unwillingness to comply with the protocol or likely inability to complete the study period
- Known allergic reactions to MBL and other human plasma products
- Participation in other investigational drug studies within the last month
- Clinically relevant abnormalities in: serum immunoglobulins IgG, IgA, IgM; blood counts; complement factors measured by AP50, CH50; urine protein and cell counts; serum creatinine and liver enzymes, as routinely determined for regular patient care.
Trial locations and contacts
2 sites in 1 country.
Netherlands2 sites
Academic Medical Centre
Amsterdam, 1105 AZ
Erasmus Medical Centre
Rotterdam, 3015 GD
About this study
Official title: Phase II Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia
The pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied.
Read the detailed description
Mannan Binding Lectin (MBL) is a member of the lectin pathway of the complement system and plays an important role in the innate immune system. MBL replacement in MBL-deficient children with chemotherapy-induced neutropenia represents a new approach to lower the risk of febrile episodes, of hospital admission, of prolonged use of intravenous antibiotics and of severe infections.
The aim of the Phase II study is to find evidence for the correct prediction of plasma levels of MBL necessary for clinical effects and biological efficacy, to confirm the dosage regimen needed to reach the required MBL plasma levels, and reconfirm the safety and lack of side-effects.
- Conditions
- MBL-Deficient, Neutropenia
- Being tested
- Mannan Binding Lectin (MBL), Mannan Binding Lectin
- Keywords
- MBL-deficient, chemotherapy, neutropenia, chemotherapy-induced neutropenia
- Medical subject headings
- Neutropenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mannan Binding Lectin (MBL)Drug
Mannan Binding LectinDrug
Also called MBL SSI
MBL dose at a twice weekly dose interval (3 or 4 days):
0.2 mg/kg, for a 3-day interval; 0.3 mg/kg, for a 4-day interval
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | MBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood. Receives: Drug: Mannan Binding Lectin (MBL); Drug: Mannan Binding Lectin |
What is being measured
The primary outcome is the main question the study is designed to answer.
pharmacokinetics of MBL
Measured: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
Secondary outcomes (6)
days of fever
Measured: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
days of hospital admission
Measured: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
use of antibiotics or antifungal medication
Measured: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
number and type of infections
Measured: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
MBL-dependent opsonizing capacity in vitro
Measured: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
safety and incidence of side effects
Measured: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2005 (estimated)
- Study startApr 2004
- Study completionOct 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MBL-DeficientStudies 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 2
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prothya Biosolutions
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Landsteiner Foundation for Blood Transfusion
- Investigators
- T W Kuijpers, MD, PdD, Principal investigator, Dept. of Paediatric Immunology, AMC, Amsterdam, The NetherlandsH N Caron, Principal investigator, Dept of Paediatric Oncology, AMC, Amsterdam, The Netherlands
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00138736
- Sponsor's protocol ID
- KB2003.02
- Status last verified
- Aug 2007
- First submitted
- 29 Aug 2005
- Last updated
- 30 Aug 2007
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Aug 2007. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study on Mannan Binding Lectin (MBL) Substitution in MBL-Deficient Children With Chemotherapy-Induced Neutropenia
- ClinicalTrials.gov ID
- NCT00138736
- Acronym / sponsor ID
- KB2003.02
- Conditions
- MBL-Deficient, Neutropenia
- Keywords
- MBL-deficient, chemotherapy, neutropenia, chemotherapy-induced neutropenia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Prothya BiosolutionsIndustry (pharma, biotech, devices)
- Collaborators
- Landsteiner Foundation for Blood Transfusion
- Study start
- Apr 2004
- Study completion
- Oct 2006 (actual)
- First posted
- 30 Aug 2005 (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 pharmacokinetics, and clinical and biological effects of MBL replacement therapy in MBL-deficient children during chemotherapy-induced neutropenia were studied.
Detailed description
Mannan Binding Lectin (MBL) is a member of the lectin pathway of the complement system and plays an important role in the innate immune system. MBL replacement in MBL-deficient children with chemotherapy-induced neutropenia represents a new approach to lower the risk of febrile episodes, of hospital admission, of prolonged use of intravenous antibiotics and of severe infections.
The aim of the Phase II study is to find evidence for the correct prediction of plasma levels of MBL necessary for clinical effects and biological efficacy, to confirm the dosage regimen needed to reach the required MBL plasma levels, and reconfirm the safety and lack of side-effects.
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: Mannan Binding Lectin (MBL), Drug: Mannan Binding LectinMBL until the patient's absolute neutrophil count (ANC) is above 500/microL blood. |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Children ages 0 - 12 years, during chemotherapy, and expected to become neutropenic
- MBL deficiency by genotype or phenotype (< 100 ng/ml)
- Informed consent and assent of patient and/or legal representative
Key exclusion criteria (4 listed)
- Inability or unwillingness to comply with the protocol or likely inability to complete the study period
- Known allergic reactions to MBL and other human plasma products
- Participation in other investigational drug studies within the last month
- Clinically relevant abnormalities in: serum immunoglobulins IgG, IgA, IgM; blood counts; complement factors measured by AP50, CH50; urine protein and cell counts; serum creatinine and liver enzymes, as routinely determined for regular patient care.
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 |
|---|---|---|
| Mannan Binding Lectin (MBL) | Drug | A |
| Mannan Binding LectinAlso: MBL SSIMBL dose at a twice weekly dose interval (3 or 4 days): 0.2 mg/kg, for a 3-day interval; 0.3 mg/kg, for a 4-day interval | Drug | A |
Primary outcome
pharmacokinetics of MBL
Time frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
6 secondary outcomes
days of fever
Time frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
days of hospital admission
Time frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
use of antibiotics or antifungal medication
Time frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
number and type of infections
Time frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
MBL-dependent opsonizing capacity in vitro
Time frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
safety and incidence of side effects
Time frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Aug 2007.
Related trials for MBL-Deficient
Enrolling by invitationObservational study
Immune System in Diabetic Kidney Disease
Diabetes, Diabetic Kidney Disease
RecruitingObservational study
Association Between Plasma Level of Mannose Binding Lectin and Human Reproduction
Mannose-Binding Lectin Deficiency, Recurrent Pregnancy Loss
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.
T W Kuijpers, MD, PdD principal investigator
Dept. of Paediatric Immunology, AMC, Amsterdam, The Netherlands
H N Caron principal investigator
Dept of Paediatric Oncology, AMC, Amsterdam, The Netherlands
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Mannan Binding Lectin (MBL) and Mannan Binding Lectin in people with MBL-Deficient and Neutropenia, run by Prothya Biosolutions at 2 sites in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. 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: finished, as of the sponsor's last update on 30 Aug 2007. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Netherlands. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
pharmacokinetics of MBL (time frame: until the patient's absolute neutrophil count (ANC) is above 500/uL blood.). 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 30 Aug 2007.