MastocytosisPhase 2NCT01266369
Masitinib in Patients With Mastocytosis With Handicap and Bearing the D816V Mutation
A Phase 2 study testing masitinib in people with Mastocytosis, run by AB Science.
Trial summary
The objective is to compare efficacy and safety of AB1010 at 3 or 6 mg/kg/day in treatment of patients with mastocytosis with handicap and bearing activating point mutations in the phosphotransferase domain of c-Kit such as the main mutation Asp-816-Val (D816V). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Mastocytosis
- What is being tested?
- masitinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- No sites listed yet
- Study dates
- Feb 2007to May 2010
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with one of the following documented mastocytosis:
- Smouldering systemic mastocytosis
- Indolent systemic mastocytosis with organomegaly
- Indolent Systemic Mastocytosis having 2 infiltrated organs (skin and bone-marrow)
- Any mastocytosis with in the last 6 months at least 3 anaphylactic shocks or syncops requiring either use of adrenaline or medical assistance
- Cutaneous Mastocytosis (CM)
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- Skin biopsy-documented mastocytosis and evaluable disease based upon:
- Histological criteria: typical infiltrates of mast cells in a multifocal or diffuse pattern in skin biopsy
- Clinical criteria: typical skin lesions (maculopapular, urticaria pigmentosa, mastocytoma)
- Missing data (c-kit molecular analysis not done) or documented presence of an activating point mutation in the phosphotransferase domain of c-kit such as D816V c-kit mutation in at least one infiltrated organ (bone marrow or skin)
- Refractory to at least one of the symptomatic treatments such as:
- Anti H1
- Anti H2
- Proton pump inhibitor
- Osteoclast inhibitor
- Cromoglycate Sodium
- Antileukotriene
- Other therapies used for the symptomatic care
- Handicap defined as at least one of the following handicaps:
- pruritus score ≥ 6
- number of flushes per week ≥ 7
- number of stools per day ≥ 4 ,
- number of mictions per day ≥ 8 ,
- QLQ-C30 score ≥ 60,
- Hamilton score ≥ 10
Who cannot join
- Patients with one of the following mastocytosis:
- Systemic Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease (SM-AHNMD)
- Mast cell leukemia (MCL)
- Aggressive systemic mastocytosis (ASM)
- Patient with a major surgery within 2 weeks prior to study entry
- No vulnerable population will be included in this study
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- Life expectancy < 6 months.
- Patient is < 5 years free of malignancy, except treated basal cell skin cancer or cervical carcinoma in situ.
- Patient with grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study)
- Patient has a severe and/or uncontrolled medical disease.
- Patient has a known diagnosis of human immunodeficiency virus (HIV) infection
About this study
Official title: A 12-week With Possible Extension, Prospective, Multicenter, Randomized, Open-label, 2-parallel Group, Phase IIa Study to Compare Efficacy and Safety of AB1010 at 3 or 6 mg/kg/Day in Treatment of Patients With Mastocytosis With Handicap and Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)
The objective is to compare efficacy and safety of AB1010 at 3 or 6 mg/kg/day in treatment of patients with mastocytosis with handicap and bearing activating point mutations in the phosphotransferase domain of c-Kit such as the main mutation Asp-816-Val (D816V).
- Conditions
- Mastocytosis
- Being tested
- masitinib
- Keywords
- mastocytosis, D816V, handicaps
- Medical subject headings
- Mastocytosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
masitinibDrug
| Study group | Role | What they receive |
|---|---|---|
| masitinib 3 mg/kg/day | Experimental | masitinib 3 mg/kg/day Receives: Drug: masitinib |
| masitinib 6 mg/kg/day | Experimental | masitinib 6 mg/kg/day Receives: Drug: masitinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
efficacy on handicaps
Pruritus score at week 12 Number of flushes per week at week 12 Hamilton score at week 12 Fatigue Impact scale at week 12
Measured: week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2010 (estimated)
- Study startFeb 2007
- Primary completionNov 2007
- Study completionMay 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MastocytosisStudies 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
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- AB Science
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Olivier Lortholary, MD, PhD, Principal investigator, Necker Hospital, Paris, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01266369
- Sponsor's protocol ID
- AB06013
- Status last verified
- Dec 2018
- First submitted
- 23 Dec 2010
- Last updated
- 10 Dec 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 12-week With Possible Extension, Prospective, Multicenter, Randomized, Open-label, 2-parallel Group, Phase IIa Study to Compare Efficacy and Safety of AB1010 at 3 or 6 mg/kg/Day in Treatment of Patients With Mastocytosis With Handicap and Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)
- ClinicalTrials.gov ID
- NCT01266369
- Acronym / sponsor ID
- AB06013
- Conditions
- Mastocytosis
- Keywords
- mastocytosis, D816V, handicaps
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- AB ScienceIndustry (pharma, biotech, devices)
- Study start
- Feb 2007
- Primary completion
- Nov 2007 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 24 Dec 2010 (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 objective is to compare efficacy and safety of AB1010 at 3 or 6 mg/kg/day in treatment of patients with mastocytosis with handicap and bearing activating point mutations in the phosphotransferase domain of c-Kit such as the main mutation Asp-816-Val (D816V).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| masitinib 3 mg/kg/day | Experimental | Drug: masitinibmasitinib 3 mg/kg/day |
| masitinib 6 mg/kg/day | Experimental | Drug: masitinibmasitinib 6 mg/kg/day |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (25 listed)
- Patients with one of the following documented mastocytosis:
- Skin biopsy-documented mastocytosis and evaluable disease based upon:
- Missing data (c-kit molecular analysis not done) or documented presence of an activating point mutation in the phosphotransferase domain of c-kit such as D816V c-kit mutation in at least one infiltrated organ (bone marrow or skin)
- Refractory to at least one of the symptomatic treatments such as:
- Handicap defined as at least one of the following handicaps:
Key exclusion criteria (11 listed)
- Patients with one of the following mastocytosis:
- Patient with a major surgery within 2 weeks prior to study entry
- No vulnerable population will be included in this study
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 |
|---|---|---|
| masitinib | Drug | masitinib 3 mg/kg/day, masitinib 6 mg/kg/day |
Primary outcome
efficacy on handicaps
Time frame: week 12
Pruritus score at week 12 Number of flushes per week at week 12 Hamilton score at week 12 Fatigue Impact scale at week 12
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 10 Dec 2018.
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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.
Olivier Lortholary, MD, PhD principal investigator
Necker Hospital, Paris, France
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing masitinib in people with Mastocytosis, run by AB Science.
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 25 inclusion and 11 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 10 Dec 2018. 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 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?
efficacy on handicaps (time frame: week 12). 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 10 Dec 2018.