MastocytosisPhase 2NCT00831974
Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
A Phase 2 study testing masitinib (AB1010) in people with Mastocytosis, run by AB Science.
Trial summary
This is a 12 weeks study aimed at assessing the safety and efficacy of 2 doses of AB1010 in patients suffering from indolent systemic mastocytosis with handicap. 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 (AB1010)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- No sites listed yet
- Study dates
- Oct 2004to Feb 2007
- 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 documented Indolent systemic mastocytosis with handicap (ISMwh) having at least 2 infiltrated* organs (skin and /or bone-marrow and/or internal organ).
- Bone-marrow, or skin or internal biopsy-documented mastocytosis and evaluable disease.
- The absence of an activating point mutation in the phosphotransferase domain of c-Kit such as D816V c-Kit mutation in at least one of the two infiltrated organs: bone marrow and/or skin and/or other tissue.
- Handicap defined as at least one of the following handicaps:
- a number of flush per day ≥ 1 ,
- a pruritus score ≥ 9 ,
Show all 10Show fewer
- a number of stools per day ≥ 4 ,
- a Pollakyuria (on a per day basis) ≥ 8 ,
- a QLQ-C30 score ≥ 83 ,
- a Hamilton rating scale for depression ≥ 12
Who cannot join
- Performance status > 2 (ECOG).
- Inadequate organ function, except if the abnormalities are due to involvement by mast cells
About this study
Official title: Phase IIa, Open-label, Randomized Study of Oral AB1010 in Patients With Systemic Indolent Mastocytosis With Handicap and Not Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)
This is a 12 weeks study aimed at assessing the safety and efficacy of 2 doses of AB1010 in patients suffering from indolent systemic mastocytosis with handicap.
Read the detailed description
Efficacy will be assessed based on:
Pruritus score Number of flush per day Pollakyuria (on a daily basis) Number of stools per day QLQ-C30 score Hamilton Rating Scale for depression
- Conditions
- Mastocytosis
- Being tested
- masitinib (AB1010)
- Keywords
- mastocytosis, indolent, handicap, wild-type, systemic, not D816V mutated
- Medical subject headings
- Mastocytosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
masitinib (AB1010)Drug
3 mg/kg/day
masitinib (AB1010)Drug
6 mg/kg/day
| Study group | Role | What they receive |
|---|---|---|
| 2 | Experimental | masitinib (AB1010) 6 mg/kg/day Receives: Drug: masitinib (AB1010) |
| 1 | Experimental | masitinib (AB1010) 3 mg/kg/day Receives: Drug: masitinib (AB1010) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Response on: Pruritus score, Number of flush per day, Pollakyuria (on a daily basis), Number of stools per day, QLQ-C30 score, Hamilton Rating Scale for depression
Measured: 12 weeks
Secondary outcomes (1)
AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010
Measured: 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2009 (estimated)
- Study startOct 2004
- Primary completionFeb 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- 23 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, Hôpital Necker, Paris, France
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00831974
- Sponsor's protocol ID
- AB04010
- Status last verified
- Dec 2018
- First submitted
- 28 Jan 2009
- Last updated
- 11 Dec 2018
- Results posted
- No
- Data monitoring committee
- No
Publications
Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmerini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol. 2010 Dec;85(12):921-5. doi: 10.1002/ajh.21894.
PubMed 21108325
Study at a glance
From the registry record, last updated by the sponsor on 11 Dec 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase IIa, Open-label, Randomized Study of Oral AB1010 in Patients With Systemic Indolent Mastocytosis With Handicap and Not Bearing Activating Point Mutations in the Phosphotransferase Domain of c-Kit Such as the Main Mutation Asp-816-Val (D816V)
- ClinicalTrials.gov ID
- NCT00831974
- Acronym / sponsor ID
- AB04010
- Conditions
- Mastocytosis
- Keywords
- mastocytosis, indolent, handicap, wild-type, systemic, not D816V mutated
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- AB ScienceIndustry (pharma, biotech, devices)
- Study start
- Oct 2004
- Primary completion
- Feb 2007 (actual)
- First posted
- 29 Jan 2009 (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 12 weeks study aimed at assessing the safety and efficacy of 2 doses of AB1010 in patients suffering from indolent systemic mastocytosis with handicap.
Detailed description
Efficacy will be assessed based on:
Pruritus score Number of flush per day Pollakyuria (on a daily basis) Number of stools per day QLQ-C30 score Hamilton Rating Scale for depression
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 2 | Experimental | Drug: masitinib (AB1010)masitinib (AB1010) 6 mg/kg/day |
| 1 | Experimental | Drug: masitinib (AB1010)masitinib (AB1010) 3 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 (10 listed)
- Patients with documented Indolent systemic mastocytosis with handicap (ISMwh) having at least 2 infiltrated* organs (skin and /or bone-marrow and/or internal organ).
- Bone-marrow, or skin or internal biopsy-documented mastocytosis and evaluable disease.
- The absence of an activating point mutation in the phosphotransferase domain of c-Kit such as D816V c-Kit mutation in at least one of the two infiltrated organs: bone marrow and/or skin and/or other tissue.
- Handicap defined as at least one of the following handicaps:
Key exclusion criteria (2 listed)
- Performance status > 2 (ECOG).
- Inadequate organ function, except if the abnormalities are due to involvement by mast cells
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 (AB1010)3 mg/kg/day | Drug | 1 |
| masitinib (AB1010)6 mg/kg/day | Drug | 2 |
Primary outcome
Response on: Pruritus score, Number of flush per day, Pollakyuria (on a daily basis), Number of stools per day, QLQ-C30 score, Hamilton Rating Scale for depression
Time frame: 12 weeks
1 secondary outcome
AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010
Time frame: 12 weeks
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 11 Dec 2018.
- Results publication: PubMed 21108325Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmerini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol. 2010 Dec;85(12):921-5. doi: 10.1002/ajh.21894.
Related trials for Mastocytosis
RecruitingObservational study
New Italian Mastocytosis Registry
Systemic Mastocytosis
RecruitingObservational study
Tissue, Blood, and Body Fluid Sample Collection From Patients With Hematologic Cancer
Chronic Myeloproliferative Disorders, Leukemia
RecruitingObservational study
Study of Factors Regulating Mast Cell Proliferation
Mastocytosis, Monoclonal
Active, not recruitingObservational study
Infection Prophylaxis and Management in Treating Cytomegalovirus (CMV) Infection in Patients With Hematologic Malignancies Previously Treated With Donor Stem Cell Transplant
Hematopoietic/Lymphoid Cancer, Accelerated Phase Chronic Myelogenous Leukemia
Active, not recruitingPhase 2
(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis
SSM, Mastocytosis, Indolent
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
Hôpital Necker, Paris, France
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing masitinib (AB1010) 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 10 inclusion and 2 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 11 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?
Response on: Pruritus score, Number of flush per day, Pollakyuria (on a daily basis), Number of stools per day, QLQ-C30 score, Hamilton Rating Scale for depression (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00831974, which also lists 1 linked publication.
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 11 Dec 2018.