Back to resultsEfficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
Primary Purpose
Mastocytosis
Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
masitinib (AB1010)
masitinib (AB1010)
Sponsored by
About this trial
This is an interventional treatment trial for Mastocytosis focused on measuring mastocytosis, indolent, handicap, wild-type, systemic, not D816V mutated
Eligibility Criteria
Inclusion Criteria:
- 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 ,
- 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
Exclusion Criteria:
- Performance status > 2 (ECOG).
- Inadequate organ function, except if the abnormalities are due to involvement by mast cells
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
2
1
Arm Description
masitinib (AB1010) 6 mg/kg/day
masitinib (AB1010) 3 mg/kg/day
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00831974
Brief Title
Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
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)
Study Type
Interventional
2. Study Status
Record Verification Date
December 2018
Overall Recruitment Status
Completed
Study Start Date
October 2004 (undefined)
Primary Completion Date
February 2007 (Actual)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AB Science
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief 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.
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mastocytosis
Keywords
mastocytosis, indolent, handicap, wild-type, systemic, not D816V mutated
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
23 (Actual)
8. Arms, Groups, and Interventions
Arm Title
2
Arm Type
Experimental
Arm Description
masitinib (AB1010) 6 mg/kg/day
Arm Title
1
Arm Type
Experimental
Arm Description
masitinib (AB1010) 3 mg/kg/day
Intervention Type
Drug
Intervention Name(s)
masitinib (AB1010)
Intervention Description
3 mg/kg/day
Intervention Type
Drug
Intervention Name(s)
masitinib (AB1010)
Intervention Description
6 mg/kg/day
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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 ,
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
Exclusion Criteria:
Performance status > 2 (ECOG).
Inadequate organ function, except if the abnormalities are due to involvement by mast cells
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Olivier Lortholary, MD, PhD
Organizational Affiliation
Hôpital Necker, Paris, France
Official's Role
Principal Investigator
12. IPD Sharing Statement
Citations:
PubMed Identifier
21108325
Citation
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.
Results Reference
result
Learn more about this trial
Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
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