Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

Study Overview
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Brief Summary
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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
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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

Official Title
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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)

Conditions
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Mastocytosis

Intervention / Treatment
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* Drug: masitinib (AB1010)
* Drug: masitinib (AB1010)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 2<br>masitinib (AB1010) 6 mg/kg/day | Drug: masitinib (AB1010)<br>* 6 mg/kg/day<br>|
| Experimental: 1<br>masitinib (AB1010) 3 mg/kg/day | Drug: masitinib (AB1010)<br>* 3 mg/kg/day<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 |  | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AFIRMM score, reduction of organ infiltration,level of tryptase, reduction on bio markers (TNFα, eosinophils, histamine levels), pharmacokinetic profile of AB1010 |  | 12 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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mastocytosis, indolent, handicap, wild-type, systemic, not D816V mutated