Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis
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.
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
Inclusion Criteria:

 1. Patients with documented Indolent systemic mastocytosis with handicap (ISMwh) having
 at least 2 infiltrated* organs (skin and /or bone-marrow and/or internal organ).

 2. Bone-marrow, or skin or internal biopsy-documented mastocytosis and evaluable disease.

 3. 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.

 4. 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:

 1. Performance status > 2 (ECOG).

 2. Inadequate organ function, except if the abnormalities are due to involvement by mast
 cells