Efficacy of AB1010 in Patients With Systemic Indolent Mastocytosis

• Status: Completed
• Trial end date: 1969-12-31
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AB Science

Criteria

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